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American society of hematology: 55th annual meeting and exposition.

Year: 2014
Type: article
Abstract: Use of investigational GMI-1070 (GlycoMimetics, Gaithersburg, Maryland), a pan-selectin inhibitor, led to improvements in clinically meaningful outcomes in patients with sickle-cell disease recently hospitalized for a vaso-occlusive crisis (VOC), according to Dr. Telen. GMI-1070 led to faster resolution of VOC, shorter hospital stays, and reduced parenteral opioid analgesia. Sickle-cell disease, the most common genetic disease in the U.S., affects 90,000 to 100,000 individuals in the U.S. and millions worldwide, Dr. Telen said. The most common clinical manifestation of sickle-cell disease, painful vaso-occlusive crises, accounts for between 75,000 and 90,000 hospital admissions annually. Symptom relief with narcotics as supportive therapy is generally all that can be offered to sickle-cell patients for VOC. Dr. Telen and colleagues explored the safety and efficacy of GMI-1070 in a prospective, randomized, multicenter, double-blind, adaptive phase 2 study, subsequent to animal studies showing restoration of blood flow with GMI-1070 and early phase human studies showing no significant safety concerns. The phase 2 study enrolled 76 individuals (ages 12–51 years; 43 GMI-1070 and 33 placebo). The primary endpoint was time to resolution of VOC. The median time to resolution of VOC was reduced by 63 hours with GMI-1070 (P = 0.187), and the mean time to resolution was reduced by 41 hours (P = 0.192). “While these were not statistically significant, the differences are actually quite large,” Dr. Telen commented. Also, the mean hourly opioid analgesic administered during hospitalization was reduced (48-hour reduction, P = 0.067), and the cumulative opioid dose was reduced by 83% (P = 0.010) with GMI-1070. Trends favoring GMI-1070 were also observed for median time to discharge from hospital (reduced by 84 hours, P = 0.092) and for the mean time to discharge (reduced by 55 hours, P = 0.096). Dr. Telen noted that GMI-1070 had an apparently benign safety profile in this population. “These results provide strong rationale for a phase 3 trial for GMI-1070 for sickle cell patients with vaso-occlusive crisis.” GlycoMimetics is collaborating with Pfizer on the development of the phase 3 study.
Language: English
Field: Medicine
Open Access status: green
OpenAlex ID: W2898145047
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