April 10, 2011
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ASH updates guidelines for immune thrombocytopenia

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The introduction of thrombopoietin receptor agonists and rituximab played a key role in the American Society of Hematology’s decision to update the 1996 guidelines for the treatment of immune thrombocytopenia.

“In contrast to the previous ASH guideline, this guideline is an evidence-based guideline that uses explicit methodology and the Grades of Recommendation, Assessment, Development and Evaluation (GRADE) Working Group system to categorize evidence based on the quality of the findings and the strength of the recommendation,” Wendy Lim, MD, assistant professor in the division of hematology and thromboembolism in the department of medicine at McMaster University in Ontario, Canada, told HemOnc Today.

Highlights of the guidelines

The new guideline is comprehensive and was based on findings from Embase and Medline databases from 1996 to 2009. Lim discussed some of the key changes from 1996:

  • Bone marrow examination is no longer a routine part of the diagnostic assessment for children presenting with typical features of ITP.
  • Rituximab (Rituxan, Genentech/Idec Pharmaceuticals) may be considered for children or adolescents with significant ongoing bleeding despite treatment with intravenous immunoglobulin, anti-D or conventional doses of corticosteroids.
  • Splenectomy receives a strong recommendation for patients who have failed initial therapy
  • Thrombopoietin receptor agonists or rituximab are recommended in adult patients at risk of bleeding who relapse after splenectomy or have a contraindication to splenectomy and have failed another therapy.
  • Testing for HIV and hepatitis C should be considered in all adult patients with acute disease.
  • Treatment should no longer be based on platelet count alone; rather, it should be based on symptoms and quality of life.

“For pediatric hematologists, it will eliminate bone marrow examinations in the work-up of many children who present with typical features of [immune thrombocytopenia],” Lim said. “Adult hematologists/oncologists will likely be looking for guidance on the sequence in which splenectomy, thrombopoietin receptor agonists and rituximab should be used, and whether they should be used before or after splenectomy.”

There is no evidence-based answer to the latter issue, and accordingly, no evidence-based recommendation could be made, according to Lim. However, she said she hoped that the guidelines would educate clinicians on the use of these agents and the context in which they were studied in clinical trials.

Rationale

“The original ASH guideline was published in 1996,” Lim said. “Since that time, there have been recent important publications on standardizing the terminology and definitions of [immune thrombocytopenia], as well as an international consensus report on the investigation and treatment of the disease.”

She said the introduction of new therapies was generating interest in the clinical community, and questions had been raised on the appropriate use of those agents.

“We felt that an update on this guideline was warranted and would be of interest to ASH and its audience,” Lim said.

The researchers said the GRADE system is used in areas where such evidence exists, but that evidence is not provided in areas where it is absent or of lower quality. In those areas, clinicians are encouraged to follow up with other consensus-based recommendations fueled by expert opinions.

“As a final point, I would like to note that the panel for these guidelines was selected for their lack of conflict of interest,” Lim said. “Specifically, none of the authors received honoraria or financial support from pharmaceutical companies that manufacture agents used in the treatment of [immune thrombocytopenia]. This was done to try to formulate guideline recommendations that were evidence based and free of the perception of conflicts of interest.”

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