Successful hematopoietic stem cell transplantation for osteopetrosis using reduced intensity conditioning.
Bella ShadurIrina ZaidmanAdeeb NaserEddinElana LokshinFatma HusseinHodaya Cohen OronBatia AvniSigal GrisariuPolina StepenskyPublished in: Pediatric blood & cancer (2018)
We conclude that transplantation in children with IMO using fludarabine, treosulphan, thiotepa, and antithymocyte globulin is safe and effective and should be performed as early as possible following diagnosis, prior to the development of severe disease sequelae.