Pediatric Year in Review: New Therapies in Sickle Cell Disease, with Shaina Willen, MD

In this interview at the American Thoracic Society (ATS) 2025 International Conference, HCPLive spoke with Shaina Willen, MD, a board-certified provider in Pediatric Hematology/Oncology as well as Pediatric Pulmonology from UC Davis Health, about her talk at ATS titled ‘Pediatric Year in Review.’

In the talk, Willen described a variety of new therapies for sickle cell disease and their relevance to the conference itself, highlighting the recent explosion in options for patients with this condition. Willen was first asked what prompted the discussion in the first place.

“Historically, since 1998 for adults and to 2017 for children, we've only had one therapy for sickle cell disease,” Willen explained. “So the only disease-modifying therapy drug related is hydroxyurea, which works very well and is a wonderful therapy. But having one option in such a prevalent and difficult-to-manage disease has been very tricky, particularly for children. That's been the only medication since 2017 that's FDA-approved to treat complications of sickle cell disease. Then, first in 2017, 2019, and now, with the advent of gene therapy in 2023, we have more options.”

Willen was asked about some of the therapies for sickle cell that she highlighted within her presentation at ATS.

“Since hydroxyurea, we now have Endari, or L-glutamine, which we talked a little bit about in the talk,” Willen said. “We have crizanlizumab, which is for teenagers aged 16 and up. And then we had voxelotor, unfortunately, that was pulled from the market in December or September of 2024. Now with the 2 gene therapy options, disease-modifying therapies, there are a lot more choices than there used to be, particularly for children. So we were just wanting to highlight those and the trials and tribulations with those new therapies, but it is exciting that now we have options that we didn't have before.”

To find out more from Willen’s presentation at ATS, view the full interview segment posted above. For more from ATS, including late-breaking data, view our latest conference coverage.

The quotes contained in the above summary were edited for clarity. Editor’s note: Willen reported no relevant disclosures.