CONTENTS:

• Patient and Physician Perceptions on Treatment Approaches
• FDA approves new drug “Doptelet” for treatment of chronic ITP

Patient and Physician Perceptions on Treatment Approaches

The ITP World Impact Survey, conducted by the top ITP medical experts and patient advocacy groups, sought to understand physicians’ and patients’ beliefs, perceptions, and attitudes toward treatment selection and treatment satisfaction for ITP. The top 5 treatments received by patients were corticosteroids, IVIg, platelet transfusions, rituximab, and TPO-RAs.

Patients and physicians were aligned in their opinions of what was important in selecting a treatment: physicians and patients prioritized the ability to offer sustained remission, reduce bleeding risk, minimize side effects, and avoid immunosuppression. Sustained remission was achieved (and expected) in a similar proportion of patients treated with TPO-RAs, splenectomy, and steroids (48-52%). TPOs were preferred by 32% of physicians for achieving sustained remission compared with splenectomy (26%), steroids (24%), rituximab (12%) and IVIg (6%).

Patients felt that their treatment was effective at treating their ITP symptoms (rituximab 83%, TPO 83%, IVIg 78%, steroids 63%), but splenectomy was the lowest at 36%. Overall satisfaction with treatment was reported by 78% of patients receiving rituximab, 76% with TPO, 69% with IVIg, and 52% with steroids. Only 38% of patients reported overall treatment satisfaction with splenectomy including 25% of splenectomized patients whose platelet counts were “normal”.

Kruse A. Differences in perceptions on treatment approaches between physicians and ITP patients. European Hematology Association. June 14, 2019. https://bit.ly/2X75DCP

Doug Cines, MD, PDSA Medical Advisor Comment:
Multiple options are now available to treat adults with ITP. However, the factors that patients and physicians consider when choosing among these options is not well characterized. Nor is it clear whether patient and physician values are aligned or whether patients view outcomes through the same lens as their physicians. Kruse et al. report that patients and physicians share the same basic treatment goals. The results this survey reinforces the variability in practice patterns in the US based on physician preference. Notably, patients treated with corticosteroids and especially those who were post-splenectomy were less likely to consider their treatment “effective” and expressed less “satisfaction” with outcomes. This study shows that a substantial number of patients with ITP did not consider their treatment “effective” or “satisfactory” even if it succeeded in raising the platelet count. It would be helpful to correlate satisfaction with outcome (e.g. platelet count, bleeding), to compare physician and patient assessment at various platelet counts, and to track patient preferences over time. The influence of physicians on patient perception deserves study as well. This study reinforces the importance of considering patient preference in the management of adults with ITP and of the need for further education to clarify expected outcomes of different treatments.

FDA approves new drug “Doptelet” for treatment of chronic ITP

Dova Pharmaceuticals has received approval for a supplemental New Drug Application to include the treatment of ITP. Doptelet (avatrombopag) has already been approved for treatment of thrombocytopenia in Chronic Liver Disease prior to surgery; the supplemental application allows physicians to recommend Doptelet as a therapy option for adult patients with chronic ITP who have had an insufficient response to a previous treatment. Doptelet is an oral TPO-RA that works to stimulate megakaryocytes in the bone marrow to produce more platelets.

Dova Pharmaceuticals, Inc. Dova Pharmaceuticals Announces FDA Approval of Supplemental New Drug Application for DOPTELET (avatrombopag) for Treatment of Chronic Immune Thrombocytopenia (ITP). Globe Newswire. June 27, 2019. https://bit.ly/2Xy92dv

James Bussel, MD, PDSA Medical Advisor Comment:
Avatrombopag, an oral thrombopoietic agent developed by Dova, was licensed by the FDA for use in patients with ITP who have failed prior therapy. The drug had been previously licensed to treat thrombocytopenia for up to 5 days in patients with liver disease undergoing procedures. Avatrombopag binds to the transmembrane domain of the TPO receptor, like eltrombopag, not at the actual TPO binding site, as does romiplostim. This approval makes avatrombopag the third TPO agent licensed for use in patients with ITP in the US. The drug’s advantages are that it is highly effective, can be taken once daily, no food effects have been described (i.e., no dietary restrictions like taking it on an empty stomach), and does not seem to affect the liver. Disadvantages are only that there is limited data: just over 100 patients were treated in the phase 2 and phase 3 ITP trials combined, and long-term usage has not been studied. For more information see the Dova prescribing information available on-line, the PDSA Platelet News article discussing avatrombopag, and the September 2018 issue of the British Journal of Haematology which reports the results of the phase 3 ITP study, the data from normal volunteers, and an accompanying commentary article.

Disclosure: Dr Bussel is a consultant for Dova, Amgen and Novartis

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