This study aims to evaluate real-world effectiveness of pacritinib through a site-based retrospective chart review of medical records of adult patients with myelofibrosis treated with pacritinib in routine clinical settings with platelet count ≥50 x 10^9/L at the time of treatment initiation. Treatment effectiveness will be based on spleen size and symptom burden, hematological parameters, progression to leukemia, survival, pacritinib treatment patterns, patient demographic and clinical characteristics, and allogenic-hematopoietic stem cell transplant (allo-HSCT)-related outcomes. Data will be collected from medical charts of patients with pacritinib initiation between 01 June 2022 and 31 December 2025 with an end of study observation on 30 June 2026.
Epidermolysis Bullosa is a genetic disorder manifesting typically at birth comprising several genetic subtypes that manifest as blistering or erosion of the skin in response to little or no apparent trauma. This clinical study is being conducted for research purposes. The cream being studied has allantoin as the active ingredient and the aim of this study is to assess the long-term safety of this cream when applied to the skin of participants with Epidermolysis Bullosa. This study is an open label extension to study SD-007. The study will be conducted by the study doctor, in other words, the physician responsible for the clinical study at your child's study site. Your child's participation in the clinical study is expected to last for approximately 12 months and approximately eighty (80) children will participate in this study. There will be a total of five (5) study visits over the course of one year. Study visits 1, 3 and 5 will be in person and visits 2 and 4 will be telephone visits.