|
|
 Previous Article | Table of Contents | Next Article 
Splenectomy and/or bone marrow transplantation in the management of the
Wiskott-Aldrich syndrome: long-term follow-up of 62 cases
CA Mullen, KD Anderson and RM Blaese
Metabolism Branch, National Cancer Institute, National Institutes of
Health, Bethesda, MD 20892.
This study describes the effects of two major treatment options,
splenectomy and/or bone marrow transplantation, on the natural history of
the Wiskott-Aldrich (WAS) syndrome. The records of 62 patients with the WAS
evaluated at the National Institutes of Health Clinical Center from 1966 to
1992 were reviewed. Nineteen patients were treated with bone marrow
transplantation (BMT) and the results were largely dependent on the source
of the graft. Twelve of 12 patients receiving HLA-matched sibling marrow
achieved satisfactory immunologic and hematologic reconstitution. By
contrast, only 2 of 7 patients receiving haploidentical, parental, or
matched unrelated marrow survived more than 1 year after BMT. Thirty-nine
patients who lacked suitable bone marrow donors early in their course
underwent splenectomy for management of their thrombocytopenia; most
received prophylactic antibiotics to minimize the risk of sepsis. Nearly
all these patients achieved normal platelet counts and the rate of serious
bleeding was reduced nearly sevenfold. Median survival in the
untransplanted splenectomy group was 25 years, compared with less than 5
years in unsplenectomized patients. We conclude that HLA-matched sibling
donor BMT is the treatment of choice for patients with WAS and that
splenectomy and daily prophylactic antibiotics provide a significant
survival advantage to those boys without a matched sibling donor.
Splenectomy should probably be used in preference to unmatched BMT until
results with alternative donor BMT significantly improve or gene therapy
becomes available.
Volume 82,
Issue 10,
pp. 2961-2966,
11/15/1993
Copyright © 1993 by The American Society of Hematology
 CiteULike  Connotea  Del.icio.us  Digg  Reddit  Technorati What's this?
This article has been cited by other articles:
|
|
|
|
 
H. Falet, M. P. Marchetti, K. M. Hoffmeister, M. J. Massaad, R. S. Geha, and J. H. Hartwig
Platelet-associated IgAs and impaired GPVI responses in platelets lacking WIP
Blood,
November 19, 2009;
114(21):
4729 - 4737.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
C. L. Balduini and J. G. Drachman
Role of splenectomy in inherited thrombocytopenias
Blood,
August 15, 2004;
104(4):
1227 - 1227.
[Full Text]
[PDF]
|
|
|
|
|
|
|
P. Narayan, H. Alwair, and A. J. Bryan
Surgical resection of sequential thoracic aortic aneurysms in Wiskott-Aldrich syndrome
Interactive CardioVascular and Thoracic Surgery,
June 1, 2004;
3(2):
346 - 348.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
K. Imai, T. Morio, Y. Zhu, Y. Jin, S. Itoh, M. Kajiwara, J.-i. Yata, S. Mizutani, H. D. Ochs, and S. Nonoyama
Clinical course of patients with WASP gene mutations
Blood,
January 15, 2004;
103(2):
456 - 464.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
D. B. Cines, J. B. Bussel, R. B. McMillan, and J. L. Zehnder
Congenital and Acquired Thrombocytopenia
Hematology,
January 1, 2004;
2004(1):
390 - 406.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
S. Dupuis-Girod, J. Medioni, E. Haddad, P. Quartier, M. Cavazzana-Calvo, F. Le Deist, G. de Saint Basile, J. Delaunay, K. Schwarz, J.-L. Casanova, et al.
Autoimmunity in Wiskott-Aldrich Syndrome: Risk Factors, Clinical Features, and Outcome in a Single-Center Cohort of 55 Patients
Pediatrics,
May 1, 2003;
111(5):
e622 - 627.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
H. Falet, K. M. Hoffmeister, R. Neujahr, and J. H. Hartwig
Normal Arp2/3 complex activation in platelets lacking WASp
Blood,
August 28, 2002;
100(6):
2113 - 2122.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
K. Yamaguchi, T. Ariga, M. Yamada, D. L. Nelson, R. Kobayashi, C. Kobayashi, Y. Noguchi, Y. Ito, K. Katamura, Y. Nagatoshi, et al.
Mixed chimera status of 12 patients with Wiskott-Aldrich syndrome (WAS) after hematopoietic stem cell transplantation: evaluation by flow cytometric analysis of intracellular WAS protein expression
Blood,
July 30, 2002;
100(4):
1208 - 1214.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
T. S. Strom, X. Li, J. M. Cunningham, and A. W. Nienhuis
Correction of the murine Wiskott-Aldrich syndrome phenotype by hematopoietic stem cell transplantation
Blood,
May 29, 2002;
99(12):
4626 - 4628.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
A. H. Filipovich, J. V. Stone, S. C. Tomany, M. Ireland, C. Kollman, C. J. Pelz, J. T. Casper, M. J. Cowan, J. R. Edwards, A. Fasth, et al.
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
Blood,
March 15, 2001;
97(6):
1598 - 1603.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
R. H. Buckley
Primary Immunodeficiency Diseases Due to Defects in Lymphocytes
N. Engl. J. Med.,
November 2, 2000;
343(18):
1313 - 1324.
[Full Text]
[PDF]
|
|
|
|
|
|
|
A. M Jones and H. B Gaspar
Immunogenetics: changing the face of immunodeficiency
J. Clin. Pathol.,
January 1, 2000;
53(1):
60 - 65.
[Full Text]
[PDF]
|
|
|
|
|
|
|
B. S. Gross, J. I. Wilde, L. Quek, H. Chapel, D. L. Nelson, and S. P. Watson
Regulation and Function of WASp in Platelets by the Collagen Receptor, Glycoprotein VI
Blood,
December 15, 1999;
94(12):
4166 - 4176.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
M. D. Gallego, M. Santamaria, J. Pena, and I. J. Molina
Defective Actin Reorganization and Polymerization of Wiskott-Aldrich T Cells in Response to CD3-Mediated Stimulation
Blood,
October 15, 1997;
90(8):
3089 - 3097.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
C. Thomas, G. de Saint Basile, F. Le Deist, D. Theophile, M. Benkerrou, E. Haddad, S. Blanche, and A. Fischer
Correction of X-Linked Hyper-IgM Syndrome by Allogeneic Bone Marrow Transplantation
N. Engl. J. Med.,
August 17, 1995;
333(7):
426 - 429.
[Full Text]
[PDF]
|
|
|
|
|
|
|
F. S. Rosen, M. D. Cooper, and R. J.P. Wedgwood
The Primary Immunodeficiencies
N. Engl. J. Med.,
August 17, 1995;
333(7):
431 - 440.
[Full Text]
[PDF]
|
|
|
|
|
