Long-term Clinical Outcome of Patients with Severe Combined Immunodeficiency Who Received Related Donor Bone Marrow Transplants without Pretransplant Chemotherapy or Post-transplant GVHD Prophylaxis
Received 7 January 2009; received in revised form 8 June 2009; accepted 21 July 2009. published online 12 October 2009.
Refers to article:
Identification of Severe Combined Immunodeficiency by T-Cell Receptor Excision Circles Quantification Using Neonatal Guthrie Cards
, 23 July 2009
Yoichi Morinishi, Kohsuke Imai, Noriko Nakagawa, Hiroki Sato, Katsuyuki Horiuchi, Yoshitoshi Ohtsuka, Yumi Kaneda, Takashi Taga, Hiroaki Hisakawa, Ryosuke Miyaji, Mikiya Endo, Tsutomu Oh–ishi, Yoshiro Kamachi, Koshi Akahane, Chie Kobayashi, Masahiro Tsuchida, Tomohiro Morio, Yoji Sasahara, Satoru Kumaki, Keiko Ishigaki, Makoto Yoshida, Tomonari Urabe, Norimoto Kobayashi, Yuri Okimoto, Janine Reichenbach, Yoshiko Hashii, Yoichiro Tsuji, Kazuhiro Kogawa, Seiji Yamaguchi, Hirokazu Kanegane, Toshio Miyawaki, Masafumi Yamada, Tadashi Ariga, Shigeaki Nonoyama
The Journal of Pediatrics
December 2009 (Vol. 155, Issue 6, Pages 829-833) Abstract |
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Objective
To determine long-term health benefits of nonablative bone marrow transplantation for severe combined immunodeficiency (SCID), we investigated our cohort of 161 related donor bone marrow–transplanted patients with SCID. Only 16 (10%) had HLA-identical donors.
Study design
All 124 survivors were sent questionnaires about their current clinical statuses. Details from clinic visits were also compiled. One hundred eleven patients (90%) were reached. We compared outcomes of patients transplanted before and after 3.5 months of life and by molecular defect.
Results
The overall survival rate was 77%, but the rate for the 48 infants transplanted in the first 3.5 months of life was 94%, compared with 70% for the 113 transplanted after 3.5 months (P = .002). Twenty-eight (76%) of the 37 deceased patients died of viral infections present at diagnosis. One or more clinical problems were reported to have been present in the past 2 years in 71 (64%) of the survivors, although 95 (86%) were considered healthy by their families.
Conclusions
Most patients with SCID transplanted with related donor marrow without pretransplant chemotherapy have done well in the long term, but those transplanted at <3.5 months of age had a superior survival rate, a lower rate of clinical problems, less need for booster transplants, and better nutritional status.
aDepartment of Pediatrics, Duke University Medical Center, Durham, NC
bDepartment of Immunology, Duke University Medical Center, Durham, NC
cDepartment of Biostatistics and Bioinformatics, Duke University Medical Center, Durham, NC
dDuke Clinical Research Institute, Duke University Medical Center, Durham, NC
Reprint requests: Dr Rebecca H. Buckley, Box 2898, 363 Jones Building, Duke University Medical Center, Durham, NC 27710.
Supported by grants from the National Institute of Allergy and Infectious Diseases (AI042951 and AI47605 ) and a grant from the National Center for Research Resources (NCRR) (#1UL1 RR024128-01), a component of the National Institutes of Health (NIH) and NIH Roadmap for Medical Research. The authors declare no conflicts of interest.
ABSTRACT