Gentler chemo tested for rare genetic disorder before Life-Saving transplant
NCT ID NCT07316595
Summary
This study is testing whether using a lower dose of a chemotherapy drug called treosulfan before a stem cell transplant is safer and effective for patients with Nijmegen breakage syndrome. This rare genetic disorder weakens the immune system and increases cancer risk. The goal is to control the disease and reduce transplant complications by adjusting the drug dose based on whether a patient already has cancer.
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Conditions
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