PDSA E-News: August 29, 2017




Researchers Confirm Alternate Clearance Site for Platelets in ITP

Blood under a microscopeThanks to a grant from the National Institutes of Health, investigators from the Oklahoma Medical Research Foundation have found new clues about the origins of platelet destruction in patients with persistent ITP. Scientists and physicians have long thought that the body attacked platelets in the spleen due to the presence of anti-platelet antibodies, as splenectomy and immunosuppressive therapies commonly mitigate platelet destruction and elevate platelet counts in about 70% of patients. However, these therapies don’t work in all ITP patients.

After following platelets deficient in “o-glycans,” sugars that the body produces, researchers noticed that these platelets had a shorter lifespan and were actually cleared in the liver instead of the spleen, resulting in a significant drop in platelet counts. This finding reveals the possibility of better targeted pathophysiological treatments in patients who don’t typically respond to traditional ITP therapies.

OMRF. “Oklahoma researchers zero in on cause of blood disorder.” Oklahoma Medical Research Foundation. 7/26/17. http://bit.ly/2vEoC8C


Alleviating Fatigue in ITP

Woman sleeping under a treeFeeling tired? You are not alone. A significant number of children and adults suffer from fatigue along with their ITP. In addition to platelet count, physicians can assess the burden of fatigue on their patients and help with strategies to reduce the problem. In ITP patients, fatigue is thought to be caused as a side-effect of the immune system’s inflammatory response and could be related to mood disorder and sleep disturbances.

A study in the British Journal of Haematology determined that a screening tool to identify significant fatigue could be utilized to address components of health related quality of life such as “sleep disturbance, anemia or iron deficiency, mood disorder or psychosocial issues, co-morbid disorders and medication side effects.” Identifying and treating these symptoms early would mitigate the impact of fatigue in chronic illness patients, either by use of pharmacological agents or through behavioral changes such as stress reduction, exercise, education, yoga, sleep therapies, and counselling. Researchers are currently testing the use of melatonin, serotonin, dopamine, and thyroptropin releasing hormone in other chronic diseases that present with fatigue symptoms, with mixed results. Encourage your physician to address your fatigue and other quality of life symptoms including lifestyle, bleeding symptoms, and platelet count when discussing potential therapy regimens.

Hill, Q. A. and Newland, A. C. “Fatigue in immune thrombocytopenia.” Br J Haematol, 170: 141–149. 2015. http://bit.ly/2w2UN2C


Protalex Awarded FDA Grant to Further Develop ITP Treatment

FDA Entrance SignThe U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has awarded Protalex, Inc., a clinical-stage biopharmaceutical company, a $403,000 grant to support future clinical development activity of PRTX-100 as a treatment for ITP. The grant program was specifically established to encourage the development of new rare disease therapies. Upon hearing the news, Richard J. Francovitch, Ph.D., Protalex’s vice president, ITP programs said, “We are delighted to have been granted this funding from the FDA’s OOPD as it underscores the great need for innovative, effective treatments for this rare autoimmune disease, and recognizes the potential benefits that PRTX-100 may provide for patients with ITP.”

PRTX-100, an immunomodulatory drug, is in the early testing phase and results are encouraging. One of three patients demonstrated a sustained platelet response to treatment in the early phases of the trial, which examines the best dose, efficacy, and product response. The study is open for additional enrollment and can enroll up to 36 patients in six cohorts. Patients are administered four weekly intravenous doses of the drug and will be monitored for the remainder of the year. For more information, see PDSA’s Clinical Trials page.

Fields, Anne Marie. “Protalex Awarded Grant From FDA Office of Orphan Products Development to Foster Clinical Development of PRTX-100 for the Treatment of Immune Thrombocytopenia.” BusinessWire. 8/17/17. http://bit.ly/2vH3pLh

Fields, Anne Marie. “Protalex Completes Data Analysis of Second Dose Cohort of US Phase ½ Study of PRTX-100 in ITP.” BusinessWire. 8/1/17. http://bit.ly/2wQzfUg



Don’t Worry, Be Happy

Choosing good over bad“Even when faced with an incurable illness, positive feelings and thoughts can greatly improve one’s quality of life,” says Dr. Wendy Schlessel Harpham. This sentiment was echoed by Dr. Peter Powers, the keynote speaker at the PDSA ITP conference, other speakers, and many of the attendees. Affirmations and gratitude are key.

In a study completed at Northwestern University Feinberg School of Medicine, professor Judith Moskowitz developed eight skills to help cultivate positive emotions for those coping with illness. Skills included “recognizing a positive event each day, telling a friend about the event, starting a daily gratitude journal, listing a personal strength and noting how you used it, setting an attainable goal and noting your progress, reporting a minor stress and listing ways to reappraise the event positively, recognizing and practicing small acts of kindness daily, and practicing mindfulness, focusing on the present.”

The skills were proven to have a tangible positive impact: researchers at the University of California followed 160 HIV patients and encouraged them to practice these affirmations. Patients were found to have a lower amount of virus, were more likely to adhere to therapy regimens, and were less likely to need antidepressants.

These results were replicated in subsequent studies for patients with diabetes, advanced breast cancer, and dementia. For those with chronic illness, practicing optimism, gratitude, and mindfulness improves quality of life, increases adherence to medication, cultivates healthy behaviors, and strengthens social connections and attentiveness to positivity.

Brody, Jane. “A Positive Outlook May Be Good for your Health” The New York Times. 3/27/17. http://nyti.ms/2o9yP71


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