Akshay Sharma, MBBS, on the Importance of Patient Reported Outcomes for Exa-cel


The bone marrow transplant physician at St. Jude Children’s Research Hospital discussed an analysis of the sickle cell disease gene therapy he’s presenting on at ASH’s 2023 meeting.

“Beyond measuring a reduction in painful crises, I think the most important thing that affects a patient is how the disease affects their life. That's an aspect that gets captured by asking the patients questions about their quality of life [and] how the disease has affected their day to day activities...”

On December 8, 2023, the FDA approved Vertex Pharmaceuticals' and CRISPR Therapeutics’ autologous gene-edited cell therapy exagamglogene autotemcel (exa-cel), marketed under the name Casgevy, for the treatment of severe sickle cell disease (SCD) in patients aged 12 years and older with recurrent vaso-occlusive crises.1 The agency’s decision was largely based on data from the phase 1/2/3 CLIMB-121 clinical trial (NCT03745287) and the phase 3 long-term follow-up study CLIMB-131 (NCT04208529), with reference being specifically made to the ability of therapy to bring about freedom from vaso-occlusive crises (VOCs), which formed the basis of the primary end point in CLIMB-121. Although the occurrence of VOCs has value as an objectively measurable clinical outcome, it is somewhat limited in its ability to give a more complete picture of the therapy’s impact on a patient’s life. As such, there is interest among those in the medical community in using patient reported outcomes to learn more about the therapy’s efficacy.

Akshay Sharma, MBBS, a bone marrow transplant physician at St. Jude Children’s Research Hospital, is giving a presentation entitled “Improvements in Health-Related Quality of Life after Exagamglogene Autotemcel in Patients with Severe Sickle Cell Disease” at the 2023 American Society of Hematology (ASH) Annual Meeting & Exposition, held December 9-12, in San Diego, California.2 The talk will cover patient reported outcomes data from patients who participated in CLIMB-121.

In an interview with CGTLive™, Sharma discussed the rationale behind this analysis and the key points of his presentation. He noted that the analysis included data from a number of different instruments used for patient reported outcomes and summarized the big picture finding that the data indicated an overall improvement in patients’ quality of life following treatment with exa-cel.

Click here for more coverage of ASH 2023.

1. FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. News release. FDA. December 7, 2023. Accessed December 7, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease 2. Taysha Gene Therapies reports third quarter 2023 financial results and provides corporate and clinical updates. News release. Taysha Gene Therapies, Inc.. November 14, 2023. Accessed December 7, 2023. https://ir.tayshagtx.com/news-releases/news-release-details/taysha-gene-therapies-reports-third-quarter-2023-financial
2. Sharma A, Frangoul H, Mapara M, et al. Improvements in health-related quality of life after exagamglogeneautotemcel in patients with severe sickle cell disease. Presented at: ASH 2023 Annual Meeting & Exposition. December 9-12; San Diego, CA. Abstract #4999
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