Successful treatment of an infant with CDA type II by intrauterine transfusions and postnatal stem cell transplantation

Authors

  • M. Braun MD,

    1. Pediatric Hematology and Oncology, Pediatric Stem Cell Transplantation Program, University Children's Hospital Wuerzburg, Wuerzburg, Germany
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  • M. Wölfl MD,

    1. Pediatric Hematology and Oncology, Pediatric Stem Cell Transplantation Program, University Children's Hospital Wuerzburg, Wuerzburg, Germany
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  • V. Wiegering MD,

    1. Pediatric Hematology and Oncology, Pediatric Stem Cell Transplantation Program, University Children's Hospital Wuerzburg, Wuerzburg, Germany
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  • B. Winkler MD,

    1. Pediatric Hematology and Oncology, Pediatric Stem Cell Transplantation Program, University Children's Hospital Wuerzburg, Wuerzburg, Germany
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  • K. Ertan MD,

    1. Department of Obstetrics, Gynecology and Prenatal Medicine, Klinikum, Leverkusen, Germany
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  • R. Bald MD,

    1. Department of Obstetrics, Gynecology and Prenatal Medicine, Klinikum, Leverkusen, Germany
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  • K. Schwarz MD,

    1. Institute for Transfusion Medicine, University of Ulm and Institute for Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Service, Baden-Württemberg – Hessen, Germany
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  • H. Heimpel MD,

    1. Department of Internal Medicine III, University Hospital Ulm, Ulm, Germany
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  • M. Eyrich MD,

    1. Pediatric Hematology and Oncology, Pediatric Stem Cell Transplantation Program, University Children's Hospital Wuerzburg, Wuerzburg, Germany
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  • P.G. Schlegel MD

    Corresponding author
    1. Pediatric Hematology and Oncology, Pediatric Stem Cell Transplantation Program, University Children's Hospital Wuerzburg, Wuerzburg, Germany
    • Correspondence to: Paul G Schlegel, Department of Pediatric Hematology, Oncology, Pediatric Stem cell Transplantation Program, University Children's Hospital and Comprehensive Cancer Center, Josef-Schneider Str. 2, D-97080 Wuerzburg, Germany.

      E-mail: schlegel@mail.uni-wuerzburg.de

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  • Eyrich and Schlegel contributed equally to this work.
  • Disclosure Statement: M.B. is a fellow of the Else-Kröner-Forschungskolleg Würzburg for Translational Immunology. The other authors declare no competing financial interests.
  • Conflict of Interest: Nothing to Report.

Abstract

Congenital dyserythropoietic anemias are rare hematological disorders leading to ineffective erythropoiesis with chronic anemia, complicated by iron overload. Here we present a remarkable clinical course of an infant with CDA type II who first presented as a severe fetal hydrops, requiring serial intrauterine red cell transfusions. While postnatal transfusion dependency persisted, the patient was successfully transplanted with a myeloablative conditioning regimen and peripheral blood stem cells of a matched donor. We believe that allogeneic HSCT is a reasonable therapeutic approach for patients with very severe CDA, even if only a matched unrelated donor is available. Pediatr Blood Cancer 2014;61:743–745. © 2013 The Authors. Pediatric Blood & Cancer, published by Wiley Periodicals, Inc.

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