Haematopoietic stem cell transplantation for amegakaryocytic thrombocytopenia.
Br J Haematol
; 109(4): 773-5, 2000 Jun.
Article
in En
| MEDLINE
| ID: mdl-10929028
ABSTRACT
Congenital amegakaryocytic thrombocytopenia (CAT), a rare syndrome with failure of megakaryopoiesis, cannot be cured by immunoglobulins, steroids or cyclosporin, but only by allogeneic bone marrow transplantation (BMT). We report on eight patients with CAT, all of whom were dependent at the time of BMT on platelet transfusion. Sources of haematopoietic progenitor cells were bone marrow (n = 5), peripheral stem cells (n = 2) and cord blood (n = 1). Seven patients engrafted. Both patients with matched unrelated donor transplants died, six patients are well with stable platelet counts 3-27 months after transplantation. BMT represents a curative option for CAT. The benefit of using alternative marrow donors should be carefully evaluated.
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Collection:
01-internacional
Database:
MEDLINE
Main subject:
Thrombocytopenia
/
Megakaryocytes
/
Hematopoietic Stem Cell Transplantation
Type of study:
Observational_studies
/
Prognostic_studies
Limits:
Child
/
Child, preschool
/
Female
/
Humans
/
Infant
/
Male
Language:
En
Journal:
Br J Haematol
Year:
2000
Document type:
Article
Affiliation country:
Alemania