Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2023 Nov;37(11):2314-2318.
doi: 10.1038/s41375-023-02031-7. Epub 2023 Sep 26.

Long-term utilization and benefit of luspatercept in transfusion-dependent, erythropoiesis-stimulating agent-refractory or -intolerant patients with lower-risk myelodysplastic syndromes with ring sideroblasts

Uwe Platzbecker  1 , Valeria Santini  2 , Rami S Komrokji  3 , Amer M Zeidan  4 , Guillermo Garcia-Manero  5 , Rena Buckstein  6 , Dimana Miteva  7 , Karen Keeperman  8 , Natalia Holot  8 , Jose Alberto Nadal  7 , Yinzhi Lai  8 , Sadanand Vodala  8 , Barbara Rosettani  7 , Ana Carolina Giuseppi  8 , Aylin Yucel  8 , Pierre Fenaux  9
Affiliations

Long-term utilization and benefit of luspatercept in transfusion-dependent, erythropoiesis-stimulating agent-refractory or -intolerant patients with lower-risk myelodysplastic syndromes with ring sideroblasts

Uwe Platzbecker et al. Leukemia. 2023 Nov.
No abstract available

PubMed Disclaimer

Conflict of interest statement

UP has received grant support from Amgen, Janssen, Merck, and Novartis; grant and travel support, and lecture, steering committee, and consulting fees, and travel support from Celgene/BMS; and lecture fees from Novartis. VS has received honoraria and advisory board fees from BMS, travel support from Janssen, and advisory board fees from AbbVie, Geron, Gilead, Menarini, Novartis, Servier Pharmaceuticals, and Syros. RSK has received consulting and advisory board fees from Geron; speaker and advisory board fees from AbbVie, CTI BioPharma, Jazz Pharmaceuticals, PharmaEssentia, and Servier Pharmaceuticals; advisory board fees from Novartis, Rigel Pharmaceuticals, and Taiho Pharma; and funding and advisory board fees from BMS. AMZ has received consulting fees, research funding, honoraria, and travel support from Cardiff Oncology, Novartis, and Pfizer; consulting fees, research funding, and honoraria from AbbVie, Amgen, Aprea, Boehringer-Ingelheim, Celgene/BMS, Incyte, Otsuka, Takeda, and Trovagene; consulting fees, and honoraria from Acceleron Pharma, Agios, Astellas, BeyondSpring, Cardinal Health, Daiichi Sankyo, Epizyme, Geron, Gilead, Ionis Pharmaceuticals, Janssen, Jazz Pharmaceuticals, Kura Oncology, Seattle Genetics, Syndax, Taiho Pharma, Tyme; research funding from ADC Therapeutics, Astex Pharmaceuticals, AstraZeneca, and Medimmune. GG-M has received research funding from BMS. RB has received research funding and honoraria from BMS, and Taiho Pharma, and honoraria from AbbVie. DM, NH, BR, and AY are currently employed by BMS. JAN is currently employed by BMS, is a current holder of individual stocks in a privately held company, and a current holder of BMS stock options. KK is currently employed by, is a current holder of BMS stock options, and is a current holder of Pfizer stock options. YL, ACG, and SV are currently employed by, and current holders of BMS stock options. PF has received consulting fees, research funding, and honoraria from BMS.

Figures

Fig. 1
Fig. 1. Duration of treatment, cumulative duration of RBC-TI ≥8 weeks response, and rates of achievement of RBC-TI ≥8 weeks and ≥16 weeks, and mean Hb increase ≥1.5 g/dl.
A Duration of treatment during the entire treatment period. B Rate of achievement of RBC-TI ≥8 weeks and ≥16 weeks, and mean Hb increase ≥1.5 g/dl during the entire treatment period. C Cumulative duration of all periods of RBC-TI response ≥8 weeks at any time during the study. aTreatment end date is defined as the last dose date +20 days, study discontinuation date, cutoff date or death date, whichever is earlier. bResponse is defined as the absence of any RBC transfusion during any consecutive 56-day period during the entire treatment period. cResponse rate (%) was calculated using the number of responders divided by the number of patients multiplied by 100. dCochran-Mantel-Haenszel test stratified for average baseline RBC transfusion requirement (≥6 units vs. <6 units of RBC per 8 weeks) and baseline IPSS-R score (Very low or Low vs. Intermediate). eResponse is defined as the absence of any RBC transfusion during any consecutive 112-day period during the entire treatment period. fDefined as the proportion of patients with a Hb increase ≥1.5 g/dl compared with baseline that was sustained over any consecutive 56-day period in the absence of RBC transfusions. gFisher exact test comparing the luspatercept arm with the placebo arm. hThe cumulative duration of all periods of RBC-TI ≥8 weeks is defined as the sum of all durations of non-consecutive periods of RBC-TI response ≥8 weeks for patients during the entire treatment phase. iMedian is from the Kaplan–Meier method. CI confidence interval, Hb hemoglobin, HR hazard ratio, IPSS-R Revised International Prognostic Scoring System, NE not estimable, RBC red blood cell, RBC-TI RBC transfusion independence.
Fig. 2
Fig. 2. OS by RBC-TI ≥8 weeks response, RBC-TI ≥16 weeks response, and mean Hb increase ≥1.5 g/dl response.
A Summary of OS. B OS by RBC-TI ≥8 weeks response during weeks 1–24. C OS by RBC-TI ≥16 weeks response during weeks 1–24. D OS by mean Hb increase ≥1.5 g/dL response during weeks 1–24. OS is calculated as the time from randomization date to death of any cause and is censored at the last date that the patient was known to be alive for patients who were alive at the time of analysis and for patients who discontinued from the study or were lost to follow-up. aUnstratified HR and log-rank test p value. CI confidence interval, Hb hemoglobin, HR hazard ratio, NA not applicable, NE not estimable, OS overall survival, RBC-TI red blood cell transfusion independence, SD standard deviation.
See this image and copyright information in PMC

References

    1. Fenaux P, Platzbecker U, Ades L. How we manage adults with myelodysplastic syndrome. Br J Haematol. 2020;189:1016–27. doi: 10.1111/bjh.16206. - DOI - PubMed
    1. Platzbecker U. Treatment of MDS. Blood. 2019;133:1096–107. doi: 10.1182/blood-2018-10-844696. - DOI - PubMed
    1. Malcovati L, Della Porta MG, Cazzola M. Predicting survival and leukemic evolution in patients with myelodysplastic syndrome. Haematologica. 2006;91:1588–90. - PubMed
    1. Oliva EN, Schey C, Hutchings AS. A review of anemia as a cardiovascular risk factor in patients with myelodysplastic syndromes. Am J Blood Res. 2011;1:160–6. - PMC - PubMed
    1. Carraway HE, Saygin C. Therapy for lower-risk MDS. Hematology Am Soc Hematol Educ Program. 2020;2020:426–33.. doi: 10.1182/hematology.2020000127. - DOI - PMC - PubMed

Publication types

MeSH terms