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

被引：0

作者：

Crisa, Elena ^{[1
,2
]}

Mora, Elvira ^{[3
]}

Germing, Ulrich ^{[4
]}

Bally, Cecile ^{[5
]}

Campelo, Maria Diez ^{[6
]}

Myllymaki, Mikko ^{[7
,8
]}

Jadersten, Martin ^{[9
]}

Komrokji, Rami ^{[10
]}

Platzbecker, Uwe ^{[11
]}

Haase, Detlef ^{[12
]}

Hofmann, Wolf-Karsten ^{[13
]}

Al Ali, Najla H. ^{[10
]}

Barraco, Daniela ^{[2
,14
]}

Bargay, Juan Jose ^{[15
]}

Bernal, Teresa ^{[16
]}

Cadenas, Felix Lopez ^{[17
]}

Calvisi, Anna ^{[2
,18
]}

Capodanno, Isabella ^{[2
,19
]}

Cerrano, Marco ^{[20
,21
]}

Ciancia, Rosanna ^{[2
,22
]}

Crugnola, Monica ^{[2
,23
,24
]}

Kundgen, Andrea ^{[4
]}

Finelli, Carlo ^{[2
,25
]}

Fozza, Claudio ^{[2
,26
]}

Frairia, Chiara ^{[2
,21
]}

Freja, Ebeling ^{[27
]}

Ganster, Christina ^{[12
]}

Kubasch, Anne Sophie ^{[11
]}

Jimenez, Maria Jose ^{[28
]}

Latagliata, Roberto ^{[29
,30
]}

Mohedo, Francisca Hernandez ^{[31
]}

Molero, Antonieta ^{[32
]}

Pampliega, Miriam Vara ^{[33
]}

Perez, Clara Aparicio ^{[34
]}

Pietrantuono, Giuseppe ^{[2
,35
]}

Poloni, Antonella ^{[2
,36
]}

Pomares, Helena ^{[37
]}

Recasens, Valle ^{[38
]}

Rufer, Axel ^{[39
]}

Signori, Alessio ^{[40
]}

Hellstrom-Lindberg, Eva ^{[7
,8
]}

Fenaux, Pierre ^{[41
]}

Sanz, Guillermo ^{[6
,42
]}

Santini, Valeria ^{[2
,43
]}

机构：

[1] FPO IRCCS, Candiolo Canc Inst, Candiolo, Italy

[2] Fdn Sindromi Mielodisplast FISiM ETS, Bologna, Italy

[3] Hosp Univ & Politecn La Fe, Inst Invest Sanitaria La Fe, Valencia, Spain

[4] Heinrich Heine Univ Dusseldorf, Univ Klin Dusseldorf, Dept Hematol Oncol & Clin Immunol, Dusseldorf, Germany

[5] Hop Necker Enfants Malad, AP HP, Hematol Clin, Paris, France

[6] Univ Hosp Salamanca, Inst Biomed Res Salamanca IBSAL, Hematol, Salamanca, Spain

[7] Univ Helsinki, Hematol Res Unit Helsinki, Helsinki, Finland

[8] Helsinki Univ Hosp, Comprehens Canc Ctr, Helsinki, Finland

[9] Karolinska Univ Hosp, Dept Hematol M64, Stockholm, Sweden

[10] H Lee Moffitt Canc Ctr & Res Inst, Malignant Hematol Dept, Tampa, FL USA

[11] Univ Hosp Leipzig, Dept Hematol Cellular Therapy Hemostaseol & Infect, Leipzig, Germany

[12] Univ Med Ctr Gottingen, Clin Hematol & Med Oncol, Gottingen, Germany

[13] Heidelberg Univ, Univ Hosp Mannheim, Dept Hematol & Oncol, Heidelberg, Germany

[14] Osped Circolo & Fdn Macchi, Dipartimento Ematol, ASST Sette Laghi, Varese, Italy

[15] Hosp Son Llatzer, Hematol Dept, Mallorca, Spain

[16] Hosp Cent Asturias, Hematol Dept, Oviedo, Spain

[17] Hosp Clin Univ Salamanca, Hematol Dept, Salamanca, Spain

[18] Osp San Francesco Nuoro, UOC Ematol CTMO, Nuoro, Italy

[19] SOC Ematol Azienda USL IRCSS Reggio Emilia, Reggio Emilia, Italy

[20] Univ Hosp Citta Salute & Sci, Hematol, Turin, Italy

[21] Hosp Citta Salute & Sci, Hematol, Turin, Italy

[22] IRCCS Ctr Riferimento Oncol CRO Aviano Pordenone, Aviano, Italy

[23] Univ Hosp Parma, Hematol Unit, Parma, Italy

[24] Univ Hosp Parma, BMT Ctr, Parma, Italy

[25] IRCCS Azienda Osped Univ Bologna, Inst Hematol Seragnoli, Bologna, Italy

[26] Univ Sassari, Dept Clin & Expt Med, Sassari, Italy

[27] Helsinki Univ Cent Hosp, Dept Med, Helsinki, Finland

[28] Hosp Gemans Trias & Pujol, Hematol Dept, Badalona, Spain

[29] Osped Belcolle, Unita Operat Complessa UOC Ematol, Viterbo, Italy

[30] Sapienza Univ, Div Cellular Biotechnol & Hematol, Rome, Italy

[31] Hop Unversitario Virgen Nieves, Hematol Dept, Granada, Spain

[32] Hosp Univ Vall DHebron, Hematol Dept, Barcelona, Spain

[33] Hosp Cruces, Hematol Dept, Baracaldo, Spain

[34] Hosp Univ Reina Sofia, Hematol Dept, Cordoba, Spain

[35] IRCCS CROB, Referral Canc Ctr Basilicata, Rionero In Vulture, Italy

[36] Univ Politecn Marche AOU Marche, Hematol, Ancona, Italy

[37] ICO Duran & Reynals, Hematol Dept, Lhospitalet De LLobregat, Llobregat, Spain

[38] Hosp Miguel Servent, Valle Recasens Hematol Dept, Zaragoza, Spain

[39] Univ Luzern, Luzerner Kantonsspital, Div Hematol, Luzern, Switzerland

[40] Univ Genoa, Dept Hlth Sci, Sect Biostat, Genoa, Italy

[41] Hop St Louis, AP HP, Serv Hematol Seniors, Paris, France

[42] ISCIII, CIBERONC, Madrid, Spain

[43] Univ Florence, MDS Unit, Hematol, DMSC,AOU Careggi, Florence, Italy

来源：

LEUKEMIA | 2024年 / 38卷 / 10期

基金：

欧盟地平线“2020”;

关键词：

WORLD-HEALTH-ORGANIZATION; DEPENDENT PATIENTS; MYELOID NEOPLASMS; AML PROGRESSION; TP53; MUTATIONS; SCORING SYSTEM; RISK; MDS; MULTICENTER; LEUKEMIA;

D O I：

10.1038/s41375-024-02360-1

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

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.

引用

页码：2259 / 2265

页数：7

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