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Blood, Vol. 95 No. 6 (March 15), 2000:
pp. 1918-1924
Impact of bone marrow transplantation for symptomatic sickle cell
disease: an interim report
Mark C. Walters,
Rainer Storb,
Melinda Patience,
Wendy Leisenring,
Terri Taylor,
Jean E. Sanders,
George E. Buchanan,
Zora R. Rogers,
Patricia Dinndorf,
Sally C. Davies,
Irene A. G. Roberts,
Rosarita Dickerhoff,
Andrew
M. Yeager,
Lewis Hsu,
Joanne Kurtzberg,
Kwaku Ohene-Frempong,
Nancy Bunin,
Francoise Bernaudin,
Wing-Yen Wong,
J. Paul Scott,
David Margolis,
Elliott Vichinsky,
Donna A. Wall,
Allen S. Wayne,
Charles Pegelow,
Rupa Redding-Lallinger,
Joseph Wiley,
Martin Klemperer,
William C. Mentzer,
Franklin O. Smith, and
Keith M. Sullivan for the Multicenter
Investigation of Bone Marrow Transplantation for Sickle Cell Disease
From the Fred Hutchinson Cancer Research Center, Seattle, WA;
University of Washington, Seattle, WA; University of Texas Southwestern
Medical Center, Dallas, TX; Children's Hospital National Medical
Center and George Washington University, Washington, DC; Royal
Postgraduate Medical School, London, U.K.; University of Bonn, Sankt
Augustin, Germany; Emory University, Atlanta, GA; Duke University,
Durham, NC; Children's Hospital of Philadelphia and University of
Pennsylvania, Philadelphia, PA; Hospital Henri Mondor, Creteil, France,
University of Southern California, Los Angeles, CA; Children's
Hospital of Wisconsin and Medical College of Wisconsin, Milwaukee, WI;
Children's Hospital of Oakland, Oakland, CA; St. Louis University, St.
Louis, MO; University of Miami, Miami, FL; University of North
Carolina, Chapel Hill, NC; University of South Florida, St. Petersburg,
FL; University of California, San Francisco, CA; and the Indiana
University School of Medicine, Indianapolis, IN.
Fifty children who had symptomatic sickle cell disease received
matched sibling marrow allografts between September 1991 and March
1999, with Kaplan-Meier probabilities of survival and event-free survival of 94% and 84%, respectively. Twenty-six patients (16 male,
10 female) had at least 2 years of follow-up after transplantation and
were evaluated for late effects of transplantation and for its impact
on sickle cell-related central nervous system (CNS) and pulmonary
disease. Patients ranged between 3.3 and 14.0 (median, 9.4) years of
age and had a median follow-up of 57.9 (range 38-95) months after
transplantation. Among 22 of 26 patients who had stable donor
engraftment, complications related to sickle cell disease resolved, and
none experienced further episodes of pain, stroke, or acute chest
syndrome. All 10 engrafted patients with a prior history of stroke had
stable or improved cerebral magnetic resonance imaging results.
Pulmonary function tests were stable in 22 of the 26 patients, worse in
two, and not studied in two. Seven of eight patients transplanted for
recurrent acute chest syndrome had stable pulmonary function. Linear
growth measured by median height standard deviation score improved from
 0.7 before transplantation to 0.2 after transplantation. An
adverse effect of busulfan conditioning on ovarian function was
demonstrated in five of seven evaluable females who are currently at
least 13 years of age. None of the four males tested had elevated serum gonadotropin levels. These data confirm that allogenic bone marrow transplantation establishes normal erythropoiesis and is associated with improved growth and stable CNS imaging and pulmonary function in
most patients.
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