Methods
deaths
This is an ongoing non-randomized, open-label, single-arm, multicenter Phase II trial (NCT03988608; registered 17/06/2019) investigating the efficacy and safety of eltrombopag in Chinese patients with r/r SAA. Eligible Chinese patients were ≥ 18 years with a previous diagnosis of SAA (defined as bone marrow cellularity < 25%, or 25–50%, with ˂ 30% residual hemopoietic cells and ≥ 2 of the following in peripheral blood samples: absolute neutrophil count ˂ 0.5 × 10All patients received a starting dose of eltrombopag monotherapy of 25 mg/d. This dose increased by 25 mg/d every 2 weeks, according to the platelet count, up to the maximum dose of 150 mg/d (Supplementary Fig. The primary endpoint was the hematologic response (HR) rate at Week 26, defined as the proportion of patients who met any of the International Working Group criteriaIn addition to the HR reported by investigators in the response assessment case report form, HR was also derived from International Working Group criteria programmatically using laboratory results and transfusion records, and was referred to as the derived HR.During this study, the COVID-19 pandemic resulted in the implementation of changes to the conduct of the study to ensure the safety and well-being of study patients, and to enable trial oversight and compliance with the study protocol. These protocol amendments in line with COVID-19 regulations included changes in (1) study procedures, allowing for replacement of on-site monitoring by remote monitoring at some sites when considered necessary, (2) study drug supply method, that allowed for home delivery of the study treatment, and (3) remote monitoring of study sites as applicable. These unforeseen changes were implemented as of March 2020 based on needs of individual sites to address the challenges of the pandemic. There were no reports of suspected or confirmed cases or deaths related to COVID-19 in this trial at the cutoff date.Only data available at the cutoff date of July 16, 2021, are reported here.