Definitie van ADHD
Mensen die ADHD hebben, hebben veel moeite met concentratie, het houden van hun aandacht bij datgene waarmee waar mee ze bezig zijn. Tevens zijn ze hyperactief, erg druk in hun gedrag.
Samengevat zijn mensen met ADHD rusteloos, impulsief en hebben ze concentratieproblemen.
Bij wie komt het voor?
We weten nu dat ADHD op alle leeftijden voorkomt, dus niet alleen bij kinderen.
Het is wel zo dat het op kinderleeftijd begint en deze mensen hebben een grote kans dat ze het hun leven lang houden.
Kinderen zijn zich nog aan het ontwikkelen en zijn nog druk met leren, dus deze leeftijdsgroep heeft er erg veel last van op school.
Volwassenen hebben er erg veel last van in hun werk.
Ze zijn chaotisch, rusteloos, komen vaak te laat, praten druk en veranderen veel van baan.
Beloop in de tijd.
Van de mensen die voor ADHD behandeld worden houdt een derde deze aandoening, bij een derde blijft de klachten bestaan in een mildere vorm.
Bij de laatste eenderde verdwijnen de klachten helemaal.
Hoe vaak komt het voor.
Ongeveer een op de vijfentwintig kinderen heeft ADHD, dit zijn dus 60.000 a 100.000 kinderen in Nederland. Bij de jongvolwassenen komt het bij 1 a 3 % van de personen voor.
Het aantal ADHD patiënten blijft stabiel, maar het wordt steeds beter herkent, zodat het steeds vaker behandeld wordt.
Verschijnselen – Aandachtsproblemen
Het kind heeft de meeste dagen van de week, minimaal 6 van de volgende verschijnselen:
• Onvoldoende aandacht voor details, maakt achteloos fouten.
• Heeft moeite om de aandacht bij de taak of spel te houden.
• Wekt de indruk niet te luisteren.
• Volgt aanwijzingen of maakt opdrachten niet af.
• Het kind heeft moeite met het organiseren van dingen die het doet.
• Het kind gaat taken/opdrachten uit de weg, het kind vermijd dingen die geestelijke inspanning vragen. Of het heeft een hekel aan dit soort activiteiten.
• Het kind raakt vaak dingen kwijt.
• Het kind is snel afgeleid.
• Het kind is vergeetachtig.
Verschijnselen – Hyperactiviteit
Er is ook sprake van ADHD als het kind de meeste dagen van de week zes of meer verschijnselen heeft van hyperactiviteit:
• Het kind beweegt onrustig met handen en voeten en zit te draaien op zijn stoel.
• Staat op, als het moet blijven zitten.
• Gaat rondrennen of overal op klimmen op plaatsen waar dat niet hoort.
• Kan moeilijk rustig spelen of ontspannende dingen doen.
• Is de hele tijd in de weer en draaft maar door.
• Praat aan een stuk door.
Of van impulsiviteit:
• Het kind antwoordt al, voordat de vraag helemaal gesteld is. Hij gooit het antwoord er als het ware uit.
• Het heeft moeite om op zijn beurt te wachten.
• Het verstoort bezigheden van anderen.
Een kind heeft pas ADHD, als het deze verschijnselen duidelijk meer heeft dan kinderen van dezelfde leeftijd, als het dagelijkse doen en laten er flink door beperkt wordt, en als dat allemaal minstens 6 maanden duurt.
Verder moet het kind enkele verschijnselen al voor zijn 7e jaar hebben gehad.
Volwassenen kunnen ook ADHD hebben, maar alleen als ze deze aandoening ook als kind hadden.
Oorzaken
ADHD wordt door meerdere factoren veroorzaakt, vanuit de omgeving, persoonsgebonden factoren, maar ook erfelijk.
• Jongens hebben de aandoening 2 a 3 maal vaker dan meisjes.
• Volwassen mannen hebben het even vaak als volwassen vrouwen.
• Erfelijkheid speelt een grote rol.
• Kinderen van ouders met ADHD hebben 50% kans om het ook te krijgen.
• Broertjes en zusjes van kinderen met ADHD hebben 3 a 5 maal zoveel kans om het ook te krijgen.
• Neefjes en nichtjes hebben 2 a 3 maal zoveel kans.
• In de hersenen van mensen met ADHD zijn afwijkingen gevonden mbt. de anatomie en de activiteit van verschillende hersendelen.
• Het kind loopt extra risico als de moeder tijdens de zwangerschap: rookte, hoge bloeddruk had of veel alcohol gebruikte.
• Een laag geboorte gewicht is ook een risicofactor.
Indicaties
circulatiestilstand gevolgd door apnoe;
respiratoire insufficiëntie met circulatoir arrest.
Algemene handelwijze (in volgorde van belangrijkheid)
begin hartmassage met frequentie circa 80 per minuut;
begin beademing via mond-op-mond of via mond/neus-masker; tracht zo spoedig mogelijk over te gaan op beademing via endotracheale tube, zeker wanneer reanimatie naar verwachting langer gaat duren. Verhouding beademing: hartmassage dient 1:5 te zijn;
alarmeer reanimatieteam en meld duidelijk waar reanimatie plaats heeft (op afdeling cardiologie bevindt zich een eigen noodbel);
zorg voor hartritmediagnostiek/-bewaking, wat soms ook mogelijk is via paddles van defibrillator;
zorg voor intraveneuze toegangsweg; indien niet mogelijk via armvene, dan via v. jugularis interna, v. subclavia of v. femoralis. Als reanimatie niet direct succes heeft, geniet een centrale lijn de voorkeur. Als geen i.v. toegangsweg verkregen kan worden dan, indien mogelijk, intratracheaal medicatie toedienen (de gebruikelijke dosering dan 2-3 maal verhogen);
verzamel zo veel mogelijk informatie over de patiënt:
voorgeschiedenis;
actuele ziektegeschiedenis;
prognose;
behandelend specialist;
recent ECG;
recent laboratoriumonderzoek.
verricht een beperkt lichamelijk onderzoek:
centraal-veneuze druk (CVD);
ademgeruis (spanningspneumothorax);
palpatie abdomen (aneurysma aortae abdominalis).
bepaal (geregeld) arteriële bloedgasanalysewaarden, veneuze bloedgasanalysewaarden, elektrolyten, glucose en Hb.
reanimatiepogingen staken indien alle mogelijke onderliggende factoren/ oorzaken zo optimaal mogelijk zijn gecorrigeerd (correctie zuur/base-evenwicht (pH>7,20), correctie pO2(>8 kPa), correctie elektrolytenstoornis, correctie tamponade, correctie lichaamstemperatuur, antiaritmische therapie, enz.) en desondanks adem-hartstilstand blijft bestaan.
Specifieke protocollen:
ventrikel fibrilleren/VT met bewustzijnsverliesventrikeltachycardie zonder bewustzijnsverlies
asystolie
elektromechanische dissociatie
extreme bradycardie
Na 10de blok bij persisterend VF of VT:
massage en beademing
iedere 5 minuten: adrenaline 1 mg i.v.
indien men vanuit het specifieke protocol wederom in dit protocol terecht komt, dan dit protocol vervolgen waar dit verlaten is;
geen bicarbonaat in de eerste 15 minuten, nadien alleen als pH <7,1 in dosering van 50 ml bicarbonaat 8,4%;
adrenaline via de tube oplossen met NaCl 0,9% tot 10 ml;
calcium alleen bij hyperkaliëmie of overdosering calciumantagonisten;
magnesiumsulfaat overwegen bij diureticagebruik;
getriggerde ECV verrichten onder algehele anesthesie (bv. propofol);
na toediening propofol niet langer varen op bewustzijn, maar op pols, tensie en ademhaling.
indien men vanuit het specifieke protocol wederom in dit protocol terecht komt, dan dit protocol vervolgen waar dit verlaten is;
indien tussentijds ander ritme ontstaat: specifiek protocol volgen;
adrenaline via de tube oplossen met NaCl 0,9% tot 10 ml;
geen bicarbonaat in de eerste 15 minuten geven, nadien alleen als pH <7,1 in dosering van 50 ml bicarbonaat 8,4%;
eventueel defibrillatie overwegen.
diagnostiek van de oorzaak van EMD van het allergrootste belang zodat specifieke behandeling kan gegeven worden.
eventueel externe pacemaker inbrengen voor invasief pacen;
bij bewustzijnsverlies hartmassage toepassen;
bij insufficiëntie ademhaling (bij-)beademen;
bij adequate circulatie kan isoprenaline infuus worden gegeven op geleide van de hartfrequentie.
Non-invasief pacen
plaats pads op thorax- en rugzijde patiënt;
sluit de pads aan op defibrillator door connectie van de paddles los te halen en deze vast te maken aan de pads;
kies pacer op de monitor van de defibrillator;
stel pacing frequentie in en voer pacing energie op tot capture is bereikt en mechanische output verzekerd.
the short version
Hiking and camping in rural Latin America can lead to exposure to the biting insect which carries this serious disease. The only preventives are education and information. There are no preventive medications or vaccines. Treatment exists, although it is often not completely satisfactory.
the long version
Valle de los Naranjos, Venezuela. It is late afternoon and the sun is sinking behind the mountains, bringing the first shadows of the evening. Down in the valley a campesino is tilling the soil; the quiet of the approaching night is broken only by a crop duster which periodically flies overhead and disappears further down the valley. Bertoldo, the pilot, is on his final run of the day when suddenly the engine dies, sending the plane onto the plowed field an emergency landing. The campesino drags him, stunned but unhurt, from the plane and offers his home for the night, a poorly constructed dwelling common in rural areas of Latin America known as el rancho (the hut) with walls of adobe, a dirt floor and a roof of palm fronds. On his ride back to Valencia the next day, Bertoldo reflects on his good fortune that he had only a few insect bites to show for his adventure. Bertoldo’s luck was short-lived. Two weeks after the night spent in the campesino’s hut, he came down with a fever and the right side of his face became red and puffy. He grew progressively weaker and a few weeks later he was in heart failure. As the result of the bite of an insect known as the vinchuca, Bertoldo had become infected by a parasite, Trypansoma cruzi, the cause of Chaga’s Disease. Three months later Bertoldo was dead.
THE VINCHUCA
The carrier of the Chagas’ Disease parasite is an inch long insect which depends on the blood of mammals to survive. The insect has a limited flight range; although it is furnished with two pairs of wings, these are used mainly as a parachute. “Vinchuca”, derived from the Quechua word huinchucaum, means “one who lets himself fall down”. The vinchuca is nocturnal, hiding during the day in crevices of walls of peasant huts and among palm fronds of the roof, coming out at night to feed. It is attracted to exposed parts of the body and has a preference for the face. In Europe and North America it is called the kissing or assassin bug. When the vinchuca finds the exposed face of a human victim, it releases from its proboscis a stylet with fine teeth which perforates the skin. A second hollow stylet taps a blood vessel. The feeding process lasts at least twenty minutes during which the vinchuca ingests several times its own weight in blood. Feeding is painless and the subject rarely wakens. During the feeding defecation occurs, contaminating the bite wound with feces containing parasites. Following feeding, the irritation of the bite causes the sleeping victim to rub the site, facilitating the introduction of the organism into the blood. The parasites are also capable of penetrating the thin layer of cells covering the lining of the mouth, nostrils and eye if carried there by contaminated fingers.
THE DISEASE
After one or two weeks the bite area, usually the face, becomes swollen and inflamed. This swelling, called a “chagoma” after Dr. Carlos Chagas who first described it, represents a reaction to the parasite, some of which reach the bloodstream and invade internal organs. Fever, vomiting, shortness of breath, convulsions and rigidity of the neck are symptoms of the acute generalized form of the illness which may result in rapidly progressive heart disease. Usually, however, the skin inflammation subsides and the victim continues to live an apparently healthy life. Unrecognized infection (but with a positive blood test indicating latent disease) may continue and progress slowly for years, surfacing after ten or twenty years as chronic heart disease. During this time, irreversible heart failure may appear, often with death occurring within a year.
SOCIAL IMPLICATIONS
Chagas’ Disease is a serious health problem in rural Latin America affecting mostly poor rural families. Since the 1960s, migration of rural populations has brought the infection to the periphery of large cities; in some countries the disease has reached the interior of urban areas. Besides spreading the infection to towns, this migration poses an additional risk through blood transfusions. Poor people are eager to sell their blood; infected blood donors sometimes slip through screening procedures. A recent survey in Sao Paulo shows that an average of fifteen percent of blood donors carry the infection, ten percent in Buenos Aires.
IMPACT OF CHAGAS’ DISEASE ON THE INTERNATIONAL TRAVELER
Because of the nature of their travel, hikers and campers are particularly vulnerable to Chagas’ Disease; it is essential that they know something of the disease and how to avoid it. Business travelers spending the night in the periphery or suburbs of cities ought at a minimum to check for insects in bedrooms. Chagas’ Disease is insidious-a traveler passing through Latin America could become infected and may remain apparently healthy until the first signs of chronic heart disease appear years later. Cases of Chagas’ Disease are often not recognized by doctors outside of Latin America since the symptoms of the late form are the same as other forms of chronic heart disease.
TO PREVENT CHAGAS’ DISEASE DURING YOUR TRAVELS
When traveling in areas with Chagas’ Disease, do not sleep in native huts, since parasite-carrying vinchucas hide in palm-frond roofs and the cracks of walls.
When checking into modest or older hotels, search for hidden insects under the mattress, behind pictures, in drawers or in dark corners of the room.
Before bed, apply insect repellent (available in sprays, lotions and towelettes) to exposed areas of skin. Any commercial preparation containing DEET (N.N-diethyl-meta-toluamide) is suitable.
Use a pyrethrin-containing insecticide (spray or coils) to kill insects which may be present in your sleeping quarters. Spray under your bed, inside closets, drawers and behind pictures.
Use a permethrin (Permanone) preparation on bednetting and bed clothing if at risk for bites from this crawling insect.
Protect your hands with a piece of cloth, paper, plastic or gloves if it is necessary to handle an insect.
Consider using bed nets to prevent contact with insects. Put a cloth over the bed net to prevent infected feces falling on you from the ceiling.
When camping, choose a campsite away from palm trees and stone piles where insects often hide.
If you require medical or surgical treatment involving blood transfusions, avoid private hospitals (clinicas) where blood donors may not have been adequately screened; there is apparently no risk of becoming infected by a blood transfusion at a university or municipal hospital in a major city.
Adapted from material distributed by the International Association for Medical Assistance to Travelers
001 ABL Acute Bilineage Leukemia OL
002 AISA Acquired Idiopath Siderblastic Anemia OND
003 ALD Adrenoleukodystrophy IMD
004 ALL Acute Lymphoblastic Leukemia ALL
005 AMCL Acute Mast Cell Leukemia OL
006 AMEGCT Amegakaryocy/Congenital Throm IPA IPA
007 AMF Acute Myelofibrosis OND
008 AML Acute Myelogeneous Leukemia AML
009 ATA Ataxia-Telangiectasia IIS
010 AUL Acute Undifferentiated Leukemia-MO OL
011 BGLUD Beta-glucuronidase Deficiency IMD
012 BL Burkitt Lymphoma NHL
013 BLSYND Bare Lymphocyte Syndrome IIS
014 BRCA Breast Cancer OM
015 BTHAL Beta Thalassemia Major IEA
016 C-HHYP Cartilage-Hair Hypoplasia IIS
017 CGD Chronic Granulomatous Disease OND
018 CHED Chediak-Higashi IIS
019 CID Combined Immunodeficiency IIS
020 CLL Chronic Lymphocytic Leukemia OL
021 CML Chronic Myelogeneous Leukemia CML
022 CMML Chronic Myelomonocytic Leukemia OL
023 CNSCA Central Nervous System Tumours OM
024 DIGRGE Di George Syndrome IIS
025 ETC Essential Thrombocythemia IPA
026 EWINGS Ewings Sarcoma OM
027 FA Fanconi Anemia IEA
028 FEL Familial Erythrophagocytic HIS
029 GAUCHR Gaucher's Disease IMD
030 GLTROM Glanzmanns Thrombastenia IPA
031 GLUCST Glucose Storage Disease IMD
032 HCL Hairy Cell Leukemia OL
033 HIS Histiocytic Disorders HIS
034 HISX Histiocytosis-X HIS
035 HIV HIV Infection OND
036 HL Hodgkin's Lymphoma HL
037 HLS Hurler Syndrome IMD
038 HPGC Hemophagocytosis HIS
039 HTS Hunter Syndrome IMD
040 HUNTER Hunter Syndrome (MPS-II) IMD
041 HURLER Hurler Syndrome (MPS-IH) IMD
042 I-CELL I-Cell Disease IMD
043 IEA Inherited Erythrocyte Abnormality IEA
044 IIS Inherited Immune System Disorder IIS
045 IMD Inherited Metabolic Disorder IMD
046 IMDNP Immune Deficiency + Neutropen IIS
047 IPA Inherited Platelet Abnormality IPA
048 JCML Juvenile Chronic Myelogeneous OL
049 KOSTMN Kostmann's Agranulocytosis IIS
050 KRABBE Krabbe Disease IMD
051 LAD eukocyte Adhesion Deficiency IIS
052 LN Lesch-Nyhan IMD
053 LYSOST Lysosomal Storage Disease IMD
054 MARLAM Maroteaux-Lamy (MPS-VI) IMD
055 MDS Myelodysplastic Syndromes MDS
056 MFMM Myelofibro Myeloid Metaplasia MDS
057 MLD etachromatic Leukodystrophy IMD
058 MM ultiple Myeloma PCD
059 MORQUO Morquio (MPS-IV) IMD
060 MPS ucopolysaccaridosis IMD
061 N-P Neiman-Pick Disease IMD
062 NB Neuroblastoma OM
063 NEUTAD Neutrophil Actin Deficiency OND
064 NEUTD Neutrophil Defect OND
065 NHL Non-Hodgkin's Lymphoma NHL
066 OAL Other Acute Leukemia OL
067 OCID Other Combined Immunodeficiency IIS
068 OHIS Other Histiocytic Disorders HIS
069 OIEA Other Erythrocyte Differentiation IEA
070 OIIS Other Immune System IIS
071 OIMD Other Inherited Disorders of Metabolism IMD
072 OIPA Other Abnormalities or Platelets OND
073 OL Other Leukemia OL
074 OM Other Malignancy OM
075 OMDS Other Myelodysplastic Disorder MDS
076 OMENN Omenn's Syndrome IIS
077 OMS Other Malignancy, Specify OM
078 OND Other Non-Malignant Disease OND
079 OOL Other Acute Leukemia OL
080 OPCD Other Plasma Cell Disorder PCD
081 OSCID Other SCID IIS
082 OST Osteopetrosis IMD
083 OTHER Other diseases OND
084 OTHHGP Other Hemoglobinopathy IEA
085 PCD Plasma Cell Disorder PCD
086 PCL Plasma Cell Leukemia PCD
087 PHILLY Prolymphocytic Leukemia OL
088 PNH Paroxysmal Nocturnal Hemoglobinurea MDS
089 PV Polycythemia Vera OND
090 RA Refractory Anemia OND
091 RAEB Refract Anemia-excess blasts MDS
092 RAEBIT Refract Anemia-excess blast tran MDS
093 RCA Pure Red Cell Aplasia IEA
094 RETDG Reticular Dysgenesis IEA
095 SAA Severe Aplastic Anaemia SAA
096 SANFIL Sanfillippo (MPS-III) IMD
097 SCA Sickle Cell Anemia IEA
098 SCHEIE Scheie Syndrome (MPS-IS) IMD
099 SCID Severe Combined Immunodeficiency IIS
100 SCLUNG Small Cell Lung Cancer OM
101 SF San Filippo IMD
102 TBSCID Absence of T&B Cells SCID IIS
103 THA Thalassemia IEA
104 TSCID Absence of T, Normal B SCID IIS
105 UIIS Unknown Immune System IIS
106 UIMD Unknown Disorders of Metabolism IMD
107 UIPA Unknown Abnormalities or Platelets OND
108 UMDS Unknown Myelodysplastic Disorder MDS
109 UNKN Unknown OND
110 UOL Unknown Acute Leukemia OL
111 UPCD Unknown Plasma Cell Disorder PCD
112 VARIMD Common Variable Immunodeficiency IIS
113 WAS Wiskott Aldrich Syndrome IIS
114 WMG Waldenstroms Macroglobulinemia PCD
115 WOLMAN Wolman Disease IMD
116 XLLPD X-linked Lymphoproliferative IIS
Leukemia
Types
Leukemia is classified by how quickly it progresses. Acute leukemia is fast-growing and can overrun the body within a few weeks or months. By contrast, chronic leukemia is slow-growing and progressively worsens over years.
Acute versus Chronic Leukemia The blood-forming (hematopoietic) cells of acute leukemia remain in an immature state, so they reproduce and accumulate very rapidly. Therefore, acute leukemia needs to be treated immediately, otherwise the disease may be fatal within a few months. Fortunately, some subtypes of acute leukemia respond very well to available therapies and they are curable. Children often develop acute forms of leukemia, which are managed differently from leukemia in adults.
In chronic leukemia, the blood-forming cells eventually mature, or differentiate, but they are not "normal." They remain in the bloodstream much longer than normal white blood cells, and they are unable to combat infection well.
Myelogenous versus Lymphocytic Leukemia Leukemia also is classified according to the type of white blood cell that is multiplying - that is, lymphocytes (immune system cells), granulocytes (bacteria-destroying cells), or monocytes (macrophage-forming cells). If the abnormal white blood cells are primarily granulocytes or monocytes, the leukemia is categorized as myelogenous, or myeloid, leukemia. On the other hand, if the abnormal blood cells arise from bone marrow lymphocytes, the cancer is called lymphocytic leukemia.
Other cancers, known as lymphomas, develop from lymphocytes within the lymph nodes, spleen, and other organs. Such cancers do not originate in the bone marrow and have a biological behavior that is different from lymphocytic leukemia.
There are over a dozen different types of leukemia, but four types occur most frequently. These classifications are based upon whether the leukemia is acute versus chronic and myelogenous versus lymphocytic, that is:
Acute Myelogenous (granulocytic) Leukemia (AML)
Chronic Myelogenous (granulocytic) Leukemia (CML)
Acute Lymphocytic (lymphoblastic) Leukemia (ALL)
Chronic Lymphocytic Leukemia (CLL)
Acute Myelogenous Leukemia (AML) Acute myelogenous leukemia (AML) - also known as acute nonlymphocytic leukemia (ANLL) - is the most common form of adult leukemia. Most patients are of retirement age (average age at diagnosis = 65 years), and more men are affected than women. Fortunately, because of recent advances in treatment, AML can be kept in remission (lessening of the disease) in approximately 60% to 70% of adults who undergo appropriate therapy. Initial response rates are approximately 65-75% but the overall cure rates are more on the order of 40-50%.
AML begins with abnormalities in the bone marrow blast cells that develop to form granulocytes, the white blood cells that contain small particles, or granules. The AML blasts do not mature, and they become too numerous in the blood and bone marrow. As the cells build up, they hamper the body's ability to fight infection and prevent bleeding. Therefore, it is necessary to treat this disease within a short time after making a diagnosis. AML, particularly in the monocytic M5 form, may spread to the gums and cause them to swell, bleed, and become painful. AML also may metastasize (spread) to the skin, causing small colored spots that mimic a rash.
Acute leukemia, such as AML, is categorized according to a system known as French-American-British (FAB) classification. FAB divides AML into eight subtypes:
undifferentiated AML (M0) - In this form of leukemia, the bone marrow cells show no significant signs of differentiation (maturation to obtain distinguishing cell characteristics).
myeloblastic leukemia (M1; with/without minimal cell maturation) - The bone marrow cells show some signs of granulocytic differentiation.
myeloblastic leukemia (M2; with cell maturation) - The maturation of bone marrow cells is at or beyond the promyelocyte (early granulocyte) stage; varying amounts of maturing granulocytes may be seen. This subtype often is associated with a specific genetic change involving translocation of chromosomes 8 and 21.
promyelocytic leukemia (M3 or M3 variant [M3V]) - Most cells are abnormal early granulocytes that are between myeloblasts and myelocytes in their stage of development and contain many small particles. The cell nucleus may vary in size and shape. Bleeding and blood clotting problems, such as disseminated intravascular coagulation (DIC), are commonly seen with this form of leukemia. Good responses are observed after treatment with retinoids, which are drugs chemically related to vitamin A.
myelomonocytic leukemia (M4 or M4 variant with eosinophilia [M4E]) - The bone marrow and circulating blood have variable amounts of differentiated granulocytes and monocytes. The proportion of monocytes and promonocytes (early monocyte form) in the bone marrow is greater than 20% of all nucleated (nucleus-containing) cells. The M4E variant also contains a number of abnormal eosinophils (granular leukocyte with a two-lobed nucleus) in the bone marrow.
monocytic leukemia (M5) - There are two forms of this subtype. The first form is characterized by poorly differentiated monoblasts (immature monocytes) with lacy-appearing genetic material. The second, differentiated form is characterized by a large population of monoblasts, promonocytes, and monocytes. The proportion of monocytes in the bloodstream may be higher than that in the bone marrow. M5 leukemia may infiltrate the skin and gums, and it has a worse prognosis than other subtypes.
erythroleukemia (M6) - This form of leukemia is characterized by abnormal red blood cell-forming cells, which make up over half of the nucleated cells in the bone marrow.
megakaryoblastic leukemia (M7) - The blast cells in this form of leukemia look like immature megakaryocytes (giant cells of the bone marrow) or lymphoblasts (lymphocyte-forming cells). M7 leukemia may be distinguished by extensive fibrous tissue deposits (fibrosis) in the bone marrow.
In addition, patients sometimes develop isolated tumors of the myeloblasts (early granulocytes). An example of this is isolated granulocytic sarcoma, or chloroma - a malignant tumor of the connective tissue. Individuals with chloroma frequently develop AML, so they usually are treated with an aggressive, AML-specific chemotherapy program.
Chronic Myelogenous Leukemia (CML) Chronic myelogenous leukemia (CML) is known as a myeloproliferative disorder - that is, it is a disease in which bone marrow cells proliferate (multiply) outside of the bone marrow tissue.
CML is easy to diagnose, since it has a genetic peculiarity, or marker, that is readily identifiable under a microscope. About 95% of CML patients have a genetic translocation between chromosomes 9 and 22 in their leukemic cells. This abnormality, which is known as the Philadelphia chromosome (Ph1), is named after the city in which it was discovered. The Philadelphia chromosome causes uncontrolled reproduction and proliferation of all types of white blood cells and platelets (blood clotting factors). Sadly, CML is not yet curable by standard methods of chemotherapy or immunotherapy.
CML tends to occur in middle- and retirement-aged people (the median age is 67 years). It occasionally affects people in their 20s, but it is rare in the very young; only 2% to 3% of childhood leukemias are CML. Early disease often is without symptoms (asymptomatic) and is discovered accidentally. Individuals with more advanced cases of CML may appear sickly and experience fevers, easy bruising, and bone pain. Laboratory and physical findings include enlarged spleen (splenomegaly), a high white blood cell count, and absent or low amounts of the white blood cell enzyme alkaline phosphatase.
Like other forms of leukemia, CML is not "staged". Rather, this unstable disease is categorized according to the three phases of its development: chronic, accelerated, and blast.
Chronic phase - Patients in this initial phase have fewer than 5% blast cells and promyelocytes (immature granulocytes) in their blood and bone marrow. This phase is marked by increasing overproduction of granulocytes. Individuals generally experience only mild symptoms, and they respond well to conventional treatment.
Accelerated phase - Patients in this progressive phase have more than 5%, but fewer than 30% blast cells. Their leukemic cells exhibit more chromosomal abnormalities besides the Philadelphia chromosome, and so more abnormal cells are produced. Noticeable symptoms such as fever, poor appetite, weight loss occur, and patients may not respond as well to therapy.
Blast phase (acute phase, blast crisis) - Patients in this final phase have more than 30% blast cells in their blood and bone marrow samples. The blast cells frequently invade other tissues and organs outside of the bone marrow. During this phase, the disease transforms into an aggressive, acute leukemia (70% acute myelogenous leukemia, 30% acute lymphocytic leukemia). If untreated, CML is fatal in roughly 20% of all patients each year.
Acute Lymphocytic Leukemia (ALL) Acute lymphocytic leukemia (ALL) - also known as acute lymphoblastic leukemia - is a malignant disease caused by the abnormal growth and development of early nongranular white blood cells, or lymphocytes. The leukemia originates in the blast cells of the bone marrow (B-cells), thymus (T-cells), and lymph nodes. ALL occurs predominantly in children, peaking at 4 years of age. ALL is seen more frequently in industrialized nations, and it is slightly more common among white children and boys.
ALL often is diagnosed after a patient experiences a 4- to 6-week period of illness. Initial symptoms may include a nonspecific infection (e.g., respiratory infection) that persists or recurs despite antibiotic therapy. During this period, the person may start to experience aching bone pain in the back, limbs, and/or joints. Walking difficulties may be seen in some children who have extreme swelling of the large joints. But the symptoms that most often suggest referral for a blood count (measure of the number of blood cells within the blood) are a purplish-brown rash or the onset of excessive bruising.
If ALL is T-cell in type, the thymus is involved. Leukemia-related enlargement of the thymus may lead to coughing, shortness of breath, or compression of the superior vena cava (SVC), the large vein that carries blood from the head and arms back to the heart). Such venous blockage may induce head and arm swelling and may cause a life-threatening condition known as SVC syndrome.
Both children and adults with ALL are at risk of developing complications due to central nervous system (CNS) involvement. CNS invasion is especially likely among patients with the L3 subtype of ALL. When leukemic cells infiltrate the CNS, they can cause increased pressure within the skull and paralysis of cranial nerves that connect the brain with other organs, muscles, etc.
Age is an important prognostic factor in ALL. Studies have suggested that patients who are younger than 35 years of age fare better than older patients; however, this observation may be related to the higher incidence of the Philadelphia chromosome (Ph1) among older ALL patients - a subgroup that has a poorer chance of survival. Fortunately, though, 60% to 80% of children and adults with ALL will achieve complete remission of the disease after completing appropriate therapy.
There is no standard staging system for ALL. Rather, ALL is categorized according to a system known as the French-American-British (FAB) Morphological Classification Scheme for ALL:
L1 - Mature-appearing lymphoblasts (T-cells or pre-B-cells). Cells are small with uniform genetic material, regular nuclear shape, nonvisible nucleoli (round bodies within the nucleus, the site of RNA synthesis), and little cytoplasm (substance of a cell, excluding the nucleus).
L2 - Immature and pleomorphic (variously shaped) lymphoblasts (T-cells or pre-B-cells). Cells are large and variable in size, with variable genetic material, irregular nuclear shape, one or more large nucleoli, and variable cytoplasm.
L3 - Lymphoblasts (B-cells; Burkitt's cells) are large and uniform; genetic material is finely stippled and uniform; nuclear shape is regular (oval to round); there are one or more prominent nucleoli; and cytoplasm is moderately abundant.
Chronic Lymphocytic Leukemia (CLL) Chronic lymphocytic leukemia is the most common leukemia in North America and in Europe. It is a disease of older adults and is very rare among people who are younger than 50 years of age. Men with CLL outnumber women by a 2-to-1 average. The disease usually is detected accidentally during a doctor's examination for an unrelated complaint.
CLL is thought to result from the gradual accumulation of mature, long-lived lymphocytes. Therefore, this cancer is caused not so much by overgrowth as it is by the extreme longevity and build-up of malignant cells. Although the rate of accumulation varies among individuals, the extensive tumor burden eventually causes complications in all CLL patients.
CLL is classified by one of two staging systems, although these systems are based on cytology (the study of cell characteristics) and are different from the staging used to evaluate other, nonleukemic cancers. The first system - known as Rai Classification - is used more often in the United States; the other system - known as Binet Staging - is more popular in Europe. Both methods are correlated with prognosis.
The process of becoming a Bone Marrow Donor is very simple through the Anthony Nolan Trust
1) You need to make sure you meet the following General Criteria:
- age 18 - 40 : health and circumstances permitting, you remain on the Register until your 60th birthday, so don't be put off joining if you are close to 40
- in general good health
- weight over 51kg / 8 stone
- women must not be pregnant
- women with children under the age of 12 months cannot join the register or donate marrow during that time
In addition you will need to check that you not excluded through the Medical Criteria (set out below)
Although it costs up to £70 to recruit each donor, tissue-type each sample, and maintain the donor's records there is no cost or charge for you and you can ‘sign up’ from anywhere in United Kingdom, so long as you are a UK resident.
2) Completing the Paperwork
You can obtain an information pack and medical questionnaire in four ways:
- calling Anthony Nolan hotline number - 0901 88 22 234 : calls are charged at 25p per minute and a proportion of the call charge comes to the Trust
- writing to: The Anthony Nolan Trust, Units 2 - 3, Heathgate Place, 75 - 87 Agincourt Road, London, NW3 2NU
- attending a donor recruitment clinic near you
- email: newdonor@anthonynolan.org.uk
3) Giving a Blood Sample
A blood testing kit can be sent to you by the Anthony Nolan Trust, so that you can take this to your local GP or health clinic, where a small sample of your blood will be taken - about 4ml. Needless to say, on no account should you attempt to take the sample yourself.
The sample will be forwarded to the Anthony Nolan laboratories, where it is tested to establish your tissue type. The results are stored on their database, which is, of course, confidential.
As a new member of the Register you will receive confirmation of your status as a potential life saver.
You may be found to be a match for a patient almost immediately; other people wait many years before being asked to donate - and some never are.
Every single person on our Register is still making a wonderful commitment – to make themselves available to support another person in need of a life saving transplant.
Find out more at www.anthonynolan.com
Medical Criteria
For a variety of reasons which can relate to the outcome of a transplant, or to your own welfare as a donor, certain conditions prevent you from joining the Register.
The application you complete before giving a blood sample covers all of the following medical conditions.
ALCOHOL
Sometimes Acceptable
No - if you are alcoholic or regularly consumer more than 40 units a week as a man or 30 units a week as a woman; or if you are on antabuse.
Exception - if you have recovered from alcoholism, and it has been two years or longer since recovery
ALLERGY : drug-induced
Generally Not Acceptable
Exception - if environmentally produced
ANAEMIA
Generally Acceptable
No - especially if there is a history of persistent anaemia or if someone is regularly anaemic i.e. more than three times a year.
Exception - you are only temporarily or rarely anaemic, you are not taking medication and you haemoglobin count is within the accepted range
ANKYLOSING SPONDYLITIS
Definitely not acceptable
ANGINA
Definitely not acceptable
ASTHMA
Generally not acceptable
No - if you are on continuous high dose medication (more than 8 inhalations /puffs per day) as people suffering from asthma are more likely to have post-operative complications.
Exception - if medication is taken rarely, less than 8 inhalations/puffs per day or if the asthma is brought on by an allergy
BACK PROBLEMS
Generally Not Acceptable
No - if upper back / neck problems, chronic lumbar vertebrae problems, such as slipped discs or sciatica, chronic lower back problems, or any injury which means that you are have to avoid certain sports, heavy lifting, prolonged seating
Exception - minor muscular problems or minor problems in the cervical or thoracic areas of the spine
BRAIN SURGERY
Generally Not Acceptable
Exception - if the brain surgery was performed after September 1992
CANCER
Definitely Not Acceptable
No - if any form of cancer including skin cancer even if you have been given the all clear by your treating physician.
CHRISTMAS DISEASE
Definitely Not Acceptable
CIRRHOSIS
Definitely Not Acceptable
No - there is a risk with general anaesthetic and bleeding.
COAGULATION DISORDERS
Definitely Not Acceptable
No - if you have a history of DVT, PE, Leiden factor, Von Willebrands.
CELIAC DISEASE
Definitely Not Acceptable
No - intolerance to gluten may relate to the auto-immune system
CROHN'S DISEASE
Definitely Not Acceptable
No - auto-immune
CYSTIC FIBROSIS
Definitely Not Acceptable
No - medical risks associated with general anaesthetic
DEPRESSION
Generally Acceptable
No - if endogenous, manic or severe.
Acceptance criteria - mild, short-term reactive depression with short term medication
N.B. individual assessment is obligatory
DIABETES
Generally Not Acceptable
No - insulin or tablet dependent diabetes can be associated with auto-immune disease which could be passed on to the patient.
Exception - non insulin-dependent diabetes, well controlled by diet
DERMATITIS HERPETIFORMIS
Definitely Not Acceptable
No - as often associated with coeliac disease.
DOWNS SYNDROME
Definitely Not Acceptable
ECZEMA
Generally Acceptable
No - if atopic (since childhood and severe) or if auto-immune related
Exception - mild and irregular
ENDOMETRIOSIS
Generally Acceptable
Acceptance Criteria - if well controlled.
EPILEPSY
Generally Not Acceptable
No - if on medication, due to risks associated with general anaesthetic
Exception - if you have not had a seizure for a minimum of 3 years and have not taken medication during that time. Donors must be aware that there is a small risk of fitting when awakening from the anaesthetic but this can be well-controlled
GALL STONES
Generally Not Acceptable
Exception - If you have undergone surgery and have no current problems.
GASTRO-INTESTINAL SYMPTOMS such as ulcers, general gastritis
Generally Acceptable
Exception - If the condition is well-controlled, with no internal bleeding or other complications.
GLANDULAR FEVER
Generally Acceptable
No - if a current condition or if you are still suffering from after effects
Acceptance criteria - if in the past and mild.
GRAVES DISEASE
Definitely Not Acceptable
No - auto-immune
HEART CONDITIONS
Generally Not Acceptable
No - angina, cardiomegeneral anaestheticity, cardiomyopathy, past history of heart attacks, heart arrhythmia, pacemaker, or a congenital condition such as thickened valve, hole in heart, heart murmur or any other condition which requires individuals to take antibiotic cover at any time, as all are considered a risk under general anaesthesia
Exception - if you have a congenital heart condition but do not have to take any prophylactic antibiotic cover at any time and are in good health
HEPATITIS A
Generally Acceptable
Acceptance criteria - if you have been clear for longer than 12 months and you are currently asymptomatic.
HEPATITIS B OR C
Definitely Not Acceptable
No - these viruses are found in body fluids, including blood and bone marrow.
HIGH BLOOD PRESSURE
Generally Not Acceptable
Exception - if the condition is well-controlled by medication and the most recent blood pressure reading is below: 150/95
HIP REPLACEMENT
Generally Acceptable
Acceptance Criteria - as long as no current problems at all but you must be aware that some short-term back pain could arise if you donate
HIV EXPOSURE
Definitely Not Acceptable
No - if you have been knowingly exposed to HIV you are considered a high risk category
HUMAN PITUITARY EXTRACT TREATMENT
Generally Not Acceptable
No - if treated with growth hormones or gonadotrophins
Exception - if treatment started after 1987
HYPOTHYROIDISM (MYXOEDEMA)
Generally Not Acceptable
Exception - if the cause is not auto-immune related
HYSTERECTOMY
Generally Acceptable
Acceptance Criteria - if the operation was over 6 months ago and there were no malignancies
ITP
Definately Not Acceptable
No - auto-immune
JAUNDICE
Generally Not Acceptable
Exception - the jaundiced was experienced as a baby or at a young age.
LUPUS (SLE)
Definitely Not Acceptable
No - auto-immune
MAJOR SURGERY OR OPERATION
Generally Not Acceptable
Exception - if at least six months must have elapsed since the operation and you are clear of all post operative symptoms.
MALARIA
Generally Not Acceptable
No - especially if within the last 12 months
Exception - if you have taken a full course of anti-malarial tablets, the timespan was longer than 12 months ago, and no symptoms are being experienced
ME / POST VIRAL SYNDROME
Generally Acceptable
Acceptance Criteria - only one episode over 12 months ago and no relapses have occurred
MIGRAINE
Generally Acceptable
Acceptance Criteria - if mild or infrequent (less than every 2 months
MS
Definitely Not Acceptable
No - auto-immune
MYASTHENIA GRAVIS
Not Acceptable
No - Autoimmune
OCD (OBSESSIVE COMPULSIVE DISORDER)
Generally Not Acceptable
No - especially if severe
Exception - depends on severity/ treatment - individual assessment necessary
OSTEO-ARTHRITIS
Generally Acceptable
No - especially if severe
Acceptance Criteria - if the treatment is mild, you are not on steroids and the hip and spine areas are unaffected
OSTEOGENESIS IMPERFECTA
Definitely Not Acceptable
No - there is a risk of fracture
OSTEOPOROSIS
Definitely Not Acceptable
No - a risk of bone damage
PANCREATITIS
Generally Not Acceptable
No - especially if chronic and with diarrhoea.
Exception - if there has been just one episode and you are fully recovered
PARAPLEGIC / TETRAPLEGIC
Not Acceptable
PERICARDITIS
Generally Not Acceptable
Exception - if fully recovered and no antibiotic cover needed
PERNICIOUS ANAEMIA
Definitely Not Acceptable
No - auto-immune
PITUITARY TUMOUR
Generally Not Acceptable
Exception - if benign, with no post-operative complications
PLEURISY
Generally Not Acceptable
Exception - if just a single incident with no recurrence
POLIO
Generally Not Acceptable
No - with serious physical disabilities which could be aggravated by donating marrow
Exception - if mild and no serious physical disabilities
POLYCYSTIC KIDNEY
Not Acceptable
PRE-CANCEROUS CELLS
Generally Not Acceptable
Exception - if your last two smear tests have been clear
PREGNANCY
Generally Not Acceptable
Exception - you can only join after your baby is 12 months old
PROCTITIS
Generally Not Acceptable
No - If autoimmune
PSORIASIS
Generally Not Acceptable
Exception - if very mild
PSORIATIC ARTHRITIS
Definitely Not Acceptable
No - auto-immune
PULMONARY EMBOLISM
Definitely Not Acceptable
PUPURA
Definitely Not Acceptable
No - could be auto-immune
RAYNAUD'S SYNDROME
Generally Not Acceptable
No - if associated with SLE
ROSACEA
Generally Not Acceptable
Exception - if localised, e.g. on the face.
RHEUMATIC FEVER
Definitely Not Acceptable
RHEUMATOID ARTHRITIS
Definitely Not Acceptable
No - auto-immune disease
SARCOIDOSIS
Definitely Not Acceptable
No - the cause is unknown, can affect any system, heart, lungs bone
SCIATICA
Generally Not Acceptable
Exception - the condition is mild and does not interfere with your normal lifestyle
SCHEUERMANNS DISEASE
Generally Not Acceptable
Exception - no current problems
SCHIZOPHRENIA
Definitely Not Acceptable
No - medication given is usually a contra-indication to general anaesthetic
SCOLIOSIS
Generally Not Acceptable
Exception - if you have no current back problems or rods inserted
SICKLE CELL DISEASE
Generally Not Acceptable
Exception - as trait only
SLEEP APNOEA
Definitely Not Acceptable
No - there is a risk with airway post general anaesthetic
SLE
See Lupus
SPINA BIFIDA OCCULTA
Generally Not Acceptable
Exception - if you are free from problems or mobility problems
TACHYCARDIA
Generally Not Acceptable
Exception - if mild and you are not on medication
TATTOOS / BODY PIERCING (Recent)
Generally Not Acceptable
Exception - if 12 months since tattoo or piercing
TAY SACHS DISEASE
Generally Not Acceptable
Exception - as trait only
TESTOSTERONE INJECTIONS
Generally Not Acceptable
Exception - synthetic injection
THALASSAEMIA
Generally Not Acceptable
Exception - as trait only
THROMBOCYTOPENIA
Definitely Not Acceptable
THYROID
Generally Acceptable
No - if you have Graves disease or Hashimoto's disease, due to auto-immune association
Acceptance Criteria - if you are on thyroxine. If you are on other drugs individual assessment is necessary
TB
Generally Not Acceptable
Exception - if you have been clear for more than 24 months and are not on medication
TURNERS SYNDROME
Generally Not Acceptable
Exception - if the donor has no heart problems
ULCERATIVE COLITIS
Definitely Not Acceptable
No - auto-immune.
URTICARIA
Generally Not Acceptable
Exception - if the condition is mild. Individual assessment is necessary
VITILIGO
Definitely Not Acceptable
No - can be associated with auto-immune disorders.
The following medical conditions do not preclude donating, but should be noted on your registration to join our bone marrow register :
- Gilberts' syndrome
- Hormone replacement therapy
- Irritable bowel syndrome
- PID (Pelvic Inflammatory Disease)
- peritonitis
- polycystic ovary
- renal colic (kidney stone)
- shingles
- termination of pregnancy
- Toxoplasmosis
Indications - Allogeneic
I.
Histocompatibility - All potential allogeneic transplant recipients must have a five of six HLA antigen matched related or unrelated donor. The recipient and donor must be HLA-DR identical. Also, all unrelated donor/recipients must be HLA-DR Beta 1 identical by DNA analysis.
II. Type of Transplant by Disease Category
A. Related, Matched (myeloablative and non-myeloablative)
1. Acute Non-lymphocytic Leukemia in 1st, 2nd or greater remission
2. Refractory Acute Non-lymphocytic Leukemia (Considered on a case by case basis)
3. Acute Non-lymphocytic Leukemia in early relapse (<30 blasts)
4. Acute Lymphocytic Leukemia in 1st, 2nd or greater remission (adult) and Acute Lymphocytic Leukemia in 2nd or greater remission (children)
5. Chronic Myelogenous Leukemia in chronic or accelerated phase
6. Chronic Myelogenous Leukemia in 2nd chronic phase (after blast crisis)
7. Chronic Lymphocytic Leukemia - symptomatic advanced Stage III and IV
8. Juvenile Chronic Myelogenous Leukemia
9. Myelodysplasia
10. Hodgkin's Disease or Non-Hodgkin's Lymphoma
11. Aplastic Anemia
12. Multiple Myeloma
B. Unrelated, Matched
1. Acute Non-lymphocytic Leukemia in 1st, 2nd or greater remission (adult) and Acute Non-lymphocytic Leukemia in 2nd or greater remission (children)
2. Acute Non-lymphocytic Leukemia in early relapse (<30% blasts)
3. Acute Lymphocytic Leukemia in 2nd or greater remission
4. Chronic Myelogenous Leukemia in chronic or accelerated phase
5. Myelodysplasia (Refractory Anemia with Excess Blasts)
III. Source of progenitor cells
A. Bone Marrow
B. Peripheral blood stem cells
C. Cord blood
Indications - Autologous
I.
Disease Status - All patients must demonstrate chemotherapy responsive disease prior to autologous transplantation and evidence of normal cellular, malignancy - free bone marrow on marrow aspirate and biopsy. Peripheral blood stem cells will be the primary source of progenitor cells for autologous transplantation.
II. Diagnoses (Adults and Children)
A. Advanced Stage, Recurrent Hodgkin's Disease; non-purged
B. Advanced Stage, Recurrent Intermediate and/or High Grade, Non-Hodgkin's Lymphoma; non-purged
C. Low Grade Non-Hodgkin's Lymphoma, non-purged (adult)
D. Acute Nonlymphocytic Leukemia in 1st, 2nd or greater remission; non-purged (adults only)
E. Acute Nonlymphocytic Leukemia in 1st remission; purged (children only)
F. Neuroblastoma; purged and non-purged (adult and children)
G. Multiple Myeloma
H. Selected Solid Tumors
III. Source of Progenitor cells
A. Peripheral blood stem cells
B. Bone marrow with or without peripheral blood stem cells
Contraindications
I. Relative
A. Age
B. Cardiopulmonary Disease
C. Renal Disease
D. Liver Disease
E. Active Infection
F. Psychosocial dysfunction
II. Absolute
A. HIV seropositivity
B. Chronic Active Hepatitis
C. Any active soft tissue infection
D. Inability to give informed consent
Allogeneic and Autologous Eligibility Criteria
Adults and Adolescents (Allogeneic)
Patients must be >12 years but < 60 years of age for related donor transplants, and >12 years but < 60 years of age for non-related donor transplants, and < 75 years for non myeloablative, related donor transplant.
Patients must have an ECOG Performance Status of 2 or less.
Patients must have no evidence of active infection at the time of transplantation.
Patients must be HIV nonreactive.
Patients must have a multi-organ assessment prior to transplantation with the following outcome:
A resting ejection fraction of 50% or greater which increases with exercise as demonstrated by Resting/Exercise MUGA
A diffusion capacity of 50% or greater of predicted, a FEV1 of 60% or greater, and PO2 of 80 mm Hg as demonstrated on pulmonary functions testing
A serum creatinine of less than or equal to 2.0 mg/dL and a creatinine clearance of 50 ml/min or greater on 24 hr. urine collection
A total bilirubin of <2.5 mg/dL and/or a AST <2 times the upper limit of normal.
Patients who have had any complication that makes the risk of death during transplantation from non-malignant causes greater than the risk of relapse are ineligible.
Patients who have any active infection such as a soft tissue infection, sinus infection, dental infections, fungal infections or hepatitis including chronic active hepatitis are ineligible; if the infection is successfully treated, the patient may be reconsidered for transplantation at a later date.
Patients who have diabetes mellitus will be considered on a case-by-case basis. However, patients with diabetes who are not controlled by medical management will be ineligible.
Psychosocial assessment by the bone marrow transplant team may identify individuals for whom this form of therapy may be contraindicated. These decisions will be based upon estimated adequacy of patient support systems and prediction of patient's compliance with medications, required diagnostic procedures and/or follow-up care.
Patients or their guardians must give written informed consent in accordance with institutional and federal regulations.
Patients or their guardians must demonstrate proof-of-payment.
Small Children and Infants (Allogeneic)
Patients must be <12 years of age for related donor transplants, and >1year but <12 years of age for non-related donor transplants.
Patients must have an ECOG Performance Status of 2 or less, or appropriate for age.
Patients must have no evidence of active infection at the time of transplantation.
Patients must be HIV non-reactive.
Patients must have a multi-organ assessment before transplantation with the following outcome:
A normal echocardiogram
O2 saturation >95% by pulse oximetry
A serum creatinine within normal limits for age
Patients who have had any complication that makes the risk of death during transplantation from non-malignant causes greater than the risk of relapse are ineligible.
Patients who have any active infection such as a soft tissue infection, sinus infection, dental infections, fungal infections or hepatitis including chronic active hepatitis are ineligible; if the infection is successfully treated, the patient may be reconsidered for transplantation at a later date.
Patients who have diabetes mellitus will be considered on a case-by-case basis. However, patients with diabetes who are not controlled by medical management will be ineligible.
Guardian must give written informed consent in accordance with institutional and federal regulations.
Guardian must demonstrate proof-of-payment.
Adults and Adolescents (Autologous)
Patients must be ≥12 years but ≤ 70 years of age.
Patients must have a life expectancy without therapy of >4 weeks.
Patients must have an ECOG Performance Status of 2 or less, or appropriate for age.
Patients must have no evidence of active infection at the time of transplantation.
Patients must be HIV non-reactive.
Patients who have had any complication that makes the risk of death during transplantation from non-malignant causes greater that the risk of relapse are ineligible.
Patients must have a multi-organ assessment before to transplantation with the following outcome:
A resting ejection fraction of 45% or greater which increases with exercise as demonstrated by Resting/Exercise MUGA with no documented coronary artery disease, history of congestive heart failure, or uncontrolled cardiac dysrhythmia.
A PO2 greater than 60% on room air, O2 saturation of greater than 90% on room air, a FEV1 of greater than 50% and diffusion capacity greater than 40% of predicted.
A serum creatinine of less than 2.0 mg/dL and a creatinine clearance of 50 ml/min or greater on 24 hr. urine collection.
A total bilirubin <2.5 mg/dL, AST<3 times normal.
Patients who have diabetes mellitus will be considered on a case-by-case basis. However, patients with diabetes who are not controlled by medical management will be ineligible.
Psychosocial assessment by the bone marrow transplant team may identify individuals for whom this form of therapy may be contraindicated. These decisions will be based upon estimated adequacy of patient support systems and prediction of patient's compliance with medications, required diagnostic procedures and/or follow-up care.
Patients or their guardians must give written informed consent in accordance with institutional and federal regulations.
Patients or their guardians must demonstrate proof-of-payment.
Small Children and Infants (Autologous)
Patients must be > 3 months but < 18 years of age at time of diagnosis.
Patients must have no evidence of active infection at the time of transplantation.
Patients must be HIV non-reactive.
Patients must have a multi-organ assessment before transplantation with the following outcome:
A normal echocardiogram
O2 saturation >95% by pulse oximetry
A serum creatinine within normal limits for age
Patients who have had any complication that makes the risk of death during transplantation from non-malignant causes greater than the risk of relapse are ineligible.
Patients who have any active infection such as a soft tissue infection, dental infections, fungal infections or hepatitis including chronic active hepatitis are ineligible; if the infection is successfully treated, the patient may be reconsidered for transplantation at a later date.
Patients who have diabetes mellitus will be considered on a case-by-case basis. However, patients with diabetes who are not controlled by medical management will be ineligible.
Guardian must give written informed consent in accordance with institutional and federal regulations.
Guardian must demonstrate proof-of-payment.
Indications and Contraindications for Retransplantation
Allogeneic
Related Donor - Patients who have a related donor are eligible for a second allogeneic bone marrow transplant if they do not demonstrate evidence of engraftment or if graft failure occurs due to rejection or graft versus host disease. Patients must not have evidence of overwhelming infection or end-stage organ dysfunction.
Unrelated Donor - Patients who have an unrelated donor transplant are eligible for a second unrelated donor transplant if they do not demonstrate evidence of engraftment or if graft failure occurs. However, our Bone Marrow Transplant Program must abide by the retransplantation policy and procedures of the National Marrow Donor Program. Those patients who experience a recurrence of their disease following allogeneic bone marrow transplantation will be eligible for a second transplant if they are greater than six months from the first transplant, (first transplant which included ablative chemotherapy alone or in combination with radiotherapy), and if the status of their disease (malignancy only) is minimal or a remission has been achieved with salvage therapy. In addition, patients must have no evidence of major organ dysfunction and/or evidence of active infection. Each patient will be considered by the transplant team on a case-by-case basis.
Autologous
It is not our policy to perform second autologous transplants for patients with recurrent disease.
At this time, we do not perform tandem transplants, however, we do participate in an ECOG study that investigates the efficacy of tandem transplants for patients with testicular cancer.
Evaluation
Patient
A transplant physician in the outpatient clinic evaluates all patients considered for bone marrow transplantation. This evaluation consists of a review of medical records, pathology slides and x-ray films to determine medical history, diagnosis, stage (if applicable) and current disease status. Prior to transplantation, a multi-organ assessment is performed to demonstrate there are no contraindications to transplantation. Appropriate consultations are obtained when indicated. In addition, all transplant candidates and family members are interviewed by the transplant coordinator, primary outpatient nurse and social worker.
Donor
Potential donors are HLA-A, B and DR typed in addition to ABO typing. State- of-the-art, molecular, DR typing is also performed. All HLA testing must be performed at UWHC. Prior to transplantation, the potential donor must have an evaluation which includes: 1) complete history and physical; 2) CBC with differential and platelet count; 3) prothrombin time (PT) and partial thromboplastin time (PTT); 4) chemistry panel that includes glucose, electrolytes, and liver and renal function tests; 5) antibody screen for Hepatitis B and C; 6) antibody screen for toxoplasmosis, Epstein-Barr virus, RPR, HTLV I/II, cytomegalovirus and HIV antigen and antibody; 7) urinalysis. Other tests may be ordered when necessary, such as chest x-ray, electrocardiogram, and pregnancy test.
Also, adult donors may donate one unit of blood for autologous transfusion if it is planned that more than 500 ml of bone marrow will be collected. Donors who are minors will be evaluated on an individual basis regarding the necessity of transfusion following the marrow collection and/or the feasibility of their donating blood for autologous transfusion.
A detailed list of tests performed during the evaluation follows the Summary Statistics.
Acceptance
Patients are accepted/rejected as transplant candidates after the transplant team, which is comprised of transplant physicians, nurses, coordinators and social worker, reviews the initial evaluation. Patients who are accepted for transplantation must satisfy UWHC's financial requirements through written confirmation of insurance coverage or alternative financial arrangements.
When patients are not accepted for transplantation, either because they do not meet the indications or have a condition, which is contraindicated, the transplant physician informs them. When necessary, support from a social worker is available. In addition, we communicate information regarding non-acceptance to the appropriate referring or primary physician to whom the patient is referred back for continuing medical care.