View Single Post
  #3  
Old Tue Apr 8, 2014, 01:03 PM
Neil Cuadra Neil Cuadra is offline
Owner
 
Join Date: Jul 2006
Location: Los Angeles, California
Posts: 2,553
Notes on the conference

I thought the Los Angeles patient conference was excellent. I've been going to patient conferences since 1996 and I still learn new information at every one!

Dr. Ramon Tiu gave two detailed presentations on aplastic anemia treatment. In one he surveyed a number of drugs that have been used with or instead of ATG. In each case he explained ways in which the drug has been successful or unsuccessful.

I know I'm oversimplifying here, but here's what I noted:
  • ATG+CsA+Cellcept (mycophenolate mofetil) showed no benefit over ATG+CsA alone. (ATG is anti-thymocyte globulin. CsA is cyclosporine.)

  • Patients taking ATG+CsA+Rapamune (sirolimus) did worse than those taking ATG+CsA alone.

  • Patients taking Campath (alemtuzumab)+CsA did better than those taking ATG+CsA, with a better 2-year survival percentage. In time, we'll have longer-term survival statistics but the results so far are very promising.

  • High-dose Cytoxan (cyclophosphamide) produced a higher mortality rate from fungal infections, but has shown better 10-year survival than ATG.

  • Promacta (eltrombopag) boosts platelets in ITP patients, but has been found to increase all three blood lines, making it another promising treatment choice.

  • Prograf (tacrolimus) acts much like cyclosporine but may be better for kidneys and blood pressure. Most doctors are sticking with CsA until more study data is available for Prograf's use with aplastic anemia.

  • GCSF has little effect on response rates but can lessen the chance of relapse.
We often hear that patients are worried that ATG hasn't worked when their counts go for months without rising. Dr. Tiu explained that you shouldn't rely on blood counts for the first 3 months after ATG, and determine success or failure only at the 6-month mark. That takes a lot of patience! Although 70% to 80% of patients achieve at least some remission from ATG+CsA, only 30% achieve complete remission, and that's disappointing.

I learned two unrelated facts about "pre-existing conditions":
  • When a patient has AA but is not diagnosed with PNH (this is the case two thirds of the time), and is later found to have PNH after treatment for AA, it's probably because the PNH condition was already present, but problems with cell production due to AA prevented the PNH cells from proliferating.

  • Patients who have muscle or nerve pain after ATG probably had underlying causes before their disease and treatment, with ATG treatment aggravating the pre-existing condition. Long-term muscle or nerve pain is rarely caused by ATG.
I heard interesting news about biomarkers from Dr. Tiu and I got to talk with Dr. Bejar about it too. Biomarkers are clinical factors or lab tests that correlate to disease treatment outcomes and can be used to predict the likely outcome for patients having those biomarkers. For example, patients with higher absolute reticulocyte counts (ARC) and absolute lymphocyte counts (ALC) have higher ATG response rates. That's been known for some time but new biomarkers are being found all the time. Study continues on telomeres, where shorter telomere length predicts higher relapse rates, higher evolution (AA to MDS or MDS to leukemia), and lower survival rates. The key with biomarkers is to find ones that can be used to make the best treatment decisions, not just predict how a patient will do.

I always enjoy listening to talks by Dr. Isabel Schuermeyer, and this time was no different. She spoke about fatigue and the effects of a major disease on our lives. I learned that the fatigue that often accompanies bone marrow failure isn't directly related to blood count levels. It's a side effect of the disease and the body's fight against it, not just a result of anemia. It turns out that many patients under-report their fatigue because they fear that they'll get less aggressive treatment. Studies show that physicians tend to underestimate the impact that fatigue has on their patients' quality of life, so fatigue is an area that deserves more attention from everyone.

I often use the phrase "the new normal" to describe how a family's routines, habits, and life change when a major illness strikes. Dr. Schuermeyer said that some of her patients dislike that phrase, saying there's nothing normal about it. They prefer the phrase "the new reality".

The AA&MDSIF staff was as friendly and helpful as always, and the hotel services were fine. The only way in which I found the patient conference lacking was that there was no session on transplants, a subject that will continue to be very important to patients making treatment decisions.
Reply With Quote