News from the American Society of Hematology Annual Meeting
Each year the American Society of Hematology (ASH) meeting attracts thousands of clinicians and scientists, worldwide, to learn about and report on the latest research. This year’s meeting, December 7 to 10 in New Orleans, featured more than 10 hours of presentations and 90 pages of abstracts about ITP and related diseases. Below are some trends.
(The ASH abstract numbers are in parentheses. You can search on the number and read the complete abstract at https://ash.confex.com/ash/2013/webprogram/start.html, after you agree with the terms.)
Mixing Current Treatments
Understanding More about TPO Treatments
Making a Correct Diagnosis
Improving the Treatment of Children
Mixing Current Treatments
Dr. Howard Liebman: "By using a combination of agents it may be possible to achieve a greater efficacy with acceptable toxicity."
Triple Therapy Attacks Three Sources (1084)
Treating with a single therapy targets only one of the three possible cell types involved in lowering platelets in ITP so researchers experimented with treatment combinations that addressed all three. They treated 26 patients with various doses of rituximab (B-cells), prednisone or dexamethasone (T-cells) and cyclosporine or mycophenolate mofetil (antigen-presenting cells). After a year, half of the patients were still in remission.
Dexamethasone Plus Rituximab Improves Outcome (2310)
Only 20% of people taking rituximab (Rituxan®) achieve a long-term remission. However, when newly diagnosed (less than 2 years) patients were treated with a combination of rituximab and 3 cycles of dexamethasone, about 75% had a positive response. After five years, 59% maintained a complete response.
Understanding More about TPO Treatments
Bone Marrow Changes Rare (326)
When the TPO treatments (romiplostim/Nplate®, eltrombopag/Promacta®) were approved, some doctors and regulators had concerns about patients developing problematic changes in their bone marrow. In a study of the bone marrow of patients taking eltrombopag (Promacta®) for up to 5 years, independent experts found that bone marrow reticulin (scarring) was either absent or only mildly increased in 98% of the patients tested. No patients developed lasting bone marrow problems due to the treatments.
Off Treatment, Still in Remission (327, 328, 2323)
In one study, 15% of the patients who received TPO treatments remained in remission after treatments were discontinued. In a second study of patients who participated in TPO clinical trials, those who had ITP fewer than 5 years were more likely to sustain a remission after discontinuation. A third study mirrored the positive results and showed that patients who were treated about a month could achieve a lasting remission.
Making a Correct Diagnosis
Know your H. pylori Status (2321, 2324, 3548)
H.pylori bacteria have been associated with ITP and past research has shown that eradication of the bacteria can improve platelet counts. However, the results were not consistent. At ASH, three studies addressed the connection between H.pylori and ITP. All three reported some positive platelet response after eradicating H.pylori. One study was from Iran, another from Korea, and a third from an ethnically diverse patient population in Los Angeles. This confirms earlier findings that patient ethnicity can be a factor in the success of this treatment.
New Genetic Cause Found (565)
ITP is a diagnosis of exclusion, when all other causes of low platelets are ruled out. However more causes of low platelets are being discovered each year. Researchers did genetic studies on several members of a family in Michigan who were diagnosed with ITP as children. They discovered that a mutation to a gene producing nitrous oxide (NOS3) was the cause of low platelets in this family, not ITP. For more information on low platelets and families see:
http://www.pdsa.org/about-itp/and-families.html
Improving the Treatment of Children
Observation Increasing (772)
The 2011 updated ASH Guidelines for ITP recommend that children be managed with observation alone if they have only mild or no bleeding symptoms, regardless of platelet count. To determine if these guidelines were changing practice, a large hospital in Pennsylvania looked at how they managed newly-diagnosed children with ITP. They did find a marked decrease in treatment over the years. The percent of children observed and not treated increased from 34% in 2007-2010 to 72% in 2012.
Longer-term TPO Results Positive (3530, 3541)
In one study, children treated with romiplostim (Nplate®), some more than three years, continued to maintain a platelet count greater than 50,000. In another report, 82% of the 33 children treated with either romiplostim or eltrombopag (Promacta®) responded to the treatments. Some of the children had additional therapies and a few were able to stay in remission after therapy was discontinued. No severe side effects were reported.
