Abstract
Summary
This is a plain language summary of an article published in the scientific journal The Lancet in 2023. The ADVANCE IV study involved people living with immune thrombocytopenia, called ITP. In primary ITP your immune system attacks platelets in the blood. People with ITP have a lower number of platelets than normal. Some people may bruise or bleed more easily, and bleeding can become serious if platelet counts are very low. ITP is called persistent when low platelet counts continue for 3 to 12 months and chronic if low platelet counts continue for more than a year from diagnosis.
Participants received efgartigimod or a placebo (inactive medicine) through a needle into the vein (intravenous) weekly. The main aim was to see how many people with chronic ITP had increased platelet counts over several weeks during the treatment period. Researchers looked at how well intravenous efgartigimod worked in improving symptoms such as bleeding in adults with chronic or persistent primary ITP. They also looked at the side effects people had during the study.
Overall, 131 people with ITP participated in the study between December 2019 and February 2022. On average, people had received their ITP diagnosis over 10 years before the start of the study. Around 7 in 10 people (67%) had previously received at least 3 other treatments for ITP. More people (22%) who received intravenous efgartigimod had increased platelet counts than those who received a placebo (5%). Over half of the people who received efgartigimod had improvements when taking platelet counts and bleeding into account. The most common side effects were tiny purple, red, or brown spots on the skin (petechiae), red blood cells in the urine, and headaches. These were seen at similar rates in people receiving efgartigimod and those receiving a placebo.
These results show that intravenous efgartigimod helps increase platelet counts in people with chronic or persistent primary ITP who had tried several previous treatments for ITP.
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Footnotes
Acknowledgements
The authors gratefully acknowledge the participants, caregivers, participant advocates, clinicians, and support staff who have collaborated on the design and execution of the ADVANCE IV trial. Writing support for this plain language summary of publication was provided by Jacqui Oliver, PhD, of Envision Ignite, an Envision Medical Communications agency, a part of Envision Pharma Group, and funded by argenx. The authors also acknowledge and appreciate the support of Shelley Gerson in identifying a patient author for this publication.
Ethics approval and consent to participate
As described in the original publication, the ADVANCE IV trial was conducted in accordance with the protocol and consensus ethical principles derived from international guidelines, including the Declaration of Helsinki, Council for International Organizations of Medical Sciences International Ethical Guidelines, applicable International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use Good Clinical Practice Guidelines, and other applicable laws and regulations. The protocol was approved by an institutional review board or independent ethics committees at each participating site.
Consent for publication
Not applicable.
Author contributions
Catherine Broome: conceptualization, formal analysis, investigation, writing – review and editing. Yoshitaka Miyakawa, Monica Carpenedo and Hanny Al-Samkari: investigation, writing – review and editing. Jaume Ayguasanosa: conceptualization, methodology, formal analysis, investigation, writing – review and editing. John Phillips: writing – review and editing. Francesco Rodeghiero: methodology, investigation, writing – review and editing.
Funding
This manuscript was funded by argenx. The funder was involved in the study design, data collection and analysis, decision to publish, and preparation of the manuscript.
Competing interests
Catherine Broome reports honoraria from Alexion, Apellis, argenx, and Sanofi; and advisory fees from Incyte and Novartis. Yoshitaka Miyakawa reports grant support from argenx, Alexion, Kissei, Sanofi, Chugai, Novartis, Novo Nordisk, Janssen, Recordati, UCB; consulting or advisory fees from argenx, Kyowa Kirin, Sanofi, UCB, and Zenyaku Kogyo; and honoraria from Alexion, Argenx, Chugai, Kissei, Kyowa Kirin, Novartis, Pfizer, and Sanofi. Monica Carpenedo reports consulting or advisory fees from Amgen, argenx, and Novartis; and honoraria from Sobi. Hanny Al-Samkari reports grant support from Agios, Amgen, Novartis, Sobi, and Vaderis; and consulting or advisory fees from Agios, argenx, Forma, Moderna, Novartis, Rigel, and Sobi. Jaume Ayguasanosa reports employment by argenx. John Phillips reports honoraria for advisory roles from argenx, Novartis, and Sobi, Inc. and speaker fees from Sanofi. Francesco Rodeghiero reports consulting or advisory fees from argenx.
Availability of data and materials
argenx is committed to responsible data sharing regarding the clinical trials it funds. Included in this commitment is access to anonymized individual-level and trial-level data (analysis data sets) and other information (eg, protocols and clinical study reports), as long as the trial is not part of an ongoing or planned regulatory submission. These clinical trial data can be requested by qualified researchers who engage in rigorous independent scientific research and will only be provided after review and approval of a research proposal and statistical analysis plan and execution of a data sharing agreement. Data requests can be submitted at any time, and the data will be accessible for 12 months. Requests can be submitted to esr@argenx.com