- In the News
- Welcome to PDSA
FDA Issues Final Guidance Documents for Expanded Access Program
"The National Organization for Rare Disorders (NORD) applauds the Food and Drug Administration (FDA) for finalizing its efforts to streamline the process used by patients and their physicians seeking expanded access to investigational new drugs and biologics.
Their release of the final Individual Patient Expanded Access Investigational New Drug Application - Form FDA 3926 - can be used by physicians to request expanded access, often referred to as “compassionate use,” for patients suffering from serious or immediately life-threatening diseases and for whom no comparable or satisfactory alternative therapy is available. The shortened form reduces the time to completion from hours to only 45 minutes; a drastic improvement in alleviating the burden on patients and their physicians.
- From NORD Press Release.
Patients with chronic ITP may have increased infection risk before diagnosis
"Patients with primary chronic immune thrombocytopenia have an increased risk for infections up to 5 years before diagnosis, according to results of an epidemiological study.
Of the 3,121 patients diagnosed with ITP between 2006 and 2012, 1,087 patients were diagnosed with incident chronic ITP (men, n = 531; women, n = 556). The observed annual incidence of chronic ITP was 2.3 per 100,000 people (95% CI, 2.15-2.45)."
- By Healio.com. Ekstrand C, et al. J Thromb Haemost. 2016;doi:10.1111/jth.13267.
First U.S. Zika death reported in Puerto Rico
"A 70-year-old Puerto Rican man died from complications of the Zika virus in February, the Centers for Disease Control and Prevention and the Puerto Rico Department of Health announced Friday (4/29/16). This is the first time in the United States or territories that Zika infection contributed to a death, the CDC said.
The elderly man, who lived in the San Juan metro area, contracted Zika and was treated for symptoms that lasted less than a week, said Tyler Sharp, an epidemiologist at the Centers for Disease Control and Prevention Dengue Branch in San Juan, Puerto Rico.
'"A few days after his recovery, he returned to the hospital with signs of a bleeding disorder and was diagnosed with immune thrombocytopenic purpura, or ITP,' Sharp said."
- By Sandee LaMotte, CNN.
PDSA medical advisor Dr. Michael Tarantino published in The Lancet
The Bleeding & Clotting Disorders Institute (BCDI) is proud to announce that our Medical Director, Dr. Michael Tarantino, is the lead investigator and lead author on an international clinical trial that was published in The Lancet. The abstract describes a study of Nplate® showing reduced rate of bleeding in children affected by immune thrombocytopenic purpura (ITP).
- From BCDI Press Release
Voluntary Market Withdrawal – April 21, 2016: Octagam [Immune Globulin Intravenous (human)] 5% Liquid Preparation
DATE NOTIFICATION INITIATED: April 21, 2016
PRODUCT / LOT NUMBER / Expiration:
Octagam [Immune Globulin Intravenous (human)] 5% Liquid Preparation
Lot Number K551A8441
Expiration Date: December 17, 2017
Octapharma USA, Inc.
Effective immediately, Octapharma USA Inc. is initiating a voluntary market withdrawal of Octagam 5% [Immune Globulin Intravenous (human)] 5% Liquid Preparation] that is labeled with lot number K551A8441. Although there have been no reports of serious injury at this time, Octapharma has determined, through consultation with the public health authorities at FDA, the most prudent course of action is to suspend further administration of this Octagam from this production lot.
Customers that received this product from a distributor and not directly from Octapharma, should contact the distributor for return instructions.
Nplate® (Romiplostim) study in The Lancet shows significant increase in durable platelet response among children with Immune Thrombocytopenia
Nplate Reduced Rates of Bleeding by More Than 30 Percent in Children Affected by Blood Disorder
THOUSAND OAKS, Calif. (April 19, 2016) – Amgen (NASDAQ:AMGN) today announced that The Lancet published results from a Phase 3 randomized, double-blind, placebo-controlled study of Nplate® (romiplostim) in children with symptomatic immune thrombocytopenia (ITP). The study showed that 52 percent of Nplate patients achieved a durable platelet response, compared with 10 percent of placebo-treated patients (p=0.002, odds ratio 9.1, 95 percent CI: 1.9, 43.2).
- From Amgen press release
"Making the world of ITP a more manageable place to be"
The Platelet Disorder Support Association is dedicated to enhancing the lives of people with immune thrombocytopenia (ITP) and other platelet disorders through:
Education • Advocacy • Research • Support
Comprehensive information on conventional therapies usually prescribed for ITP as well as therapies outside of the usual medical mainstream that some patients with ITP have used successfully.
Advocacy helps build awareness and support for ITP and other platelet disorders. Everyone interested in having more people know about ITP, having greater access to treatment, and getting more funding ITP and related diseases can help.
ITP Conference DVDs, free booklets and pamphlets, diet and lifestyle information, PDSA newsletters, journal articles and abstracts, PDSA E-News.