Long-Term Response in a Patient with del(5q) Myelodysplastic Syndrome Who Discontinued Lenalidomide and Obtained a Good Response and Tolerance to Rechallenge

Abstract

Background: The introduction of the immunomodulatory drug lenalidomide has revolutionized the treatment of patients with myelodysplastic syndromes (MDS) and deletion of the long arm of chromosome 5. Treatment with lenalidomide results in transfusion independence in the majority of patients, but some questions remain unresolved, among them the duration of treatment. Moreover, a number of unexpected long-term remissions in patients who stopped lenalidomide for various reasons have been observed. Case Report: We report the case of a 60-year-old Caucasian male with deletion of the long arm of chromosome 5 and International Prognostic Scoring System (IPSS)-defined low-risk MDS who was treated with lenalidomide, achieving complete cytogenetic remission and erythroid response. After tapering off and interrupting the treatment, the patient relapsed and showed a new response by lenalidomide retreatment. Six years after the initial treatment, we registered a durable erythroid long-term response and good tolerance, but there was no evidence of a very profound cytogenetic response compared to using lenalidomide as a first-line treatment. Cytogenetic and fluorescence in situ hybridization together with hemoglobin level, mean corpuscular volume (MCV) and vitamin B₁₂ level helped us to monitor the patient response; during the various phases of lenalidomide treatment, MCV and vitamin B₁₂ normalization correlated with good response. Conclusion: Lenalidomide interruption and rechallenge in some 5q- MDS patients, with low risk according to the IPSS, is safe and feasible but does not result in a profound cytogenetic response.

8-year-sustained response in a patient with isolated del(5q) myelodysplastic syndrome undergoing continuous treatment with lenalidomide: a case report

“…In a small patient series, drug discontinuation after having achieved complete cytogenetic remission was associated with prolonged responses [17] with patients who relapsed to transfusion dependence responding to reexposure of lenalidomide. A similar observation has been reported [18] in a patient with isolated del(5q) MDS who was treated with lenalidomide, achieved complete hematologic and cytogenetic remission and relapsed to transfusion dependence after a 14-month treatment interruption. A durable hematologic response was observed after reexposure to lenalidomide, but cytogenetic response was not as profound as after the initiation of lenalidomide.…”

Durable response to lenalidomide in a patient with myelodysplastic syndrome associated with isolated 5q deletion and JAK2 V617F mutation despite discontinuation of treatment

“…Although it was recently shown that lenalidomide treatment in MDS del(5q) patients does not appear to increase the risk to AML transformation (14), other long-term effects of immunomodulation have not been elucidated and one could argue that intermittent use of lenalidomide may be prudent is certain cases. The present case indicated that durable hematological response may be sustained despite discontinuation of lenalidomide, whereas others have shown that re-challenge may also be safe and feasible, albeit with no profound cytogenetic response (15). However, in order to draw safe conclusions regarding the treatment duration and time of discontinuation, a randomized clinical trial is required to compare continuous treatment until progression, vs. treatment discontinuation after having achieved sustained complete cytogenetic response.…”