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Prepublished online as a Blood First Edition Paper on August 8, 2002; DOI 10.1182/blood-2002-02-0583.
Submitted February 22, 2002
Accepted July 18, 2002
Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hematopoietic allograft: a survey of the European experience 1985 - 2000
Reinhard A Seger*, Tayfun Gungor, Bernd H Belohradsky, Stephane Blanche, Pierre Bordigoni, Paolo Di Bartolomeo, Terence Flood, Paul Landais, Susanna Mueller, Hulya Ozsahin, Justen H Passwell, Fulvio Porta, Shimon Slavin, Nico Wulffraat, Felix Zintl, Arnon Nagler, Andrew Cant, and Alain Fischer
Division of Immunology/Hematology, University Children's Hospital, Zurich, Switzerland
Dr. von Haunersches Kinderspital, Ludwig Maximilians University, Munich, Germany
Unite d'Immuno Hematologie et Service de Biostatistique, Hopital Necker - Enfants Malades, Paris, France
Service de Medecine Infantile, Centre Hospitalier Universitaire, Nancy, France
Dipartimento di Ematologia e Oncologia, Ospedale Civile, Pescara, Italy
Pediatric Immunology Unit, Newcastle General Hospital, Newcastle, United Kingdom
BMT, Universitaetskinderklinik Ulm, Ulm, Germany
Department of Pediatrics, Chaim Sheba Medical Center, Tel-Hashomer, Israel
Clinica Pediatrica, University of Brescia, Brescia, Italy
Department of Bone Marrow Transplantation, Hadassah University Hospital, Jerusalem, Israel
Wilhelmina Kinderziekenhuis, University of Utrecht, Utrecht, The Netherlands
Klinik fuer Kinder- und Jugendmedizin, University of Jena, Jena, Germany
* Corresponding author; email: rseger{at}kispi.unizh.ch.
Treatment of chronic granulomatous disease (CGD) with myeloablative bone marrow transplantation is considered risky. This study investigated complications and survival according to different risk factors present at transplantation. The outcomes of 27 transplantations for CGD, from 1985 to 2000, reported to the European Bone Marrow Transplant Registry for primary immunodeficiencies were assessed. Most transplant recipients were children (n=25), received a myeloablative busulphan-based regimen (n=23) and had unmodified marrow allografts (n=23) from HLA-identical sibling donors (n=25). After myeloablative conditioning all patients fully engrafted with donor cells, after myelosuppressive regimens 2 out of 4 patients. Severe (grades III or IV) GvHD disease developed in 4 patients: 3 of 9 with preexisting overt infection, 1 of 2 with acute inflammatory disease. Exacerbation of infection during aplasia was observed in 3 patients, inflammatory flare at the infection site during neutrophil engraftment in 2: all 5 patients belonging to the subgroup of 9 with preexisting infection. Overall survival was 23/27, with 22/23 cured of CGD (median follow up 2 years). Survival was especially good in patients without infection at the moment of transplantation (18/18). Preexisting infections and inflammatory lesions have cleared in all survivors (except in one with autologous reconstitution). Myeloablative conditioning followed by transplantation of unmodified hemopoietic stem cells is a valid therapeutic option for children with CGD having an HLA-identical donor, if performed at the first signs of a severe course of the disease.
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