Haematopoietic stem cell transplantation for amegakaryocytic thrombocytopenia

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...

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Bibliographic Details
Published in:British journal of haematology 2000-06, Vol.109 (4), p.773-775
Main Authors: Lackner, A., Basu, O., Bierings, M., Lassay, L., Schaefer, U. W., Révész, T., Havers, W., Kremens, B.
Format: Article
Language:English
Subjects:
allogeneic bone marrow transplantation
Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy
Biological and medical sciences
Bone Marrow Cells
Bone marrow, stem cells transplantation. Graft versus host reaction
Child
Child, Preschool
congenital amegakaryocytic thrombocytopenia
Female
Fetal Blood
Follow-Up Studies
Hematology
Hematopoietic Stem Cell Transplantation - methods
Humans
Infant
Male
Medical sciences
Megakaryocytes
Survival Rate
Thrombocytopenia - genetics
Thrombocytopenia - mortality
Thrombocytopenia - surgery
Transfusions. Complications. Transfusion reactions. Cell and gene therapy
Transplantation, Homologous
Treatment Outcome
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Summary: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.
ISSN:0007-1048
1365-2141
DOI:10.1046/j.1365-2141.2000.02099.x