Lenalidomide (LEN) can induce red blood cell-transfusion independence (RBC-TI) in 60-70% of del(5q) myelodysplastic neoplasm (MDS) patients. Current recommendation is to continue LEN in responding patients until failure or progression, with likelihood of toxicity and a high cost for healthcare systems. This HARMONY Alliance study investigated the outcome of MDS del(5q) patients who discontinued LEN while RBC-transfusion independent. We enrolled 118 patients with IPSS-R low-intermediate risk. Seventy patients (59%) discontinued LEN for intolerance, 38 (32%) per their physician decision, nine (8%) per their own decision and one (1%) for unknown reasons. After a median follow-up of 49 months from discontinuation, 50/118 patients lost RBC-TI and 22/30 who underwent cytogenetic re-evaluation lost complete cytogenetic response. The median RBC-TI duration was 56 months. In multivariate analysis, RBC-TI duration after LEN discontinuation correlated with low transfusion burden before LEN therapy, treatment >= 12 LEN cycles, younger age and higher Hb level at LEN withdrawal. Forty-eight patients were re-treated with LEN for loss of response and 28 achieved again RBC-TI. These data show that stopping LEN therapy in MDS del(5q) patients who reached RBC-TI allows prolonged maintenance of TI in a large subset of patients.

Transfusion independence after lenalidomide discontinuation in patients with del(5q) myelodysplastic neoplasm: a HARMONY Alliance study / Crisà, Elena; Mora, Elvira; Germing, Ulrich; Bally, Cecile; Diez Campelo, Maria; Myllymäki, Mikko; Jädersten, Martin; Komrokji, Rami; Platzbecker, Uwe; Haase, Detlef; Hofmann, Wolf-Karsten; Al Ali, Najla H.; Barraco, Daniela; Bargay, Juan José; Bernal, Teresa; López Cadenas, Felix; Calvisi, Anna; Capodanno, Isabella; Cerrano, Marco; Ciancia, Rosanna; Crugnola, Monica; Kündgen, Andrea; Finelli, Carlo; Fozza, Claudio; Frairia, Chiara; Freja, Ebeling; Ganster, Christina; Kubasch, Anne Sophie; Jimenez, Maria Jose; Latagliata, Roberto; Hernandez Mohedo, Francisca; Molero, Antonieta; Vara Pampliega, Miriam; Perez, Clara Aparicio; Pietrantuono, Giuseppe; Poloni, Antonella; Pomares, Helena; Recasens, Valle; Rüfer, Axel; Signori, Alessio; Hellstrom-Lindberg, Eva; Fenaux, Pierre; Sanz, Guillermo; Santini, Valeria. - In: LEUKEMIA. - ISSN 0887-6924. - 38:10(2024), pp. 2259-2265. [10.1038/s41375-024-02360-1]

Transfusion independence after lenalidomide discontinuation in patients with del(5q) myelodysplastic neoplasm: a HARMONY Alliance study

Poloni, Antonella;
2024-01-01

Abstract

Lenalidomide (LEN) can induce red blood cell-transfusion independence (RBC-TI) in 60-70% of del(5q) myelodysplastic neoplasm (MDS) patients. Current recommendation is to continue LEN in responding patients until failure or progression, with likelihood of toxicity and a high cost for healthcare systems. This HARMONY Alliance study investigated the outcome of MDS del(5q) patients who discontinued LEN while RBC-transfusion independent. We enrolled 118 patients with IPSS-R low-intermediate risk. Seventy patients (59%) discontinued LEN for intolerance, 38 (32%) per their physician decision, nine (8%) per their own decision and one (1%) for unknown reasons. After a median follow-up of 49 months from discontinuation, 50/118 patients lost RBC-TI and 22/30 who underwent cytogenetic re-evaluation lost complete cytogenetic response. The median RBC-TI duration was 56 months. In multivariate analysis, RBC-TI duration after LEN discontinuation correlated with low transfusion burden before LEN therapy, treatment >= 12 LEN cycles, younger age and higher Hb level at LEN withdrawal. Forty-eight patients were re-treated with LEN for loss of response and 28 achieved again RBC-TI. These data show that stopping LEN therapy in MDS del(5q) patients who reached RBC-TI allows prolonged maintenance of TI in a large subset of patients.
2024
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11566/333739
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