Juma Natsheh1, Genady Drozdinsky2, Natalia Simanovsky3, Ron Lamdan4, Odeya Erlich5, Natan Gorelik5, Reuven Or6, Michael Weintraub5, Polina Stepensky5. 1. Department of Pediatrics, Hadassah Hebrew University Medical Center, Jerusalem, Israel. 2. Faculty of Medicine, Hadassah Hebrew University Medical Center, Jerusalem, Israel. 3. Department of Medical Imaging, Hadassah Hebrew University Medical Center, Jerusalem, Israel. 4. Department of Orthopedic Surgery, Hadassah Hebrew University Medical Center, Jerusalem, Israel. 5. Department of Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel. 6. Department of Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
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.
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.
Authors: Sara Penna; Valentina Capo; Eleonora Palagano; Cristina Sobacchi; Anna Villa Journal: Front Endocrinol (Lausanne) Date: 2019-02-19 Impact factor: 5.555
Authors: A C Lankester; M H Albert; C Booth; A R Gennery; T Güngör; M Hönig; E C Morris; D Moshous; B Neven; A Schulz; M Slatter; P Veys Journal: Bone Marrow Transplant Date: 2021-07-05 Impact factor: 5.483