Open-Label Pilot Study of Interferon Gamma-1b in Patients With Non-Infantile Osteopetrosis.

ABSTRACT: The only treatment currently available for patients with severe infantile osteopetrosis is hematopoietic cell transplantation (HCT). HCT-related toxicity and mortality risks typically preclude its use in non-infantile patients, and other therapies are needed for these patients who have significant disease-related morbidity. Interferon gamma-1b is currently approved by the U.S. Food and Drug Administration (FDA) for treatment of severe infantile osteopetrosis (autosomal recessive osteopetrosis [ARO]). However, little is known about the effects of interferon gamma-1b in non-infantile osteopetrosis. Thus, this pilot study aimed at testing the safety and tolerability of interferon gamma-1b in patients with non-infantile osteopetrosis and assessing the clinical effects. We performed a 12-month,

SUBMITTER: Nguyen A 

PROVIDER: S-EPMC8914146 | biostudies-literature | 2022 Mar

REPOSITORIES: biostudies-literature