PDSA E-News: July 27, 2016

This issue of the e-news is devoted to patient–centered research findings reported at this year’s ITP conference in Orlando, FL.





Treating ITP: Balancing the Risks vs. Benefits

Doctors on stage at ITP ConferenceDuring the ITP Conference 2016 Saturday morning session, Drs. Arnold, Tarantino, Kessler, and Bussel presented four leading ITP treatments, explaining how they work and possible drawbacks.  Dr. Donald Arnold, of McMaster University in Canada, discussed the thrombopoietin receptor agonists (TPOs) that include Nplate® (romiplostim) and Promacta/Revolade® (eltrombopag).  Both TPOs increase platelet production in the patient’s bone marrow.  He said they are remarkably safe and effective.  They have been found safe when used long term, with no major side effects.  We have about a decade of results now.  In about 25-30% of ITP patients TPO-induced remissions have occurred.  Both Nplate and Promacta/Revolade have been found to have a 70-90% success rate of raising platelet counts.

Dr. Michael Tarantino, of the Bleeding and Clotting Disorders Institute, discussed the pros and cons of IVIg as an ITP treatment.  On the good side, IVIg raises platelets rapidly, usually in 1-3 days.  The platelet-raising effects last from weeks to sometimes months.  It is usually well-tolerated.  On the bad side, IVIg has a high cost (thousands of dollars per treatment) and possible side effects such as headaches, fevers, or infusion reactions.  These occur in about one-third of patients.  And, IVIg doesn’t stop ITP—it raises platelet counts temporarily. 

Dr. Craig Kessler, of Georgetown University, described using rituximab (Rituxan®) for ITP.  This second-line ITP treatment is infused to humans and affects what is causing the ITP.  It is very effective at attacking the B cells that turn on the antibody action that causes attacks on the platelets.  The first rituximab treatment is the most likely to cause an allergic reaction.  Rituximab has a 50% response rate and the platelet raising effect is durable (lasting up to five years).  If rituximab is combined with other types of corticosteroids patients may get a better response.  Time to response is approximately 4 - 6 weeks.  The main drawbacks of rituximab are: it’s expensive and the first dose must be given with medical supervision.  Also, rituximab cannot be given in pregnancy and a serious allergic reaction can be fatal. 

Dr. James Bussel, of Cornell University, discussed splenectomy for treating ITP patients.   In the past, ITP treatment was usually steroids, then splenectomy, until IVIg was available in 1981.  Most splenectomies today are done with laparoscopic surgery with recovery time about 1 - 2 weeks.  If a splenectomy ‘works’ the patient’s platelet count may increase right away.  He said the highest rate of ITP ‘cure’ with surgery is splenectomy.  The highest rate of cure (long –term remission) without surgery or ongoing treatment is rituximab (Rituxan®).

Most ITP patients don’t want a spleen removal now because it is only successful at raising platelet counts in about 60-65% of patients.  Prior to the 1980s it was widely used.  Also, Dr. Bussel mentioned the possible serious side effects of the surgery, including bleeding, infection and clots.  Some patients have developed serious post-splenectomy sepsis (blood infection). 


Can We Cure ITP? Research that Considers Patients' Needs

Dr. David Kuter on stage at ITP ConferenceAt this Saturday session at the ITP Conference PDSA Medical Advisors Drs. Craig Kessler, James Bussel, and David Kuter revealed emerging research and clinical trials for ITP.  First, Dr. Kessler explained the pathophysiology of ITP, demonstrating that each step of platelet production and destruction is a potential treatment target, which provides better and more tailored care for ITP patients.

Dr. Bussel described fostamatinib (from Rigel), PRTX-100 (from Protalex), and dexamethasone/rituximab (Rituxan®).  Fostamatinib blocks signaling of the macrophage to bind to an anti-platelet antibody.  It’s currently in phase 3 of clinical trials to examine toxicity and patient response; phase 2 trials yielded 50% response.  PRTX-100 binds IgG molecules limiting platelet destruction; open label trials started recently, yielding good responses, and few side effects with low doses of the drug have been observed. 

Cycling dexamethasone and rituximab has produced good responses in 70% of patients.  He said it’s important to note the development of two new anti-CD20 antibodies similar to rituximab: ofatumumab and obinutuzumab.  These also inhibit lymphocyte activation, reducing the cells available that produce platelet-coating antibodies.  ITP patients who have had adverse reactions to rituximab cannot use that drug again.  Ofatumumab and obinutuzumab could be administered instead if that was deemed the best option for care.

Dr. Kuter, of Massachusetts General Hospital, discussed the development of anti-CD40 ligand antibodies and FcRn pathway inhibitors.  Anti-CD40 antibodies (IDEC-131, phase 2) reduce the production of anti-platelet antibodies and regulate immune response.  These antibodies provide a good platelet response; however, some patients in initial clinical trials experienced unexpected thromboembolism.  Researchers noted that blood clots can be prevented by modifying the Fc receptor.  Development for FcRn pathway inhibitors has been underway as well.  FcRn is vital for IgG lifespan and protects the antibody from degradation.  When FcRn is blocked, pathogenic IgG antibodies are degraded, T-cell activation is inhibited, inflammation is blocked, and clearance of anti-platelet antibodies is increased.




Patient-Centered Outcomes Research (PCOR): What is it and Why is it Important to Patients with a Rare Disease?

Dr. Danielle Whicher on stage at the ITP ConferenceIn addition to showing videos of patient questions before sessions, providing a just-for-teens track, and creating new patient-centered research guidelines during the 2016 ITP Conference, PDSA had the pleasure of introducing a representative from the nonprofit Patient-Centered Outcomes Research Institute (PCORI), Dr. Danielle Whicher, to educate patients about Patient-Centered Outcomes Research and its importance in rare diseases.  PCORI promotes research that emphasize hypotheses answering critical questions from patients and caregivers.  They focus specifically on generating results that can be easily applied to patients and underscore the importance of engaging patients throughout the research process.  They believe that patient-engaged research will culminate in findings that will be increasingly utilized in a clinical setting.

What exactly is Patient-Centered Outcome Research?  PCOR investigates commonly used treatment options of a clinical disease in a real-world setting.  It fills evidence gaps such as “are there different ways of managing the clinical condition of interest?  Is it difficult to choose?  Do different doctors recommend different treatment approaches?”  Most importantly, patients are involved in research design, ensuring that the study provides a solution to vital patient questions.  PCOR answers the question “which of the available options is best and for which patients?” and produces evidence that helps patients and physicians make more informed healthcare decisions.

PCOR is crucial for patients with rare diseases such as ITP.  Considering there isn’t a cure for ITP, there tend to be differences in the way this disorder and its symptoms are treated in a clinical setting.  While it’s wonderful that patients have a plethora of therapy options, questions still exist regarding the best method to treat ITP.  Ultimately, PCOR will be invaluable for PDSA and ITP patients, as the development of patient-centered studies will surely yield meaningful information on the best way to manage ITP for each patient.




Care for the Caregiver

Jane Pernotto Ehrman on stage at the ITP ConferenceIn another Saturday session, attendees listened as Jane Pernotto Ehrman, of the Cleveland Clinic, described the symptoms of caregiver burnout and what caregivers can do to improve their quality of life, enabling them to improve their own health and be the best caregivers.  

Caregivers may be experiencing burnout if they find themselves being cynical, overly critical, irritable, and impatient with family members and others.  They may find themselves procrastinating, hating Mondays, and having difficulty getting started at work, along with experiencing low energy and poor concentration.  They may not get enough sleep or have poor quality sleep and ‘numb out’ with food, substance, or screens (TV, phone and computer). 

Ultimately, burnout consequences can include:  chronic stress, fatigue, insomnia, anxiety, depression, addiction and chronic health issues like heart disease, Type 2 diabetes, stroke, obesity or frequent illness.  At work, it can cause decreased productivity, lack of engagement with others, poor attitude, inability to work on teams, more pressure and added responsibility on co-workers, and increased absenteeism.

She described ways to take of yourself as a caregiver.  These include:  eating nourishing food  (not junk food); getting adequate, higher quality sleep; increasing physical activity several times a  week; finding healthy stress relief such as meditation, exercise or journaling; setting aside some ‘alone time’; finding ways to bring laughter to your life; keeping up friendships; and learning ways to play (which offers relaxation).

She guided the group through a short mindfulness/meditation exercise intended to help caregivers be in the moment, focusing on one thing, while sitting relaxed with eyes closed.  As she talked the group through the steps, including focus on their breathing, the attendees felt stress relief and calmness.


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