Treosulfan-based conditioning regimen in a second matched unrelated peripheral blood stem cell transplantation for a pediatric patient with CGD and invasive aspergillosis, who experienced initial graft failure after RIC

  • Maja Agnieszka Klaudel-Dreszler
  • Krzysztof Kalwak
  • Magdalena Kurenko-Deptuch
  • Beata Wolska-Kusnierz
  • Edyta Heropolitanska-Pliszka
  • Barbara Pietrucha
  • Bożena Mikoluc
  • Ewa Gorczyńska
  • Marek Ussowicz
  • Alicja Chybicka
  • Ewa Bernatowska
Case Report

Abstract

Chronic granulomatous disease (CGD) is a rare primary immunodeficiency caused by a defect of phagocyte NADPH-oxidase and characterized by severe, recurrent bacterial and fungal infections. Invasive aspergillosis (IA) is the leading cause of mortality in patients with CGD. We report the case of a 3-year-old boy with CGD, who developed IA despite antifungal prophylaxis. His treatment consisted of a 10-month-long multi-drug antifungal therapy, together with surgery, but these did not cause any substantial clinical improvement. BMT in high-risk patients with CGD remains a challenge due to both, higher risk of graft rejection and inflammatory flare in the course of immune recovery. Our patient rejected the first matched unrelated donor (MUD) allograft after RIC regimen recommended by the EBMT Inborn Errors Working Party for high-risk patients. After treosulfan-based conditioning and second MUD peripheral blood stem cell transplantation both, full reconstitution of the granulocytic series and complete recovery from IA, were achieved.

Keywords

Treosulfan Peripheral blood stem cell transplantation Invasive aspergillosis Primary immunodeficiency 

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Copyright information

© The Japanese Society of Hematology 2009

Authors and Affiliations

  • Maja Agnieszka Klaudel-Dreszler
    • 1
  • Krzysztof Kalwak
    • 2
  • Magdalena Kurenko-Deptuch
    • 1
  • Beata Wolska-Kusnierz
    • 1
  • Edyta Heropolitanska-Pliszka
    • 1
  • Barbara Pietrucha
    • 1
  • Bożena Mikoluc
    • 3
  • Ewa Gorczyńska
    • 2
  • Marek Ussowicz
    • 2
  • Alicja Chybicka
    • 2
  • Ewa Bernatowska
    • 1
  1. 1.Department of ImmunologyChildren’s Memorial Health InstituteWarsawPoland
  2. 2.Department of Pediatric Hematology, Oncology and Bone Marrow TransplantationWroclaw Medical UniversityWroclawPoland
  3. 3.Department of Pediatrics and Developmental Disorders of Children and AdolescentsMedical UniversityBialystokPoland

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