Improved Outcomes of Hematopoietic Stem Cell Transplantation in Patients With Infantile Malignant Osteopetrosis Using Fludarabine‐Based Conditioning. Issue 3 (20th October 2015)

• Record Type: Journal Article
• Authors: Natsheh, Juma; Drozdinsky, Genady; Simanovsky, Natalia; Lamdan, Ron; Erlich, Odeya; Gorelik, Natan; Or, Reuven; Weintraub, Michael; Stepensky, Polina
• Is Part Of: Pediatric blood & cancer. Volume 63:Issue 3(2016)
• Page Start: 535
• Page End: 540
• Publication Date: 2015-10-20
• Subjects: conditioning -- fludarabine -- hematopoietic -- otsteopetrosis -- protocols -- transplantation; Tumors in children -- Periodicals; Blood -- Diseases -- Periodicals; Cancer in children -- Periodicals; 618.92
• DOI: 10.1002/pbc.25801
• Languages: English
• ISSNs: 1545-5009

Abstract

Abstract : Background: Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for infantile malignant osteopetrosis (IMO), but is associated with a high incidence of adverse outcomes. In this study, we present our experience with HSCT for IMO patients comparing different types of conditioning regimens. Methods: Thirty‐eight patients with IMO (aged from 1 month to 6 years, median 0.66 years) who underwent allogeneic HSCT from 1983 in our hospital were included in this retrospective study. Fludarabine‐based conditioning regimens were used in 26 patients and 12 patients were transplanted using other conditioning regimens. Results: The overall survival after conditioning with fludarabine was 96% (25/26) versus 58% (7/12) for the alternative regimens ( P = 0.004), with significantly fewer adverse effects including hypercalcemia and veno‐occlusive disease of liver. All patients who survive are clinically well. Conclusions: We conclude that fludarabine‐based conditioning regimens are safe and effective in patients with IMO, improving morbidity and mortality related to HSCT.