Refractory primary immune thrombocytopenia with subsequent del(5q) MDS: complete remission of both after lenalidomide

Abstract

A patient with refractory primary immune thrombocytopenia (ITP) characterised by severe skin and mucosal bleedings was treated with several ITP-directed therapies including cyclophosphamide. He later developed therapy-related del(5q) myelodysplastic syndrome with no dysplastic morphological features in bone marrow. He remained severely thrombocytopenic, which suggests ongoing immune mediated platelet destruction. After two 3 week cycles of low-dose lenalidomide, complete cytogenetic remission and complete normalisation of platelet count were observed. This suggests that lenalidomide may be a viable treatment option for ITP in the presence of del(5q) not responding to standard treatments.

Refractory Immune Thrombocytopenia or Myelodysplastic Syndrome Presenting with Isolated Thrombocytopenia? A Review of Literature and a Case Report

“…There are several although scattered reports of immune-mediated platelet destruction following variable periods of treatment with lenalidomide, all of them characterized by an overall good response to corticosteroids [36][37][38][39]. Moreover, there are even reports of co-existent ITP and 5q-deleted MDS both successfully treated with single-agent lenalidomide [40]. One of the above mentioned studies, however, unexpectedly reports on steroidresistance and subsequent findings of bone marrow aplasia (both red cells and megakaryocyte) in the bone marrow, with the newly discovered presence of a PNH clone.…”

Techniques for detecting chromosomal aberrations in myelodysplastic syndromes

“…Moreover, selection of particular treatment may rely on the presence of specific chromosomal aberrations. Low-risk, transfusion-dependent MDS patients with del(5q) are reported to respond well to lenalidomide [ 28 – 30 ]. So accurate detection of del(5q) is not only important for precise diagnosis of MDS, but also vital for individualized treatment of MDS patients.…”