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

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 syste...

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Main Authors: Crisà, Elena (Author) , Mora, Elvira (Author) , Germing, Ulrich (Author) , Bally, Cecile (Author) , Diez Campelo, Maria (Author) , Myllymäki, Mikko (Author) , Jädersten, Martin (Author) , Komrokji, Rami (Author) , Platzbecker, Uwe (Author) , Haase, Detlef (Author) , Hofmann, Wolf-Karsten (Author) , Al Ali, Najla H. (Author) , Barraco, Daniela (Author) , Bargay, Juan José (Author) , Bernal, Teresa (Author) , López Cadenas, Felix (Author) , Calvisi, Anna (Author) , Capodanno, Isabella (Author) , Cerrano, Marco (Author) , Ciancia, Rosanna (Author) , Crugnola, Monica (Author) , Kündgen, Andrea (Author) , Finelli, Carlo (Author) , Fozza, Claudio (Author) , Frairia, Chiara (Author) , Freja, Ebeling (Author) , Ganster, Christina (Author) , Kubasch, Anne Sophie (Author) , Jimenez, Maria Jose (Author) , Latagliata, Roberto (Author) , Hernandez Mohedo, Francisca (Author) , Molero, Antonieta (Author) , Vara Pampliega, Miriam (Author) , Perez, Clara Aparicio (Author) , Pietrantuono, Giuseppe (Author) , Poloni, Antonella (Author) , Pomares, Helena (Author) , Recasens, Valle (Author) , Rüfer, Axel (Author) , Signori, Alessio (Author) , Hellstrom-Lindberg, Eva (Author) , Fenaux, Pierre (Author) , Sanz, Guillermo (Author) , Santini, Valeria (Author)
Format: Article (Journal)
Language:English
Published: October 2024
In: Leukemia
Year: 2024, Volume: 38, Issue: 10, Pages: 2259-2265
ISSN:1476-5551
DOI:10.1038/s41375-024-02360-1
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1038/s41375-024-02360-1
Verlag, lizenzpflichtig, Volltext: https://www.nature.com/articles/s41375-024-02360-1
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Author Notes:Elena Crisà, Elvira Mora, Ulrich Germing, Cecile Bally, Maria Diez Campelo, Mikko Myllymäki, Martin Jädersten, Rami Komrokji, Uwe Platzbecker, Detlef Haase, Wolf-Karsten Hofmann, Najla H. Al Ali, Daniela Barraco, Juan José Bargay, Teresa Bernal, Felix López Cadenas, Anna Calvisi, Isabella Capodanno, Marco Cerrano, Rosanna Ciancia, Monica Crugnola, Andrea Kündgen, Carlo Finelli, Claudio Fozza, Chiara Frairia, Ebeling Freja, Christina Ganster, Anne Sophie Kubasch, Maria Jose Jimenez, Roberto Latagliata, Francisca Hernandez Mohedo, Antonieta Molero, Miriam Vara Pampliega, Clara Aparicio Perez, Giuseppe Pietrantuono, Antonella Poloni, Helena Pomares, Valle Recasens, Axel Rüfer, Alessio Signori, Eva Hellstrom-Lindberg, Pierre Fenaux, Guillermo Sanz and Valeria Santini

MARC

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520 |a 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. 
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