Thursday, March 31, 2016

ACTH is more effective than other standard therapies for infantile spasms

Knupp KG, Coryell J, Nickels KC, Ryan N, Leister E, Loddenkemper T, Grinspan
Z, Hartman AL, Kossoff EH, Gaillard WD, Mytinger JR, Joshi S, Shellhaas RA,
Sullivan J, Dlugos D, Hamikawa L, Berg AT, Millichap J, Nordli DR Jr, Wirrell E;
Pediatric Epilepsy Research Consortium. Response to treatment in a prospective
national infantile spasms cohort. Ann Neurol. 2016 Mar;79(3):475-84.

Infantile spasms are seizures associated with a severe epileptic encephalopathy presenting in the first 2 years of life, and optimal treatment continues to be debated. This study evaluates early and sustained response to initial treatments and addresses both clinical remission and electrographic resolution of hypsarrhythmia. Secondarily, it assesses whether response to treatment differs by etiology or developmental status.
The National Infantile Spasms Consortium established a multicenter, prospective database enrolling infants with new diagnosis of infantile spasms. Children were considered responders if there was clinical remission and resolution of hypsarrhythmia that was sustained at 3 months after first treatment initiation. Standard treatments of adrenocorticotropic hormone (ACTH), oral corticosteroids, and vigabatrin were considered individually, and all other nonstandard therapies were analyzed collectively. Developmental status and etiology were assessed. We compared response rates by treatment group using chi-square tests and multivariate logistic regression models.
Two hundred thirty infants were enrolled from 22 centers. Overall, 46% of children receiving standard therapy responded, compared to only 9% who responded to nonstandard therapy (p < 0.001). Fifty-five percent of infants receiving ACTH as initial treatment responded, compared to 39% for oral corticosteroids, 36% for vigabatrin, and 9% for other (p < 0.001). Neither etiology nor development significantly modified the response pattern by treatment group.
Response rate varies by treatment choice. Standard therapies should be considered as initial treatment for infantile spasms, including those with impaired development or known structural or genetic/metabolic etiology. ACTH appeared to be more effective than other standard therapies.

 It's a happy day at Questcor!
Courtesy of a colleague.

1 comment:

  1. At the heart of Valeant's woes is whether or not its business model can be justified by lawmakers. Acquiring and merging is a perfectly acceptable practice in the healthcare sector and other industries. What's being called into question is whether or not drugmakers have the ability/right to drastically increase the prices of acquired drugs without changing the formulations or manufacturing processes. In other words, the value of branded medication is currently on trial, whether you realize it or not.

    Valeant is certainly not the only company to find itself in the spotlight for its drug-pricing practices. Mallinckrodt(NYSE:MNK) relies heavily on Acthar Gel, an anti-inflammatory medicine acquired when it purchased Questcor Pharmaceuticals for $5.6 billion in 2014. Now priced at about $35,000 per vial, Acthar Gel's price has risen by more than 2,000% since 2005. But in Mallinckrodt's defense, Questcor was responsible for the majority of those price hikes.