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Poloxamer 188 for Sickle Cell Disease

Poloxamer 188 for Sickle Cell Disease Research Editor's Note Poloxamer 188 for Sickle Cell Disease Editor's Note Jody Zylke, MD Although effective treatments exist to prevent painful The trial by Casella et al was completed in 2016, 5 years vaso-occlusive episodes in patients with sickle cell dis- ago, and is much older than the randomized clinical trials that ease (ie, hydroxyurea), finding therapies to reduce the se- JAMA usually publishes. Why did the editors decide to ac- verity or duration of episodes once they have begun has proved cept this report? Two factors influenced the decision. elusive. Inhaled nitric oxide, The first is that JAMA published the trial by Orringer intravenous magnesium, and et al, and the editors thought it was important for these con- Related article page 1513 intravenous sevuparin have tradictory results to be published. The second relates to the been tried without success. In 2001, JAMA published a reason for the delay after trial completion. The neutral results phase 3 multicenter, randomized, placebo-controlled clinical of the study were announced in 2016 when the trial was com- trial of poloxamer 188 in 255 children and adults with sickle pleted. The company that funded the trial, Mast Therapeu- cell disease hospitalized with a vaso-occlusive http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png JAMA American Medical Association

Poloxamer 188 for Sickle Cell Disease

JAMA , Volume 325 (15) – Apr 20, 2021

Poloxamer 188 for Sickle Cell Disease

Abstract

Research Editor's Note Poloxamer 188 for Sickle Cell Disease Editor's Note Jody Zylke, MD Although effective treatments exist to prevent painful The trial by Casella et al was completed in 2016, 5 years vaso-occlusive episodes in patients with sickle cell dis- ago, and is much older than the randomized clinical trials that ease (ie, hydroxyurea), finding therapies to reduce the se- JAMA usually publishes. Why did the editors decide to ac- verity or duration of episodes once they...
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Publisher
American Medical Association
Copyright
Copyright 2021 American Medical Association. All Rights Reserved.
ISSN
0098-7484
eISSN
1538-3598
DOI
10.1001/jama.2021.3399
Publisher site
See Article on Publisher Site

Abstract

Research Editor's Note Poloxamer 188 for Sickle Cell Disease Editor's Note Jody Zylke, MD Although effective treatments exist to prevent painful The trial by Casella et al was completed in 2016, 5 years vaso-occlusive episodes in patients with sickle cell dis- ago, and is much older than the randomized clinical trials that ease (ie, hydroxyurea), finding therapies to reduce the se- JAMA usually publishes. Why did the editors decide to ac- verity or duration of episodes once they have begun has proved cept this report? Two factors influenced the decision. elusive. Inhaled nitric oxide, The first is that JAMA published the trial by Orringer intravenous magnesium, and et al, and the editors thought it was important for these con- Related article page 1513 intravenous sevuparin have tradictory results to be published. The second relates to the been tried without success. In 2001, JAMA published a reason for the delay after trial completion. The neutral results phase 3 multicenter, randomized, placebo-controlled clinical of the study were announced in 2016 when the trial was com- trial of poloxamer 188 in 255 children and adults with sickle pleted. The company that funded the trial, Mast Therapeu- cell disease hospitalized with a vaso-occlusive

Journal

JAMAAmerican Medical Association

Published: Apr 20, 2021

References