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. 2022 Nov 17;140(20):2170-2174.
doi: 10.1182/blood.2022016171.

Longer-term benefit of luspatercept in transfusion-dependent lower-risk myelodysplastic syndromes with ring sideroblasts

Amer M Zeidan  1 , Uwe Platzbecker  2 , Guillermo Garcia-Manero  3 , Mikkael A Sekeres  4 , Pierre Fenaux  5 , Amy E DeZern  6 , Peter L Greenberg  7 , Michael R Savona  8 , Joseph G Jurcic  9 , Amit K Verma  10 , Ghulam J Mufti  11 , Rena Buckstein  12 , Valeria Santini  13 , Jeevan K Shetty  14 , Rodrigo Ito  15 , Jennie Zhang  15 , George Zhang  15 , Xianwei Ha  15 , Jay T Backstrom  16 , Rami S Komrokji  17
Affiliations

Longer-term benefit of luspatercept in transfusion-dependent lower-risk myelodysplastic syndromes with ring sideroblasts

Amer M Zeidan et al. Blood. .

Abstract

Luspatercept is an approved therapy for selected patients with lower risk myelodysplasia requiring transfusion despite erythropoiesis-stimulating agents, based on the early results of a randomized trial against placebo. Zeidan and colleagues report that after a median of 26 months follow-up, 27% of patients commencing luspatercept were continuing therapy. Their updated analyses confirm that a significant minority (45%) of eligible patients can achieve transfusion independence, with a median durability of 30 weeks. These longer follow-up data better quantify the incremental benefit of luspatercept over placebo.

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Conflict of interest statement

Conflict-of-interest disclosure: A.M.Z. has received grant support, consulting fees, honoraria, and fees for serving on a clinical trial committee from AbbVie, Bristol Myers Squibb, and Novartis; consulting fees and honoraria from Acceleron Pharma, Agios, Astellas, BeyondSpring, Cardinal Health, Daiichi Sankyo, Janssen, Jazz Pharmaceuticals, Seagen, Syndax, Taiho, and TYME; grant support from ADC Therapeutics, Astex Pharmaceuticals, and MedImmune/AstraZeneca; grant support, consulting fees, and honoraria from Amgen, Aprea, Boehringer Ingelheim, Cardiff Oncology, Incyte, Otsuka, Pfizer, Takeda, and Trovagene; consulting fees, honoraria, and fees for serving on a clinical trial committee from Gilead and Kura; and fees for serving on a clinical trial committee from Geron. U.P. has received grant support, consulting fees, and honoraria from Bristol Myers Squibb and Celgene, a Bristol-Myers Squibb Company; grant support and honoraria from Janssen and Novartis; grant support from Amgen and Merck; and honoraria from Abbvie, Geron, and Takeda. G.G-M. has received grant support and consulting fees from Astex Pharmaceuticals, Bristol Myers Squibb, Genentech, and Helsinn Healthcare; and grant support from AbbVie, Amphivena Therapeutics, Aprea, Bristol Myers Squibb, Curis, Forty Seven, H3 Biomedicine, Janssen, Merck, Novartis, and Onconova Therapeutics. M.A.S. has served on advisory boards for Bristol Myers Squibb, Gilead Sciences, Novartis, and Pfizer. P.F. has received consulting fees from Celgene, a Bristol-Myers Squibb Company. A.E.D. has received honoraria from Bristol Myers Squibb and Novartis; and consulting fees from Taiho and Takeda. P.L.G. has received grant support (paid to Stanford University) from Bristol Myers Squibb. M.R.S. has received fees for serving on a steering committee and fees for serving on a data and safety monitoring board from Bristol Myers Squibb, Geron, Ryvu, Sierra Oncology; consultancy and advisory fees for AbbVie, CTI BioPharma, Karyopharm, Novartis, Ryvu, Taiho, Takeda, and TG Therapeutics; grant funding from ALX Oncology, Astex, Incyte, Takeda, and TG Therapeutics; and holds equity in Karyopharm and Ryvu. J.G.J. has received grant support (paid to Columbia University), advisory board fees, and travel support from AbbVie; grant support (paid to Columbia University) from Arog Pharmaceuticals, Astellas, Forma Therapeutics, Genentech, Gilead Sciences, PTC Therapeutics, and Syros Pharmaceuticals; advisory board fees from AstraZeneca; grant support (paid to Columbia University), advisory board fees, and travel support from Bristol Myers Squibb; grant support (paid to Columbia University) and consulting fees from Daiichi Sankyo; and fees for serving on an endpoint committee from Novartis. A.K.V. has provided consultancy for and received research funding from Bristol Myers Squibb; provided consultancy for and received honoraria from Acceleron Pharma; research funding from Janssen and MedPacto; and currently holds equity in Stelexis (a private company). G.J.M. has received support from Bloodwise UK grants 10024 and 14017, Cancer Research UK grant A22324, Celgene, a Bristol-Myers Squibb Company, and Novartis. R.B. has received honoraria, consulting fees, and research funding from Bristol Myers Squibb; honoraria, consulting fees, and research funding TAIHO; and research funding from Takeda. V.S. has received advisory board fees and lecture fees from Celgene, a Bristol Myers Squibb Company; travel support from Janssen Biotech; advisory board fees from Geron, Gilead, Menarini, Novartis, and Takeda Oncology; and grant support, paid to the University of Florence, from Celgene. J.K.S. reports current employment at and currently holding equity in Bristol Myers Squibb (publicly traded company). R.I. reports former employment at and currently holding equity in Bristol Myers Squibb (publicly traded company); current employment at and currently holding equity in Eli Lilly and Company (publicly traded company). J.Z., G.Z., and X.H. report current employment at Bristol Myers Squibb. J.T.B. reports currently holding equity in Bristol Myers Squibb (publicly traded company); and current employment at and currently holding equity in Acceleron Pharma (publicly traded company). R.S.K. has received advisory board fees from AbbVie, Acceleron Pharma, Bristol Myers Squibb, Geron, Jazz Pharmaceuticals, and Novartis; and fees for serving on a speakers’ bureau from Bristol Myers Squibb and Jazz Pharmaceuticals.

Figures

Figure 1.
Figure 1.
Patient baseline characteristics, RBC-TI ≥8 weeks and mHI-E response. Baseline characteristics of patients in the MEDALIST trial by transfusion burden (A). Rates of RBC-TI for ≥8 weeks and mHI-E response during weeks 1 to 48, overall and by transfusion burden (B). Time to first response and duration of RBC-TI ≥8 weeks and mHI-E response (C). mHI-E response was defined according to IWG 2006 criteria as a mean hemoglobin increase ≥1.5 g/dL among patients with a baseline RBC transfusion burden <4 units per 8 weeks or a reduction of ≥4 RBC units among patients with baseline RBC transfusion burden ≥4 units per 8 weeks, sustained over a consecutive 56-day period. ∗Last value measured on or before the date and time of the first dose of luspatercept per placebo. †Highest value within 35 days before the first dose of luspatercept per placebo. ‡Only 2 patients with HTB who received placebo achieved RBC-TI; therefore, a median duration could not be reliably estimated. IWG, International Working Group; mHI-E, modified hematologic improvement-erythroid; SD, standard deviation; sEPO, serum erythropoietin; SF3B1, splicing factor 3b subunit 1.
Figure 2.
Figure 2.
Transfusion burden reduction, TEAEs, and RBC-TI ≥16 weeks. Rates of ≥50% and ≥75% reduction in RBC transfusion burden from baseline over ≥24 weeks during the entire treatment phase, overall, and by transfusion burden (A). Summary of TEAEs during the entire treatment period (B). Rates of RBC-TI ≥16 weeks during weeks 1 to 48. Data are n (%). Transfusion events and TEAEs are reported during weeks 1 to 48. ∗Those occurring in ≥10% in any group. CI, confidence interval; TEAE, treatment emergent.
See this image and copyright information in PMC

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