Open Label Multicenter Study of Eltrombopag for the Treatment of Immune ThrombocytoPenia (ITP) Secondary to Chronic Lymphoproliferative Disorders (LPDs)
The denomination of Chronic Lymphoproliferative Disorders (LPD) encompasses a variety of
indolent lymphomas grouped into a single clinical category and, as such, this terminology is
not included in the current WHO classification. With indolent lymphomas clinicians refer to
those lymphomas not associated with an aggressive clinical course and in which often
treatment can be delayed. Specifically the following lymphomas by the WHO classification
will be considered among indolent lymphomas: small lymphocytic lymphoma/chronic lymphocytic
leukemia, follicular lymphoma, marginal zone lymphoma, mantle cell lymphoma,
lymphoplasmacytic lymphoma, hairy-cell leukemia, Hodgkin's lymphoma. In 1 to 5% of the
different LPDs (lowest in follicular lymphoma, highest in chronic lymphocytic leukemia) a
clinically relevant thrombocytopenia, often complicated by bleeding symptoms, may complicate
the clinical course, frequently still when the tumor burden is low and not demanding
treatment. This thrombocytopenia, when not accompanied by massive bone marrow tumor
infiltration or not secondary to chemotherapeutic treatment, is thought to share an immune
pathogenic mechanism similar to primary immune thrombocytopenia (ITP).
With conventional treatments (i.e. iv Ig, steroids) the overall response rate of ITP
secondary to LPD is generally lower than in primary ITP, and usually not higher than 50%
(95% CI 27-72). Therefore, any new treatment having a response rate above 50% but not
inferior than 20% could be considered a promising treatment for ITP secondary to LPD.
Furthermore, no significant platelet increase is expected without treatment in ITP
secondary to LPD. Eltrombopag which has proved very effective in primary ITP could be
effective also in ITP secondary to LPDs.
This novel ITP specific treatment might spare these patients not only from bleeding risk but
also from toxic or inappropriate cytotoxic therapies, not otherwise demanded by the burden
of the underlying disease.
Phase 2, single arm, open-label, prospective, multicenter, safety/efficacy study.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Proportion of responders to eltrombopag as defined by changes in the platelet count, in platelet transfusion requirements and/or in the bleeding symptoms during the 6 months of treatment.
6 months of treatment for each patient
Carlo Visco, MD
Department of Hematology, San Bortolo Hospital, Vicenza, Italy
Italy: The Italian Medicines Agency