Nowak, Richard J.
Habib, Ali A.
Klink, Andrew J.
Muppidi, Srikanth
Parthan, Anju
Sader, S. Chloe
Balanean, Alexandrina
Gajra, Ajeet
Howard, James F. Jr https://orcid.org/0000-0002-7136-8617
,
Funding for this research was provided by:
Alexion, AstraZeneca Rare Disease
Article History
Accepted: 23 September 2024
First Online: 29 October 2024
Declarations
:
: Research funding for this study and funding for medical writing assistance and open-access fees were provided by Alexion, AstraZeneca Rare Disease.
: Richard J. Nowak has received research support from Alexion, AstraZeneca Rare Disease, argenx, Genentech, Grifols, Immunovant, Inc., Momenta Pharmaceuticals, the Myasthenia Gravis Foundation of America, the National Institutes of Health (National Institute of Neurological Disorders and Stroke and National Institute of Allergy and Infectious Diseases), Ra Pharmaceuticals (now UCB), and Viela Bio Inc. (now Horizon Therapeutics); and consultancy fees from Alexion, AstraZeneca Rare Disease, argenx, Cour Pharmaceuticals, Grifols, Immunovant, Inc., Momenta Pharmaceuticals, Ra Pharmaceuticals (now UCB), Roivant Sciences, and Viela Bio (now Horizon Therapeutics). Ali A. Habib has served as a medical advisor and speaker on behalf of Alexion and AstraZeneca Rare Disease; he has received research support from Alexion, AstraZeneca Rare Disease, and Cabaletta Bio; and has received research support and honoraria from argenx, UCB, Pfizer, Genentech, Viela Bio, and Regeneron. Andrew J. Klink is a salaried employee of and owns stock in Cardinal Health, which received funding from Alexion, AstraZeneca Rare Disease for work performed on this study. Srikanth Muppidi has served on advisory board meetings for Alexion, AstraZeneca Rare Disease, argenx, Horizon Therapeutics, and Ra Pharmaceuticals (now UCB). Anju Parthan was an employee of Alexion, AstraZeneca Rare Disease, and owned stock in AstraZeneca at the time the study was conducted and analyzed. S. Chloe Sader is an employee of Alexion, AstraZeneca Rare Disease and owns stock in AstraZeneca. Alexandrina Balanean is a salaried employee of Cardinal Health, which received funding from Alexion, AstraZeneca Rare Disease for work performed on this study. Ajeet Gajra was a salaried employee of and owned stock in Cardinal Health, which received funding from Alexion, AstraZeneca Rare Disease for work performed on this study, at the time the study was conducted. James F. Howard Jr has received research support (paid to institution) from Ad Scientiam, Alexion, AstraZeneca Rare Disease, argenx, Cartesian Therapeutics, the Centers for Disease Control and Prevention (Atlanta, GA, USA), the Myasthenia Gravis Foundation of America, the Muscular Dystrophy Association, the National Institutes of Health (including the National Institute of Neurological Disorders and Stroke and the National Institute of Arthritis and Musculoskeletal and Skin Diseases), PCORI, and UCB Pharma; honoraria from Alexion AstraZeneca Rare Disease, Amgen, argenx, Biohaven Ltd, Biologix Pharma, CheckRare CME, F. Hoffman-LaRoche, Medscape CME, Merck EMD Serono, NMD Pharma, Novartis Pharma, PeerView CME, Physicians’ Education Resource CME, PlatformQ CME, Regeneron Pharmaceuticals, Sanofi US, UCB Pharma, and Zai Lab; and non-financial support from Alexion, AstraZeneca Rare Disease, argenx, Biohaven Ltd, Toleranzia AB, UCB Pharma, and Zai Lab.
: The study was reviewed by the Western Institutional Review Board and determined exempt from requiring ethical approval on the basis that the study collected only secondary data, which were limited and comprised only necessary protected health information (e.g., treatment dates, date of death), and that all data were de-identified and aggregated.
: As this study involves anonymized structured data, which according to applicable legal requirements do not contain data subject to privacy laws, obtaining informed consent from patients was not required.
: Not applicable.
: Alexion, AstraZeneca Rare Disease will consider requests for disclosure of clinical study participant-level data provided that participant privacy is assured through methods such as data de-identification, pseudonymization, or anonymization (as required by applicable law), and if such disclosure was included in the relevant study an informed consent form or similar documentation. Qualified academic investigators may request participant-level clinical data and supporting documents (statistical analysis plan and protocol) pertaining to Alexion-sponsored studies. Further details regarding data availability and instructions for requesting information are available in the Alexion Clinical Trials Disclosure and Transparency Policy at .
: Not applicable.
: Study concept: RJN, AJK, SM, AP, SCS, AG. Study design: RJN, AJK, SM, AP, SCS, AB, AG, JFH Jr. Data acquisition: AAH, JFH Jr. Data analysis: RJN, AJK, AP, SCS, AB, AG. Data interpretation: all authors. Drafting/revision of the article for content: all authors. All authors read and approved the final version.