Hematopoietic stem cell transplantation (Tehran University of Medical Sciences)

This is an allogeneic hematopoietic stem cell transplantation (HSCT) therapy developed by the Tehran University of Medical Sciences for the treatment of malignant infantile osteopetrosis (MIOP). MIOP is a rare, life-threatening genetic disorder characterized by defective osteoclast function, which leads to abnormally dense bone, marrow failure, and neurological complications. The therapy involves the infusion of healthy donor-derived hematopoietic stem cells—sourced from matched siblings, unrelated umbilical cord blood, or haploidentical donors—following a chemotherapy-based conditioning regimen (typically including busulfan, cyclophosphamide, fludarabine, and/or antithymocyte globulin) without total-body irradiation. The transplanted stem cells differentiate into functional osteoclasts, which restore normal bone resorption and hematopoiesis, offering a potentially curative outcome for this condition.

Other names

HSCT for Malignant Infantile OsteopetrosisAllogeneic Hematopoietic Stem Cell TransplantationHematopoietic stem cells