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PDSA E-News: September 27, 2017



TPO Comes Out on TOP for ITP in Pregnancy

Blood logoPregnancy can be difficult on women, managing life with ITP is difficult for everyone, but how difficult are the challenges for expectant mothers with ITP? PDSA Medical Advisor Dr. James Bussel and Dr. Eun-Ju Lee, both of Weill Cornell Medical Center, have published a new overview of treatment options for pregnant women with ITP in the American Society for Hematology’s Blood Journal.

Previous research has found a low incidence of severe bleeding in the fetus and a less than 1% finding of fetal brain bleeds in mothers with ITP. The major issue, however, is that platelet levels typically fall for all pregnant women, posing a unique threat to women with ITP. As a result, close monitoring of the mother and her platelet count are required throughout gestation.

Common treatments to elevate platelet counts during pregnancy include IVIG, which increases counts temporarily, and thus is helpful in an emergency or during planned delivery. Low doses of prednisone (20 mg daily) is also recommended for the first two trimesters, but higher doses may be required closer to delivery. Pregnant women should avoid Rituximab, Anti-D, and Azathioprine, as results of the use of these therapies during pregnancy have been inconclusive or found to be harmful to either the mother or the fetus.

A review of a multicenter study in China examined women during their first pregnancy who had previously failed to raise platelet counts following steroids or IVIG. Researchers administered an initial 300 units/kg of recombinant human TPO (rhTPO) for two weeks, and then adjusted their dose based on their response to the therapy. RhTPO stimulates the bone marrow to produce more platelets like other TPO treatments, however, it is a larger molecule made from human cells that does not cross the placenta. The results were promising: seventy-five percent of the study participants elevated their platelet count above 100,000 x 109/L following therapy.

It is unknown whether recombinant human TPO (which is different from the TPO mimetics Eltrombopag and Romiplostim which could affect fetal bone marrow) could affect the baby’s immune system while breastfeeding. A one-year follow up of neonate development was encouraging and did not show an increased TPO level in cord blood. Unfortunately, recombinant human TPO is not currently approved in the United States. Drs. Bussel and Lee recommend a replication of the aforementioned study using the available Eltrombopag and Romiplostim in the U.S., which could improve treatment options for pregnant women with ITP.

Bussel, James, MD and Lee, Eun-Ju, MD. “TPO for ITP in pregnancy.” Blood. 2017.

Genetic Variants in ITP: A Review

Medical researcher looking at test tubeEvidence has shown a number of immune responses that could be responsible for ITP development and perpetuating bleeding symptoms. Pharmacist and medical writer, James Nam, explains some of the key potential changes that could occur in ITP:

Signalling molecules are secreted by some immune cells. An imbalance of T-helper cells increase these molecules which cause inflammation, elevating rates of platelet destruction. T-helper cells also activate macrophages that “eat” platelets. Suppressing the molecules that signal the T-helper cells that cause this inflammatory response would increase platelet counts.

Another component of the immune system, B-cells are activated by an inflammatory reaction, increasing antibody production. This causes suppression of platelet production and could serve as a biomarker to monitor disease progress. A special protein regulates immune response by differentiating between native (self) and foreign cells; detection of genetic variants that diminish this protein’s activity are correlated with increased risk for ITP. B-cell antibody production is regulated by how many antibodies bind to specific receptors on cells; binding power of antibodies to receptors is increased when changes occur to the shape of the receptor, which enhances platelet destruction.

Researchers recommend scrutinizing genetic variants through “screening, early detection, and control of disease” to help personalize treatment for each ITP patient.

Nam, James, PharmD. “Role of Polymorphisms in Idiopathic Thrombocytopenic Purpura: A Review.” Oncology Nurse Advisor. 8/30/17.



Understanding the Uniqueness of Medical Care for Rare Diseases

two sided scaleUsing “value” to justify treatment cost has traditionally been the easiest way to analyze healthcare. However, therapy value differs between diseases and is difficult to quantify for rare disorders due to the relatively small number of patients with one specific rare disease. As ITP patients know, research and development for rare diseases are complicated by the degree of complexity, small clinical trial sizes, and limited availability to specialists. Different considerations are needed for the way that analysts evaluate the advantages of high per-patient costs of research in and treatment for rare diseases:

  1. Development of rare disease therapies helps current patients and better prepares for those at risk for diagnosis in the future, creating an “insurance value;” severe diseases represent greater risk to the healthy because they more greatly impact quality-of-life.
  2. The public values equal accessibility to improve overall health, especially for those already at a disadvantage.
  3. Rare diseases impact caregivers, leading to long-term financial burdens and declines in physical, mental, and emotional well-being in addition to the already established negative impacts to rare disease patients; traditional cost-benefit analyses exclude the spillover “cost of care.”
  4. Rare diseases, which are typically genetically-based instead of environmentally induced, could have higher incentives to find a “cure.” Gene therapy has been proven to be an effective, yet extremely expensive, method of tailoring disease treatment based on biomarkers. The value of gene therapy is high because of increased probability of a cure, eliminating the cost of future therapy.

Awareness of these perspectives is important for providers to recognize and for patient advocates to understand the value of innovation in rare disease research and development.

Jena, Anupam and Lakdawalla, Darius. “Value Frameworks for Rare Diseases: Should They Be Different?” Health Affairs. 4/12/2017.




Probiotics Pack a Punch Against Sepsis, Other Infections

Probiotic pillsSepsis, caused by an immune response to an infection in the bloodstream, has become an increasingly common and difficult-to-treat disease in hospitals, with one million Americans developing Sepsis each year and over half dying from the infection. Patients who’ve had a splenectomy have an increased risk of developing Sepsis. While traditional treatments have failed, a new therapy has shown promise, and it might already be in your home.

A study conducted in India demonstrated that taking probiotics significantly lowers the risk of developing sepsis. A specific bacterium, Lactobacillus plantarum, which is found in fermented vegetables was demonstrated to be the most efficient strain, and also reduced the risk for respiratory and other bacterial infections. Probiotics may be more powerful than traditional antibiotics because of the way that the “good” bacteria interacts with “bad” bacteria in the gut, strengthens the intestines, and bolsters immune systems.

Mercola, Joseph, MD. “Probiotics Offer Powerful Protection Against Sepsis in Infants.” Mercola. 8/28/2017.

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