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Journal Articles

Efficacy, safety, and pharmacokinetics of alpelisib (ALP) in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS): Epik-P4, a Phase II study

Alexandra J Borst, Denise M Adams, Juan C Lopez-Gutierrez, Friedrich Kapp, Ines Vendrell, Fotis Polydoros, Elise Burmeister-Getz, Nydia Caro, Guillaume Canaud

Blood (2025) 146 (Supplement 1): 8227.

https://doi.org/10.1182/blood-2025-8227

Published: 2025

Journal Articles

Epik-L1: A Phase 2/3 study of alpelisib in pediatric and adult patients with PIK3CA-driven lymphatic malformations

Bryan Sisk, Guillaume Canaud, Juan C Lopez-Gutierrez, Friedrich Kapp, Mario Stegert, Qinxia Wang, Elise Burmeister-Getz, Nydia Caro, Denise M Adams

Blood (2025) 146 (Supplement 1): 4893.

https://doi.org/10.1182/blood-2025-4893

Published: 2025

Journal Articles

Eltrombopag in Pediatric Patients with Previously Untreated or Refractory/Relapsed Severe Aplastic Anemia: The Phase II Escalate Trial

Akiko Shimamura, Alexey Maschan, Carolyn Bennett, Jason E Farrar, Sujith Samarasinghe, Brigitte Strahm, Winfred C. Wang, Adrianna Vlachos, Charlotte M. Niemeyer, Timothy S. Olson, Denise D'Alessio, Elise Burmeister Getz, Tomasz Lawniczek, Yunnan Xu, David A. Williams

Blood (2022) 140 (Supplement 1): 705–707.

https://doi.org/10.1182/blood-2022-158214

Published: 2022