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, Hsu Lewis, Joanne Kurtzberg, Kwaku Ohene-Frempong, Nancy Bunin, Francoise Bernaudin, Wing Yen Wong, J. Paul ScottDavid Margolis, Elliott Vichinsky, Donna A. Wall, Allen S. Wayne, Charles Pegelow, Rupa Redding-Lallinger, Joseph Wiley, Martin Klemperer, William C. Mentzer, Franklin O. Smith, Keith M. Sullivan

Research output: Contribution to journalArticle

280 Citations (Scopus)

Abstract

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. (C) 2000 by The American Society of Hematology.

Original languageEnglish (US)
Pages (from-to)1918-1924
Number of pages7
JournalBlood
Volume95
Issue number6
StatePublished - Mar 15 2000
Externally publishedYes

Fingerprint

Sickle Cell Anemia
Bone Marrow Transplantation
Bone
Transplantation
Neurology
Acute Chest Syndrome
Imaging techniques
Busulfan
Pulmonary diseases
Magnetic resonance
Gonadotropins
Allografts
Stroke
Lung
Erythropoiesis
Central Nervous System Diseases
Respiratory Function Tests
Homologous Transplantation
Growth
Lung Diseases

ASJC Scopus subject areas

  • Hematology

Cite this

Walters, M. C., Storb, R., Patience, M., Leisenring, W., Taylor, T., Sanders, J. E., ... Sullivan, K. M. (2000). Impact of bone marrow transplantation for symptomatic sickle cell disease: An interim report. Blood, 95(6), 1918-1924.

Impact of bone marrow transplantation for symptomatic sickle cell disease : An interim report. / Walters, Mark C.; Storb, Rainer; Patience, Melinda; Leisenring, Wendy; Taylor, Terri; Sanders, Jean E.; Buchanan, George E.; Rogers, Zora R.; Dinndorf, Patricia; Davies, Sally C.; Roberts, Irene A G; Dickerhoff, Rosarita; Yeager, Andrew M; Lewis, Hsu; Kurtzberg, Joanne; Ohene-Frempong, Kwaku; Bunin, Nancy; Bernaudin, Francoise; Wong, Wing Yen; Scott, J. Paul; Margolis, David; Vichinsky, Elliott; Wall, Donna A.; Wayne, Allen S.; Pegelow, Charles; Redding-Lallinger, Rupa; Wiley, Joseph; Klemperer, Martin; Mentzer, William C.; Smith, Franklin O.; Sullivan, Keith M.

In: Blood, Vol. 95, No. 6, 15.03.2000, p. 1918-1924.

Research output: Contribution to journalArticle

Walters, MC, Storb, R, Patience, M, Leisenring, W, Taylor, T, Sanders, JE, Buchanan, GE, Rogers, ZR, Dinndorf, P, Davies, SC, Roberts, IAG, Dickerhoff, R, Yeager, AM, Lewis, H, Kurtzberg, J, Ohene-Frempong, K, Bunin, N, Bernaudin, F, Wong, WY, Scott, JP, Margolis, D, Vichinsky, E, Wall, DA, Wayne, AS, Pegelow, C, Redding-Lallinger, R, Wiley, J, Klemperer, M, Mentzer, WC, Smith, FO & Sullivan, KM 2000, 'Impact of bone marrow transplantation for symptomatic sickle cell disease: An interim report', Blood, vol. 95, no. 6, pp. 1918-1924.
Walters MC, Storb R, Patience M, Leisenring W, Taylor T, Sanders JE et al. Impact of bone marrow transplantation for symptomatic sickle cell disease: An interim report. Blood. 2000 Mar 15;95(6):1918-1924.
Walters, Mark C. ; Storb, Rainer ; Patience, Melinda ; Leisenring, Wendy ; Taylor, Terri ; Sanders, Jean E. ; Buchanan, George E. ; Rogers, Zora R. ; Dinndorf, Patricia ; Davies, Sally C. ; Roberts, Irene A G ; Dickerhoff, Rosarita ; Yeager, Andrew M ; Lewis, Hsu ; Kurtzberg, Joanne ; Ohene-Frempong, Kwaku ; Bunin, Nancy ; Bernaudin, Francoise ; Wong, Wing Yen ; Scott, J. Paul ; Margolis, David ; Vichinsky, Elliott ; Wall, Donna A. ; Wayne, Allen S. ; Pegelow, Charles ; Redding-Lallinger, Rupa ; Wiley, Joseph ; Klemperer, Martin ; Mentzer, William C. ; Smith, Franklin O. ; Sullivan, Keith M. / Impact of bone marrow transplantation for symptomatic sickle cell disease : An interim report. In: Blood. 2000 ; Vol. 95, No. 6. pp. 1918-1924.
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abstract = "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. (C) 2000 by The American Society of Hematology.",
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AU - Taylor, Terri

AU - Sanders, Jean E.

AU - Buchanan, George E.

AU - Rogers, Zora R.

AU - Dinndorf, Patricia

AU - Davies, Sally C.

AU - Roberts, Irene A G

AU - Dickerhoff, Rosarita

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AU - Lewis, Hsu

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AU - Ohene-Frempong, Kwaku

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AU - Scott, J. Paul

AU - Margolis, David

AU - Vichinsky, Elliott

AU - Wall, Donna A.

AU - Wayne, Allen S.

AU - Pegelow, Charles

AU - Redding-Lallinger, Rupa

AU - Wiley, Joseph

AU - Klemperer, Martin

AU - Mentzer, William C.

AU - Smith, Franklin O.

AU - Sullivan, Keith M.

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N2 - 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. (C) 2000 by The American Society of Hematology.

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