Swedish Orphan Biovitrum
This study aims to evaluate safety information in patients with HLH/MAS in Still's disease exposed to Gamifant.
This is a prospective, low intervention, multicenter safety study to evaluate and characterize the long-term safety of Gamifant, an approved treatment for patients with HLH/MAS in known or suspected Still's disease. Pediatric and adult patients with HLH/MAS in known or suspected Still's disease, including sJIA, with inadequate response or intolerance to glucocorticoids, or with recurrent MAS, and who have either initiated Gamifant within the past 0 to 7 days or are prescribed to initiate Gamifant in accordance with the approved product label, are eligible for inclusion in the study.
Inclusion Criteria: * Has received at least 1 dose of Gamifant 0-7 days prior to ICF date or is prescribed to initiate Gamifant. * Aged ≤80 years at index Gamifant administration. * Diagnosis of secondary HLH/MAS according to the treating physician. * Diagnosis or suspicion of Still's disease (sJIA or AOSD) according to the treating physician. * Patients who have shown inadequate response or intolerance to high-dose glucocorticoids or who have recurrent MAS, according to the treating physician. Recurrent MAS is defined as any MAS episode that has occurred after resolution of the first-ever MA…
Protocol-mandated assessments include specified laboratory testing at baseline and during the initial 12 months of follow-up (Week 1, Week 2, Month 1, Month 3, Month 6, Month 12): complete blood counts, differential leukocyte count, serum biochemistry/liver function tests, CMV PCR. CMV serology is only required (per protocol) to be conducted at baseline. No laboratory testing is required per protocol after the 12-month visit.
Protocol-mandated assessments include specified laboratory testing at baseline and during the initial 12 months of follow-up (Week 1, Week 2, Month 1, Month 3, Month 6, Month 12): complete blood counts, differential leukocyte count, serum biochemistry/liver function tests, CMV PCR. CMV serology is only required (per protocol) to be conducted at baseline. No laboratory testing is required per protocol after the 12-month visit.