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Blood, 15 March 2001, Vol. 97, No. 6, pp. 1598-1603
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Impact of donor type on outcome of bone marrow transplantation
for Wiskott-Aldrich syndrome: collaborative study of the International
Bone Marrow Transplant Registry and the National Marrow
Donor Program
Alexandra H. Filipovich,
Judy V. Stone,
Sandra C. Tomany,
Michele Ireland,
Craig Kollman,
Corey J. Pelz,
James T. Casper,
Morton J. Cowan,
John R. Edwards,
Anders Fasth,
Robert Peter Gale,
Anne Junker,
Naynesh R. Kamani,
Brett J. Loechelt,
Daniel W. Pietryga,
Olle Ringdén,
Marcus Vowels,
Janet Hegland,
Aronica V. Williams,
John P. Klein,
Kathleen A. Sobocinski,
Philip A. Rowlings, and
Mary M. Horowitz
From the Statistical Center of the International Bone
Marrow Transplant Registry, Health Policy Institute, Medical College of
Wisconsin, Milwaukee, WI; the Coordinating Center of the National
Marrow Donor Program, Minneapolis, MN; Hematology/Oncology Division,
Children's Hospital Medical Center, Cincinnati, OH; Division of
Pediatric Hematology/Oncology, Medical College of Wisconsin, Milwaukee,
WI; Pediatric Blood and Bone Marrow Transplantation, DeVos Children's
Hospital, Grand Rapids, MI; Pediatric Bone Marrow Transplant Program,
University of California, San Francisco, CA; Bone Marrow Transplant
Center, Walt Disney Memorial Cancer Institute, Orlando, FL; Department
of Pediatrics, University of Göteborg, Göteborg, Sweden;
Division of Infectious and Immunological Diseases, British Columbia's
Children's Hospital, Vancouver, BC, Canada; Pediatric Bone Marrow
Transplantation, University of Texas Health Science Center, San
Antonio, TX; Children's National Medical Center, Washington, DC;
Division of Clinical Immunology, Karolinska Institute, Huddinge
University Hospital, Huddinge, Sweden; and Department of Haematology
and Oncology, Sydney Children's Hospital, Randwick, NSW, Australia.
Human leukocyte antigen (HLA)-identical sibling bone marrow
transplantation is an effective treatment for Wiskott-Aldrich syndrome.
However, most children with this disease lack such donors and many
patients receive transplants from alternative donors. This study
compared outcomes of HLA-identical sibling, other related donor, and
unrelated donor transplantation for Wiskott-Aldrich syndrome. The
outcome of 170 transplantations for Wiskott-Aldrich syndrome, from 1968 to 1996, reported to the International Bone Marrow Transplant Registry
and/or National Marrow Donor Program were assessed. Fifty-five were
from HLA-identical sibling donors, 48 from other relatives, and 67 from
unrelated donors. Multivariate proportional hazards regression was used
to compare outcome by donor type and identify other prognostic factors.
Most transplant recipients were younger than 5 years (79%), had a
pretransplantation performance score greater than or equal to 90%
(63%), received pretransplantation preparative regimens
without radiation (82%), and had non-T-cell-depleted grafts (77%).
Eighty percent received their transplant after 1986. The 5-year
probability of survival (95% confidence interval) for all subjects was
70% (63%-77%). Probabilities differed by donor type: 87%
(74%-93%) with HLA-identical sibling donors, 52% (37%-65%) with
other related donors, and 71% (58%-80%) with unrelated
donors (P = .0006). Multivariate analysis indicated
significantly lower survival using related donors other than
HLA-identical siblings (P = .0004) or unrelated donors in boys older than 5 years (P = .0001), compared to
HLA-identical sibling transplants. Boys receiving an unrelated donor
transplant before age 5 had survivals similar to those receiving
HLA-identical sibling transplants. The best transplantation outcomes in
Wiskott-Aldrich syndrome are achieved with HLA-identical sibling
donors. Equivalent survivals are possible with unrelated donors
in young children.
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