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ITP & PLATELET DISORDERS RESEARCH & TREATMENTS


Infusion of DMSO Cryopreserved Platelets (CPP) Found Safe and Effective for Increasing Platelet Counts in Patients with Low Counts

Platelets drawingBlood and plasma donations save lives every day. They can be stored anywhere from one month to one year; platelets, however, only last five days. One limitation of platelet transfusions, especially in patients with low platelet numbers, is that some thrombocytopenic patients do not respond to platelet transfusion. When platelets are cryopreserved (frozen) with 6% dimethyl sulfoxide (DMSO), however, their shelf life can extend up to two years.

An initial study demonstrated that when these frozen platelets were added to healthy volunteers, their platelets lasted significantly longer in circulation. In a laboratory setting, these platelets displayed higher levels of molecule secretion that assist in clotting and stopping the flow of blood; greater expression of phosphatidylserine, a chemical that regulates coagulation; and poorer responses to platelet activation, in comparison with room temperature stored platelets. These findings imply that frozen platelets promote accelerated and enhanced clotting in the body.

Researchers enrolled hospitalized thrombocytopenic patients with bleeding symptoms to evaluate the effectiveness of this treatment. Of 59 patients, half were transfused with varying amounts of frozen platelets. Bleeding events did not occur in the six days after transfusion. Five serious adverse events were reported but were not associated with frozen platelet transfusion. None of the participants experienced excessive blood clotting following transfusion. Significant increases in platelet counts occurred with the frozen platelets; greater amounts of platelet units transfused yielded greater increases in sustained platelet counts. All participants improved or stabilized their platelet count and bleeding symptoms. This experiment demonstrated that using cryopreserved platelets (CPP) during transfusion can result in a safe and effective elevation of platelet counts in thrombocytopenic patients.

Slichter SJ, Dumont LJ, et al. “Treatment of Bleeding in Severely Thrombocytopenic Patients with Transfusion of Dimethyl Sulfoxide (DMSO) Cryopreserved Platelets (CPP) Is Safe - Report of a Phase 1 Dose Escalation Safety Trial.” ASH, December 3-6, 2016; San Diego, CA. Abstract 1030.
http://bit.ly/2kMmO6j


FDA Approves New, Faster IVIg, Gammaplex® 10%, for Treatment of Primary Immunodeficiency and Chronic Immune Thrombocytopenia

Gammaplex 10%

There’s a new, faster treatment option for adult ITP patients. The U.S. Food and Drug Administration (FDA) has approved Gammaplex® 10% for treatment of primary immunodeficiency (PI) and for chronic immune thrombocytopenia (ITP) in adults. Gammaplex® 10% is an immune globulin intravenous (IVIg) [human] 10% liquid manufactured by Bio Products Laboratory Limited (BPL) of the UK. Gammaplex® 10% is made with the same process as BPL’s previously approved IVIg treatment, Gammaplex® 5%; however, Gammaplex® 10% is more concentrated than Gammaplex® 5% with an immune globulin G (IgG) concentration of 100 g/L and is stabilized with glycine (the smallest amino acid).

FDA based its approval on a bioequivalence study that compared Gammaplex® 10% and Gammaplex® 5% in 33 adult patients with Primary Immunodeficiency (PI). No notable differences were seen in safety and tolerability between the two products. Average infusion time for the Gammaplex® 10% in adult patients at 1 hour and 51 minutes, was 57% faster than for Gammaplex® 5%.

The most common adverse reactions reported in adult trial subjects receiving Gammaplex® 10% were headaches (12.5%), migraine (6.3%), and pyrexia (fever) (6.3%). The safety of Gammaplex® 10% has not yet been established in ITP patients. However, the safety profile for Gammaplex® 5% has been studied in ITP patients and the company anticipates that the safety profile for both IVIg formulations are comparable for ITP patients. Gammaplex® 10% should not be used in patients with a history of anaphylactic or severe reactions to IVIg, or by IgA deficient patients with antibodies to IgA.

“FDA Approves Bio Products Laboratory's Gammaplex® 10% for Treatment of Primary Immunodeficiency and Chronic Immune Thrombocytopenic Purpura.” PR Newswire, Feb. 7, 2017.
http://prn.to/2lPH0aM


HOSPITALS, INSURANCE & HEALTH CARE


Study Finds Clinicians Underestimate Harms and Overestimate Benefits of Medical Treatments, Screenings and Tests

Clinician, doctor, test tubeClinicians are health care professionals (such as physicians, psychologists, or nurses) who work as primary care givers of patients in hospitals, skilled nursing facilities, clinics, or patients’ homes. They diagnose and treat patients and follow evidence-based best practices and professional codes of conduct. With all these skills, knowledge, and best practices you might think that when their judgement is used to help decide upon the appropriate treatment, screening, or tests that it would be excellent.

In this retrospective review of 48 studies researchers calculated the percentage of clinicians who underestimated, overestimated, or answered correctly about the benefits or harms in question. The researchers discovered that clinicians are likely to underestimate harms and overestimate benefits of tests and treatments. The review showed that the majority of clinicians correctly estimated harms only 13% of the time, and benefits only 11% of the time. This is important because inaccurate clinician expectations of the benefits and harms of interventions can influence decision making and might contribute to increasing intervention overuse.

As to reasons for this misperception of harms and benefits, the researchers speculated that clinicians may focus more on the mechanisms of tests and treatments than they do on evidence for their effectiveness. In addition, clinicians’ own biases may include enthusiasm for any treatment over none, or a desire for reassurance.

Hoffmann TC and Del Mar C. “Clinicians’ Expectations of the Benefits and Harms of Treatments, Screening, and Tests: A Systematic Review.” JAMA Internal Medicine, 2017 Jan 9
https://www.ncbi.nlm.nih.gov/pubmed/28097303


GENERAL HEALTH & MEDICINE


Changing Multiple Habits Yields Better Improvements than a Single Health Resolution

Yoga poseNew year, new you? Beginning every year, many make health resolutions to become more fit, eat better, or be more mindful. It’s hard enough to stay dedicated to one of these goals, but what if you could overhaul your health by changing all your bad habits instead of making just a single small change?

Researchers at the University of California, Santa Barbara, looked at common health habits of college students. Students were separated into two groups and both received physical, cognitive, and emotional tests. One group continued their daily routines, the second group completely changed their health and well-being practices; they exercised twice a day, participated in mindfulness and stress reduction training, attended lectures on nutrition and sleep, and logged their exercise, diets, sleep, and mood.

After six weeks of health changes, both groups retook the original tests. The first group, unsurprisingly, did not change their health habits or show any testing changes from baseline. Students in the second group, according to the researchers, were “substantially stronger, fitter and more flexible. They performed much better on tests of thinking, focus and working memory. They also reported feeling happier and calmer; their self-esteem was much higher. Their brain scans showed a pattern of activity believed to indicate a greatly enhanced ability to stay focused.”

These extensive changes of this study differ significantly from past experiments when participants were asked to change only one habit. Although the researchers admit to the potential of a variety of confounding variables influencing the study, they hypothesize that one change might strengthen the effects of another.

Reynolds, Gretchen. “Out With the Old.” The New York Times. 5/12/2016.
http://nyti.ms/2lLZaGw

 

 

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