PDSA E-News: August 25, 2016




Romiplostim (Nplate®) Gives Higher Platelet Response Rate in Children with Chronic ITP

NplateRomiplostim (aka Nplate®, Amgen) has been around for several years now and has helped many adults with ITP. Surprisingly, it hasn’t yet received the Good Housekeeping Seal of Approval (i.e., FDA approval) for treatment of pediatric immune thrombocytopenia. The reason: the necessary studies had not been completed. As Dr. Michael D. Tarantino, who is on PDSA’s board of medical advisors, explained it, “Children with chronic immune thrombocytopenia are at risk for serious bleeding events due to low platelet counts, which can be very frightening for these children and their parents.” Dr. Tarantino stated that romiplostim treatment induced a high rate of platelet response in children with ITP according to results of a recent phase 3 trial. Dr. James Bussel, who is also on PDSA’s board of medical advisors contributed to this study as well.

Annual ITP conference attendees have heard our medical board explain what it takes to have evidence that a drug works. The gold standard is a randomized, double-blind, placebo-controlled study. That is exactly what Dr. Tarantino, Dr. Bussel and their colleagues did. Their study was to determine the safety and efficacy of romiplostim for children with immune thrombocytopenia (ITP) persisting more than six months (i.e., chronic ITP). Study results suggested that romiplostim could reduce the frequency and severity of bleeding events for children suffering from symptomatic ITP, thus providing them with another treatment option.

The study included 62 children and young adults (age range of 1-17 years). Participants had to have a mean platelet count of 30 x 109 /L or less and no single count greater than 35 x 109 /L. They were randomly assigned to weekly romiplostim for 24 weeks (n=42, median age = 10 years) or to placebo (n=20, median age = 7.5 years). Endpoint of the study was a “durable platelet response.” This was defined as a weekly platelet count in at least six of the final eight weeks of the study of 50 x 109 /L or higher without rescue drug use in the four preceding weeks. A greater proportion (52%) of children who took romiplostim achieved a durable platelet response, while only 10% of the children who took a placebo achieved that goal. Also 85% vs. 55% showed higher rates of any platelet response with romiplostim vs. placebo. Only one treatment-related serious adverse event was reported in the romiplostim group, which was headache and thrombocytosis. No patients withdrew from the study because of serious adverse events. This completed study is registered at clinicaltrials.gov. See https://clinicaltrials.gov/show/NCT01444417.

Tarantino M, Bussel J, Blanchette V, et al. “Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study.” Lancet 2016 Jul 2; 388(10039):45-54. doi: 10.1016/S0140-6736(16)00279-8.


Low-level Laser Therapy May Improve Treatment of Thrombocytopenia

Many health conditions can lead to thrombocytopenia (low platelets) including certain kinds of leukemia, drug side-effects, and the autoimmune disorder, immune thrombocytopenia (ITP). Now a low-intensity type of laser treatment may offer a non-invasive and drug-free way to treat thrombocytopenia. According to researchers at Massachusetts General Hospital in Boston, low-level laser (LLL) therapy increased platelet production from precursor cells called megakaryocytes (MKs) and had the same effect in several different mouse models of thrombocytopenia. The low-level laser therapy enhanced platelet production in animals with low platelets but not in those with normal counts.

Low-level lasers (also called cold lasers) emit low-powered laser light that doesn’t heat the target tissue. It is known to protect the function of mitochondria (cellular structures that provide cells with energy). Several conditions associated with impaired platelet production have been found to have abnormalities in mitochondria in the bone marrow that produces platelets. The LLL treatments of MKs increased their size, accelerated formation of proplatelets, and doubled platelet production. Having the mice infused with LLL-treated MKs led to greater platelet production than did infusion of MKs treated with normal light.

Researchers said the current main obstacle to testing LLL in human patients with thrombocytopenia is the lack of a device large enough to treat the entire human body or enough bones to stimulate platelet production by MKs in the bone marrow. The team plans to address that issue. They state that the LLL treatment may not be effective when thrombocytopenia is caused by inborn genetic defects.

Massachusetts General Hospital. “Low –level laser therapy may improve treatment of dangerous bleeding disorder: Light-based therapy may treat thrombocytopenia without hazards of current therapies.” Science Daily. 27 July 2016.



CDC Recommends Use of Flu Shot over FluMist® Nasal Spray

fluWith flu season fast approaching and our heightened concern for our immune systems and overall health, we should be attentive of recent changes made by the Centers for Disease Control (CDC) concerning flu vaccinations. In 2003, FluMist® LAIV (Live Attenuated Influenza Vaccine), a nasal spray alternative to traditional intramuscular injections, was FDA-approved for the vaccination of three and four different strains of influenza in people ages 2 to 49. FluMist contains a live, weakened form of the virus, and prevents against further infection by stimulating the immune system to develop antibodies against the virus strains. In contrast, the standard influenza virus contains inactivated viruses but also tends to protect against three of the most common strains of viruses predicted by scientists for that coming year. FluMist was the preferred method of influenza vaccination for both kids and pediatricians, due to its ease of use.

Unfortunately, data analyzed by a CDC panel of 15 immunization experts has illustrated that FluMist isn’t nearly as effective as the standard flu vaccination in protecting against the pathogens. These findings were corroborated by the American Academy of Pediatrics and the Pediatric Infectious Disease Society. Dr. Chris Ambrose of MedImmune, the company that produces FluMist, reported that the quadrivalent (four-strain) vaccine was only 46% effective, compared with the shot’s 65% effectiveness rate. However, one scientist from the CDC presented evidence that FluMist was completely ineffective against one of the strains of influenza.

The CDC, FDA, and MedImmune plan to continue scientific collaboration and work to increase FluMist’s efficacy in the future. Considering the new recommendation against nasal vaccinations, coupled with last year’s flu vaccine shortage, the CDC will ensure there is enough vaccine supply for all who need influenza vaccinations this coming year.

Scutti, Susan. “CDC panel recommends against using FluMist vaccine.” CNN. 6/23/2016.



Life’s a Beach: Extending Your Vacation beyond the Sand and Sun

footprints in the sandAs summer draws to a close and we return from family vacations, how do we hold on to that feeling of rest and relaxation as long as possible? A new study published by The Journal of Occupational Health found that vacations have positive effects on our quality of life such as decreasing tiredness, depression, stress, risk of heart disease, and increasing work productivity. However, the study unfortunately also found that the lasting effects are brief once we return to work.

How do we manufacture the benefits of vacation when research has also found that time-off has been in a steady decline since 2000, and 41% of Americans didn’t take any time-off in the past year? What do we do when we find that we can’t pull ourselves away from work while on vacation, as was discovered was common in 61% of recruiting Web site, Glassdoor, users?

The answer is easier and less expensive than you would think: activate vacation mode from your office or home to reap the health benefits of a vacation. Similar to a beach vacation or trip to Europe, participate in activities that you enjoy, relax, and maintain a regular sleep schedule to maintain your post-vacation glow. Make time for holiday-inspired activities: cook dinner with friends and family, get a massage, read a book outside, or go for a run in nature; you’re sure to feel good and your body will thank you.

Steinhilber, Brianna. “Vacation Mode: How to Manufacture the Feeling of Rest and Relaxation.” EverUp. 6/30/2016.



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