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Combination of umbilical cord blood with BM from a 2-month-old sibling as lifesaving BMT for very severe aplastic anemia

Bone Marrow Transplantation volume 42, pages 563–564 (2008)Cite this article

Very severe aplastic anemia is a well-defined life-threatening disease causing significant morbidity and mortality. Major causes of poor outcome include unresponsiveness to first-line immunosuppressive treatment (IST), infection, bleeding and absence of matched donor for hematopoietic SCT (HSCT). Here, we present a pediatric patient with very severe aplastic anemia, no matched donor at diagnosis, poor course of IST and early development of invasive mucormycosis, successfully treated with combined cord blood and BMT from his newborn brother.

A 5-year-old boy presented with progressive jaundice and elevation of liver function tests after a recent viral infection. Tests for hepatitis A, B and C, parvovirus–B19, rubella, HIV and CMV were negative. Over several weeks normalization of liver functions was observed, but simultaneously the patient developed progressive pancytopenia. His Hb reached 6.2 g per 100 ml, absolute neutrophil count 0.1 × 109/l and platelet count 4 × 109/l. BM examination supported a diagnosis of very severe aplastic anemia. Tests for chromosomal fragility and paroxysmal nocturnal hemoglobinuria were negative. An extended family search did not reveal a matched donor, so IST with thymoglobulin (2.5 mg/kg/day for 5 days) and CsA (3 mg/kg) was started. Multiple (>20) transfusions of plts and RBCs were given. The patient's mother was at this time in her 32nd week of pregnancy.

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Authors and Affiliations

  1. Hebrew University Medical Center, Jerusalem, Israel. E-mail: gashka@hadassah.org.il,

    P Stepensky, S Revel-Vilk, M Weintraub, E Waldman, M Harit, D Averbuch, J Weinberger, R Or & I Resnick

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  1. P Stepensky
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  2. S Revel-Vilk
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  3. M Weintraub
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  4. E Waldman
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  5. M Harit
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  7. J Weinberger
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  9. I Resnick
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Stepensky, P., Revel-Vilk, S., Weintraub, M. et al. Combination of umbilical cord blood with BM from a 2-month-old sibling as lifesaving BMT for very severe aplastic anemia. Bone Marrow Transplant 42, 563–564 (2008). https://doi.org/10.1038/bmt.2008.213

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