Improved Outcomes of Hematopoietic Stem Cell Transplantation in Patients With Infantile Malignant Osteopetrosis Using Fludarabine-Based Conditioning.
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.
Department of Pediatrics, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Juma
J
Natsheh
Faculty of Medicine, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Genady
G
Drozdinsky
Department of Medical Imaging, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Natalia
N
Simanovsky
Department of Orthopedic Surgery, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Ron
R
Lamdan
Department of Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Odeya
O
Erlich
Department of Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Natan
N
Gorelik
Department of Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Reuven
R
Or
Department of Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Michael
M
Weintraub
Department of Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Polina
P
Stepensky
D018572:Disease-Free Survival; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation; D006504:Hepatic Veno-Occlusive Disease / Q000209:etiology; D006801:Humans; D006934:Hypercalcemia / Q000209:etiology; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D010022:Osteopetrosis / Q000401:mortality / Q000628:therapy*; D011183:Postoperative Complications; D014019:Tissue Donors; D014022:Tissue Survival; D019172:Transplantation Conditioning / Q000379:methods*; D016896:Treatment Outcome; D014740:Vidarabine / Q000031:analogs & derivatives* / Q000494:pharmacology
D014740:Vidarabine; C024352:fludarabine
conditioning; fludarabine; hematopoietic; otsteopetrosis; protocols; transplantation