Akshay Sharma, MBBS, on Ongoing Challenges of Using Patient Reported Outcomes in Sickle Cell Disease


The bone marrow transplant physician at St. Jude Children’s Research Hospital also discussed the recent approval of exa-cel.

This is the second part of an interview with Akshay Sharma, MBBS. For the first part, click here.

“...We need to harmonize and standardize how we collect this data across different clinical trials, and across different treatment modalities, so that eventually we can compare the outcomes data that has been obtained from different clinical trials from different therapies and then actually be able to make sound decisions about whether one treatment is really superior to or equivalent to another treatment.”

Patient reported outcomes are an emerging outcomes measure of interest for clinical trials, especially in the field of hematology. For example, although an objectively measurable clinical end point, freedom of vaso-occlusive crises, served as a primary end point in the phase 1/2/3 CLIMB-121 clinical trial (NCT03745287) for Vertex Pharmaceuticals' and CRISPR Therapeutics’ sickle cell disease gene-edited cell therapy exagamglogene autotemcel (exa-cel, marketed under the name Casgevy), patient reported outcomes were measured alongside this end point in order to get a better understanding of the impact of the therapy on patients’ day-to-day lives. Akshay Sharma, MBBS, a bone marrow transplant physician at St. Jude Children’s Research Hospital, is giving a presentation on the findings from this patient reported outcomes data at the 2023 American Society of Hematology (ASH) Annual Meeting & Exposition, held December 9-12, in San Diego, California.

CGTLive™ sat down with Sharma in order to learn more about the state of patient reported outcomes data in hematology. Following a discussion on the importance of this emerging outcome measure, Sharma spoke on the challenges of implementing these outcome measures. He noted that because patient recorded outcomes have become more popular additions to clinical trials only recently, similar data from earlier trials for treatments that have become established standards of care, like bone marrow transplantation, may not be available. This makes comparisons between newer treatments like exa-cel and older treatments more difficult. Another issue Sharma mentioned is the large number of different instruments for patient reported outcomes that exist and the difficulty of comparing results from these instruments to one another.

Following this, Sharma also gave his thoughts on the approval of exa-cel by the FDA, which had occurred several hours prior to the interview on December 8, 2023. He emphasized that in order for the therapy to be effective for patients in the real-world, it needs to be affordable.

Click here for more coverage of ASH 2023.

1. Sharma A, Frangoul H, Mapara M, et al. Improvements in health-related quality of life after exagamglogene autotemcel in patients with severe sickle cell disease. Presented at: ASH 2023 Annual Meeting & Exposition. December 9-12; San Diego, CA. Abstract #4999
2. 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
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