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Amegakaryocytic Thrombocytopenia

Allogeneic stem cell transplantation for patients with congenital amegakaryocytic thrombocytopenia (CAT)

Bone Marrow Transplantation volume 33, pages 829–831 (2004)Cite this article

Summary:

Five patients with confirmed congenital amegakaryocytic thrombocytopenia (CAT) underwent stem cell transplantation (SCT) from HLA-matched related donors at King Faisal Specialist Hospital and Research Center (KFSHRC). The median age at SCT was 3.2 years (range, 0.4–5 years). Conditioning regimen consisted of busulfan (BU) 4 mg/kg p.o. for 4 days (total dose of 16 mg/kg), and cyclophosphamide (CY) 50 mg/kg once daily i.v. for 4 days (total dose of 200 mg/kg). Antithymocyte globulin (ATG) was given i.v. at a dose of 30 mg/kg for 4 days pre-SCT (total of 120 mg/kg); graft-versus-host disease (GVHD) prophylaxis was with cyclosporine and methotrexate. Four patients engrafted and are alive and transfusion independent with a median follow up time of 30 months (range, 16–45 months). One patient failed to engraft and underwent a second SCT 4 months later but died of respiratory failure. We conclude that the use of allogeneic SCT may be curative for such patients.

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Authors and Affiliations

  1. Department of Pediatric Hematology-Oncology, King Faisal Specialist Hospital & Research Center (KFSHRC), Riyadh, Saudi Arabia

    A Al-Ahmari, M Ayas, A Al-Jefri, M Al-Mahr, S Rifai & H El Solh

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  1. A Al-Ahmari
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  2. M Ayas
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  3. A Al-Jefri
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  4. M Al-Mahr
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  5. S Rifai
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  6. H El Solh
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Correspondence to M Ayas.

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Al-Ahmari, A., Ayas, M., Al-Jefri, A. et al. Allogeneic stem cell transplantation for patients with congenital amegakaryocytic thrombocytopenia (CAT). Bone Marrow Transplant 33, 829–831 (2004). https://doi.org/10.1038/sj.bmt.1704445

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  • DOI: https://doi.org/10.1038/sj.bmt.1704445

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