Predictors for improvement in patient-reported outcomes: post hoc analysis of a phase 3 randomized, open-label study of eculizumab and ravulizumab in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria.

"Declarations Ethics approval and consent to participateThe protocol for study 301 (NCT02946463) was approved by the institutional review board or independent ethics committee at each participating center, and the study was conducted in accordance with the Declaration of Helsinki and the Council for International Organizations of Medical Sciences International Ethical Guidelines. All patients gave written informed consent. Competing interestsHubert Schrezenmeier has received travel support, fees, and research support (to University of Ulm) from Alexion, AstraZeneca Rare Disease, and Novartis, and fees (to University of Ulm) from Apellis, Roche, and Sanofi. Austin Kulasekararaj has received fees for service, travel support, and consulting fees from Alexion, AstraZeneca Rare Disease. Lindsay Mitchell has received fees from Alexion, AstraZeneca Rare Disease. Prof de Latour received fees for service, travel support, consulting fees and served as a member of an advisory board for Alexion, AstraZeneca Rare Disease, Novartis and Pfizer. Timothy Devos has served as a member of an advisory board for AbbVie, Alexion, AstraZeneca Rare Disease, Celgene/Bristol Myers Squibb, and Novartis. Shinichiro Okamoto has received fees and research funding from Alexion, AstraZeneca Rare Disease. Richard Wells has received fees and research funding from Alexion, AstraZeneca Rare Disease, Celgene, and Novartis, and consulting fees from Alexion, AstraZeneca Rare Disease. Evan Popoff and Antoinette Cheung are full-time employees of Broadstreet HEOR. Yogesh Patel is a full-time employee of Alexion, AstraZeneca Rare Disease. Ioannis Tomazos and Alice Wang were full-time employees of Alexion, AstraZeneca Rare Disease at time of study. Jong Wook Lee has received grants and fees from Alexion, AstraZeneca Rare Disease, and has served as a member of an advisory board for Alexion, AstraZeneca Rare Disease. Competing interests Hubert Schrezenmeier has received travel support, fees, and research support (to University of Ulm) from Alexion, AstraZeneca Rare Disease, and Novartis, and fees (to University of Ulm) from Apellis, Roche, and Sanofi. Austin Kulasekararaj has received fees for service, travel support, and consulting fees from Alexion, AstraZeneca Rare Disease. Lindsay Mitchell has received fees from Alexion, AstraZeneca Rare Disease. Prof de Latour received fees for service, travel support, consulting fees and served as a member of an advisory board for Alexion, AstraZeneca Rare Disease, Novartis and Pfizer. Timothy Devos has served as a member of an advisory board for AbbVie, Alexion, AstraZeneca Rare Disease, Celgene/Bristol Myers Squibb, and Novartis. Shinichiro Okamoto has received fees and research funding from Alexion, AstraZeneca Rare Disease. Richard Wells has received fees and research funding from Alexion, AstraZeneca Rare Disease, Celgene, and Novartis, and consulting fees from Alexion, AstraZeneca Rare Disease. Evan Popoff and Antoinette Cheung are full-time employees of Broadstreet HEOR. Yogesh Patel is a full-time employee of Alexion, AstraZeneca Rare Disease. Ioannis Tomazos and Alice Wang were full-time employees of Alexion, AstraZeneca Rare Disease at time of study. Jong Wook Lee has received grants and fees from Alexion, AstraZeneca Rare Disease, and has served as a member of an advisory board for Alexion, AstraZeneca Rare Disease."

"Funding Open Access funding enabled and organized by Projekt DEAL. Alexion, AstraZeneca Rare Disease."