Successful hematopoietic stem cell transplantation for osteopetrosis using reduced intensity conditioning

Pediatric Blood & Cancer
Bella ShadurPolina Stepensky

Abstract

Infantile malignant osteopetrosis (IMO) is an autosomal recessive condition characterized by defective osteoclast activity, with hematopoietic bone marrow transplant being the only available cure. Over the past several years, new conditioning regimes and donor options have emerged, thus extending the possibility of cure to a greater number of patients and improving the outcomes of bone marrow transplant. Here we detail the outcomes of bone marrow transplant in a cohort of 31 patients treated with a combination of fludarabine, treosulphan, thiotepa, and antithymocyte globulin. Thirty-one patients with IMO who underwent hematopoietic stem cell transplantation with fludarabine, treosulphan, thiotepa, and antithymocyte globulin at our center from 2012 to 2017 are retrospectively reviewed in this study. Twenty-six patients were transplanted from 10/10 matched donors (13 from siblings, 11 from unrelated, and two from extended family donors), four from 9/10 matched unrelated donors, and one from a 9/10 matched family donor. Overall survival was 100% with a median follow-up of 363 days (range 74-1891). There were 12 cases of acute graft versus host disease (GvHD) (38.7%), no cases of veno-occlusive disease, and eight cases of hypercalc...Continue Reading

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Citations

Jul 2, 2019·NeoReviews·Erin Hall, Shalini Shenoy
Mar 7, 2019·Frontiers in Endocrinology·Sara PennaAnna Villa
May 18, 2020·Stem Cell Research & Therapy·Xiaojie XianIlana Moscatelli
Apr 25, 2020·Journal of Bone and Mineral Research : the Official Journal of the American Society for Bone and Mineral Research·Galina ShapiroRon Lamdan
Aug 9, 2020·International Journal of Molecular Sciences·Jean VacherMonica Pata
Sep 18, 2020·Tissue Engineering. Part B, Reviews·Eun-Jin LeeStella Alimperti
Dec 15, 2020·Pediatric Blood & Cancer·Ehud Even-Or, Polina Stepensky
May 11, 2021·Disease Models & Mechanisms·Sara PennaValentina Capo
Oct 22, 2021·Joint, Bone, Spine : Revue Du Rhumatisme·Marie-Charlotte TrojaniValérie Pierrefite-Carle

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