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Prepublished online as a Blood First Edition Paper on August 8, 2002; DOI 10.1182/blood-2002-02-0583.
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Blood, 15 December 2002, Vol. 100, No. 13, pp. 4344-4350
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Treatment of chronic granulomatous disease with myeloablative
conditioning and an unmodified hemopoietic 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 Müller,
Hulya Ozsahin,
Justen H. Passwell,
Fulvio Porta,
Shimon Slavin,
Nico Wulffraat,
Felix Zintl,
Arnon Nagler,
Andrew Cant, and
Alain Fischer
From the European Group for Blood and Marrow
Transplantation (EBMT) and the European Society for Immunodeficiencies
(ESID); Division of Immunology/Hematology, University
Children's Hospital, Zurich, Switzerland; Dr von Haunersches
Kinderspital, Ludwig Maximilians University, Munich,
Germany; Unité d'Immuno-Hématologie et
Service de Biostatistique, Hôpital Necker-Enfants Malades,
Paris, France; Service de Médecine Infantile, Centre
Hospitalier Universitaire, Nancy, France; Dipartimento di
Ematologia e Oncologia, Ospedale Civile, Pescara, Italy;
Pediatric Immunology Unit, Newcastle General Hospital, United Kingdom;
Universitätskinderklinik Ulm, Germany; Department of
Pediatrics, Chaim Sheba Medical Center, Tel-Hashomer,
Israel; Clinica Pediatrica, University of Brescia,
Italy; Department of Bone Marrow Transplantation, Hadassah
University Hospital, Jerusalem, Israel; Wilhelmina
Kinderziekenhuis, University of Utrecht, The Netherlands;
and Klinik für Kinder-und Jugendmedizin, University of Jena,
Germany.
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 human leukocyte antigen
(HLA)-identical sibling donors (n = 25). After myeloablative
conditioning, all patients fully engrafted with donor cells; after
myelosuppressive regimens, 2 of 4 patients fully engrafted.
Severe (grade 3 or 4) graft-versus-host disease (GVHD) disease
developed in 4 patients: 3 of 9 with pre-existing 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
belonged to the subgroup of 9 with pre-existing infection. Overall
survival was 23 of 27, with 22 of 23 cured of CGD (median follow-up, 2 years). Survival was especially good in patients without infection at
the moment of transplantation (18 of 18). Pre-existing 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, if performed at
the first signs of a severe course of the disease, is a valid
therapeutic option for children with CGD having an HLA-identical donor.
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