Monoclonal Antibody Crizanlizumab Reduces Sickle Cell Pain Crisis Interview with:

Kenneth I. Ataga, MD Division of Hematology/Oncology University of North Carolina at Chapel Hill Chapel Hill, NC

Dr. Kenneth I. Ataga

Kenneth I. Ataga, MD
Division of Hematology/Oncology
University of North Carolina at Chapel Hill
Chapel Hill, NC What is the background for this study? What are the main findings?
Response: The available treatments for acute painful episodes (also referred to as vaso-occlusive crises), the most common complication of sickle cell disease, are limited.

Findings from the Phase II SUSTAIN study showed that crizanlizumab (formerly SelG1) at 5 mg/kg reduced the median rate of sickle cell disease-related pain crises per year by 45.3% vs. placebo in patients with or without concomitant hydroxyurea therapy. In addition, clinically meaningful reductions in the frequency of painful crises were observed regardless of sickle cell disease genotype. What should readers take away from your report?

Response: Crizanlizumab significantly reduces the frequency of sickle cell-related pain crises and represents the first potentially novel disease-modifying therapeutic option for patients since hydroxyurea was approved for use in sickle cell anemia almost 20 years ago. What recommendations do you have for future research as a result of this study?

Response: Based on the results of this study, Novartis plans to continue development of crizanlizumab and consult with relevant health authorities. Is there anything else you would like to add?

Response: The results of the SUSTAIN study are very exciting because patients with sickle cell disease could potentially benefit from the availability of a new therapy for the prevention of vaso-occlusive crises. Thank you for your contribution to the community.

Citation: Abstract presented at the December 2016 ASH conference
 SUSTAIN: A Multicenter, Randomized, Placebo-Controlled, Double-Blind, 12-Month Study to Assess Safety and Efficacy of SelG1 with or without Hydroxyurea Therapy in Sickle Cell Disease Patients with Sickle Cell-Related Pain Crises
Session: Plenary Scientific Session

Sunday, December 4, 2016, 2:00 PM-4:00 PM

Kenneth I. Ataga, MD1, Abdullah Kutlar, MD2, Julie Kanter, MD3, Darla Liles, MD4, Rodolfo Cancado, M.D., Ph.D.5, João Friedrisch, MD, PhD6*, Troy H. Guthrie, MD7*, Jennifer Knight-Madden, MBBS, PhD8*, Ofelia A. Alvarez, MD9, Victor R. Gordeuk, MD10, Sandra Gualandro, MD, PhD11*, Marina Pereira Collela, MD, PhD12, Wally R. Smith, MD13, Scott A. Rollins, PhD14*, Jonathan W. Stocker, PhD14* and Russell P. Rother, PhD14

Note: Content is Not intended as medical advice. Please consult your health care provider regarding your specific medical condition and questions.

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Last Updated on December 6, 2016 by Marie Benz MD FAAD