PDSA E-News: May 23, 2016




PRTX-100, a Potential New Treatment for ITP, Gets Continuing Positive Reviews

Protalex logoGood news for adults with persistent/chronic ITP. Protalex, a biopharmaceutical company, announced in May that its independent safety monitoring committee is continuing enrollment and increasing the dose for patients in its “203 trial” in the European Phase 1b study of PRTX-100. They are currently enrolling new ITP patients at several medical centers in France to participate in this escalated dose trial. There is a parallel “202 trial” in the United States. It is common practice to try dose escalation in a phase 1b study so that doctors can decide the optimal dosage for new drugs.

PRTX-100 is known to modify aspects of the human immune system. Since ITP is an autoimmune disease it’s not surprising such a drug might be useful. PRTX-100 has the ability, at very low concentrations, to bind to human B-lymphocytes and macrophages and modulate immune processes. PRTX-100 may have the potential to treat ITP by reducing the immune-mediated destruction of platelets. Contrast this with the two most recently approved drugs to treat ITP, Nplate® (romiplostin) and Promacta®/Revolade™ (eltrombopag). They both increase production of platelets but do not appear to affect the underlying platelet destruction process.

The safety, tolerability and pharmacokinetics of PRTX-100 have been characterized in five clinical studies. It received Orphan Drug Designation in the U.S. and Europe for the treatment of ITP. In two Phase 1b clinical trials in adult patients with active Rheumatoid Arthritis (RA), PRTX-100 was generally safe and well tolerated at all dose levels. An open label dose escalation study of PRTX-100 in Adults with Chronic ITP is on-going in the U.S. They are still recruiting for this clinical trial. For details go to http://clinicaltrials.gov Type in study identifier NCT02401061.

“Protalex Receives Positive Interim Report from Independent Safety Monitoring Committee in European Phase 1b Study of PRTX-100 to Treat Immune Thrombocytopenia.” Press release, Protalex, Inc., May 2, 2016.


Blood Article Describes Treatment of Refractory ITP in Children and Adults

Feet and arrows going multiple directionsIn the March issue of Blood, the premiere journal for experimental and clinical hematology, Dr. Adam Cuker (University of Pennsylvania) and Dr. Cindy E. Neunert (Augusta University) shared an overview of their approach to treating refractory primary immune thrombocytopenia in children and adults. When you first were diagnosed with ITP, your path may have felt like the photo to the right.

Overwhelmed, you wondered which way to go? You saw a doctor and took one or more of the paths. Still your ITP lingers. You may have a case of primary ITP (pITP) that is refractory, meaning your pITP resists treatment. Normal treatments don’t have the desired result of raising your platelets. There is good news -- most of the time your refractory pITP can be managed. Here is an overview of the treatment approach the doctors outlined:

Step 1: Critical reassessment of the diagnosis and a deliberate attempt to exclude non-autoimmune causes of thrombocytopenia and secondary ITP.
Step 2: Achieve confirmation that the patient truly has refractory pITP.
Step 3: Consider Observation without treatment. This is usually only appropriate for patients with a platelet count of ≥ [20,000 /µL – 30,000 / µL] (some patients soldier-on with counts slightly below 20,000 /µL). The key is that whatever base platelet count they settle at, patients need to be symptom free (e.g., no bleeds, petechiae, excessive bruising, etc.).
Step 4: The doctors then describe a three-tiered approach to treatments for patients needing to raise their platelet count.

  • Tier 1: Rituximab, thrombopoietin receptor agonists (i.e., Nplate® or Promacta®), or low-dose corticosteroids. All tier 1 options are exhausted before moving to tier 2 options. Also note that splenectomy would be an option, but this study stated that the patients had excluded that choice.
  • Tier 2: A host of immunosuppressive agents with relatively lower response rates and /or greater toxicity. Some used for ITP include azathioprine (Imuran®) and mycophenolate mofetil (CellCept®). Tier 2 drugs may be prescribed alone; however, they are often administered in combination with one of the Tier 1 drugs or a second Tier 2 drug with a different mechanism of action.
  • Tier 3: These strategies are a “court of last resort”. They are strategies of uncertain benefit and/or high toxicity. These are reserved for the very rare patient who does not respond to the Tier 1 and 2 strategies.


Cuker A and Neunert C. “How I Treat Refractory Immune Thrombocytopenia.” Blood, April 6, 2016.




Patients Should Know the 50 Most Dangerous Drugs

pillsAre you aware of potential side effects of your ITP treatment medications? MSN Medicine has published the top 50 “most dangerous drugs,” which include a few used as ITP therapies. Although all medications have potential side effects, benefits actually far outweigh risks for many therapies. Be sure to educate yourself on your disorder and your treatment regimen, and discuss any questions or concerns with your hematologist.

Two of the five most dangerous drugs on the list, #1 cyclophosphamide (Cytoxan®) and #4 rituximab (Rituxan®) are common ITP medications. Cytoxan restricts cell growth and suppresses the immune system, whereas Rituxan diminishes antibody-producing B cells in your immune system. In patients taking Cytoxan and Rituxan, 97% and 96%, respectively, of adverse reactions reported were serious. Three other medications: Prednisolone (Millipred®), Bevacizumab (Avastin®), and Clozapine (Clozaril®), all reported similar statistics and are used most commonly as metabolic and immunological agents and antipsychotics.

Four other ITP medications: dexamethasone (Decadron®), methylprednisolone (Medrol®), cyclosporine (Sandimmune®), and prednisone were also included on the list. Dexamethasone (#6), methylprednisolone (#11,) and prednisone (#42) reported 93%, 90% and 76%, respectively, adverse reactions as serious. These medications are used as first-line therapies for ITP that function as corticosteroids that control inflammation, stress response, and metabolism. Of the patients who reported having a bad reaction to cyclosporine (#37), 76% reported their response to the medication as serious. Cyclosporine, an immunosuppressant that hinders T-cell activity, has in some cases been found to destroy platelets within the body.

Perry, Sabrina. “The 50 Most Dangerous Drugs.” MSN Medicine. 4/11/2016.




The Benefits of Folic Acid in Food

orange”Folic Acid” seems to be one of the new nutrition buzzwords these days -- but did you know it can help produce healthy platelets too? Folate (Vitamin B9) and Folic Acid (the synthetic version of folate found in supplements and fortified foods) are necessary for cellular growth and regeneration, assist in DNA synthesis and blood cell creation, and prevent fetal abnormalities, Alzheimer’s, and cancer. Thankfully, a healthy diet can provide just the right amount of folate (400-600 micrograms). A plate of dark leafy greens, like spinach, collard greens, kale, and lettuce gives you all the folate your body needs for the day. A serving of asparagus provides 65% of your daily requirement of folate, but also contributes Vitamins K, C, A, and Manganese. Broccoli, beets, and Brussels sprouts eaten raw or lightly steamed are some of the best detox foods and great sources of folate.

Surprisingly citrus foods can provide you with a healthy amount of folate as well: add some oranges, papaya, grapefruit, strawberries, and raspberries to your breakfast or as an afternoon snack. Beans, peas, lentils, and okra can cleanse your digestive tract in addition to providing some of your folate needs. Avocados, which continue to grow in popularity, also offer 22% of your daily folate intake on top of replenishing your body with fatty acids, vitamin K, and dietary fiber. Just adding a few of these foods to your diet each day can do wonders for your overall health and help to boost your platelet counts.

Group, Edward, DC, NP, DACBN, DCBCN, DABFM. “15 Foods High in Folic Acid.” Global Healing Center. MSN Medicine. 11/19/2015.



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