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Fludarabine-based Protocol for Haploidentical Peripheral Blood Stem Cell Transplantation in Hurler Syndrome

Kapelushnik, J. M.D.; Mandel, H. M.D.; Varadi, G. M.D.; Nagler, A. M.D., M.Sc.

Journal of Pediatric Hematology/Oncology: September-October 2000 - Volume 22 - Issue 5 - p 433-436
Clinical And Laboratory Observations
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To assess the feasibility of performing a haploidentical peripheral blood stem cell transplantation (PBSCT) in a child with Hurler syndrome after a novel conditioning regimen consisting of fludarabine monophosphate, anti-T-lymphocyte globulin, low-dose busulfan, and single-dose total body irradiation of 750 cGy. A 16-month old boy with Hurler syndrome underwent haploidentical PBSCT from his 3/6 HLA-matched sister. Pretransplant conditioning consisted of fludarabine (30 mg/m2 per day) from day −10 to day −5, busulfan (4 mg/kg per day) on days −7 and −6, rabbit anti-T-lymphocyte globulin (10 mg/kg per day) from day −4 to day −1, and total body irradiation of 750 cGy on day −1. In vitro T-cell depletion was carried out with rat antihuman CDw52 monoclonal antibody (Campath-1G). The fludarabine-based protocol was well-tolerated, with mild toxicity and no major transplant-related complications or graft-versus-host disease. Engraftment was complete and stable. Chimerism was 100% donor origin, as determined by restriction fragment length polymorphism. Cytogenetic and polymerase chain reaction-various number of tandem repeats (PCR-VNTR) analyses of peripheral blood and bone marrow showed 100% reconstitution with female donor cells. The patient underwent the transplant 30 months ago and is in good clinical condition, with normal counts, no signs of graft-versus-host disease, and no infectious episodes; neurologic signs have stabilized. Haploidentical PBSCT, T-cell-depleted by means of Campath-1G, may serve as a therapeutic alternative for patients with Hurler syndrome when a fully matched sibling is not available.

From the Department of Bone Marrow Transplantation (J.K., G.V., A.N.), Hadassah University Hospital, Jerusalem, Israel; and the Department of Pediatrics (H.M.), Rambam Medical Center, Haifa, Israel.

Submitted for publication June 1, 1999; accepted November 17, 1999.

Dr. Kapelushnik is currently with the Department of Pediatrics, Soroka Medical Center, Beersheba, Israel.

Address correspondence and reprint requests to A. Nagler, MD, MSc, Bone Marrow Transplantation Department, Hadassah University Hospital, P.O.B. 12000, Jerusalem, Israel IL-91120.

© 2000 Lippincott Williams & Wilkins, Inc.