A Novel Insight Into Luspatercept: Therapeutic Implications in Myelodysplastic Syndrome Patients With Elevated Pulmonary Artery Systolic Pressure.
4 affiliations
- 1Department of Cardiology, Zhongshan Hospital, Fudan University Shanghai Institute of Cardiovascular Diseases Shanghai China.
- 2State key Laboratory of Cardiovascular Diseases, Zhongshan Hospital Fudan University Shanghai China.
- 3National Clinical Research Center for Interventional Medicine Shanghai China.
- 4Department of Pharmacy, Zhongshan Hospital Fudan University Shanghai China.
Abstract
Luspatercept enhances late-stage erythroid maturation by sequestering transforming growth factor-β superfamily ligands and attenuating pathological signaling, with a mechanism highly analogous to that of sotatercept, a breakthrough therapeutic agent for pulmonary hypertension (PH). Despite this solid mechanistic basis, the potential vascular and prognostic effects of luspatercept in PH patients remain largely uncharacterized. This retrospective proof-of-concept study aimed to evaluate the impact of luspatercept on pulmonary artery pressure and surrogate prognostic markers, and to explore its novel therapeutic potential in patients with increased pulmonary artery systolic pressure (PASP). We retrospectively analyzed serial echocardiographic parameters among patients with myelodysplastic syndrome (MDS) who received luspatercept treatment, focusing on individuals with elevated baseline PASP. Dynamic changes in cardiopulmonary indicators and laboratory biomarkers were comprehensively compared before and after intervention. A total of five MDS patients with elevated baseline PASP were enrolled in the final analysis. Although formal statistical comparisons failed to reach significance, which was largely attributable to the limited sample size, consistent clinically beneficial improvements were observed after luspatercept treatment. The median PASP decreased from 62.0 (IQR 14) mmHg at baseline to 33.0 (IQR 18.5) mmHg following treatment. Parallel reductions in N-terminal pro-brain natriuretic peptide (NT-proBNP) and uric acid levels were identified, accompanied by a mild yet favorable elevation in hemoglobin concentration. In this small cohort of MDS patients with elevated PASP, luspatercept treatment was associated with decreased pulmonary artery pressure, reduced NT-proBNP and uric acid levels, and improved hemoglobin levels. These preliminary findings suggest novel pleiotropic benefits of luspatercept in improving pulmonary arterial pressure, cardiac function, and anemia status, providing a compelling rationale for further prospective clinical studies to verify its therapeutic value in PH populations.
Related
- Luspatercept in myelodysplastic syndrome with ring sideroblasts: a case report of intrapulmonary vascular dilatation syndrome causing protracted inexplicable hypoxia
- Pulmonary Leukocytoclastic Vasculitis Associated with Myelodysplastic Syndrome
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- LUSPATERCEPT INITIATED AT THE MAXIMUM-APPROVED DOSE IN PATIENTS WITH LOWER-RISK MYELODYSPLASTIC SYNDROMES WHO REQUIRE TRANSFUSIONS: PRIMARY ANALYSIS FROM MAXILUS
- Exerkines in Cardiovascular–Kidney–Metabolic Syndrome: An Integrative Framework for Mechanisms and Therapeutic Implications—A Narrative Review
BibTeX
@article{jin2026novel,
title = {{A Novel Insight Into Luspatercept: Therapeutic Implications in Myelodysplastic Syndrome Patients With Elevated Pulmonary Artery Systolic Pressure.}},
author = {Qi Jin and Dan Tian and Dandan Chen and Mingfei Li and Shiqiang Hou and Xiaochun Zhang and Wenzhi Pan and Daxin Zhou and Lihua Guan and Junbo Ge},
journal = {Pulmonary circulation},
year = {2026},
month = oct,
doi = {10.1002/pul2.70405},
url = {https://europepmc.org/article/MED/42948081},
note = {CC BY-NC},
}
RIS
TY - JOUR
TI - A Novel Insight Into Luspatercept: Therapeutic Implications in Myelodysplastic Syndrome Patients With Elevated Pulmonary Artery Systolic Pressure.
AU - Qi Jin
AU - Dan Tian
AU - Dandan Chen
AU - Mingfei Li
AU - Shiqiang Hou
AU - Xiaochun Zhang
AU - Wenzhi Pan
AU - Daxin Zhou
AU - Lihua Guan
AU - Junbo Ge
PY - 2026
DA - 2026/10/27
JO - Pulmonary circulation
DO - 10.1002/pul2.70405
UR - https://europepmc.org/article/MED/42948081
AB - Luspatercept enhances late-stage erythroid maturation by sequestering transforming growth factor-β superfamily ligands and attenuating pathological signaling, with a mechanism highly analogous to that of sotatercept, a breakthrough therapeutic agent for pulmonary hypertension (PH). Despite this solid mechanistic basis, the potential vascular and prognostic effects of luspatercept in PH patients remain largely uncharacterized. This retrospective proof-of-concept study aimed to evaluate the impact of luspatercept on pulmonary artery pressure and surrogate prognostic markers, and to explore its novel therapeutic potential in patients with increased pulmonary artery systolic pressure (PASP). We retrospectively analyzed serial echocardiographic parameters among patients with myelodysplastic syndrome (MDS) who received luspatercept treatment, focusing on individuals with elevated baseline PASP. Dynamic changes in cardiopulmonary indicators and laboratory biomarkers were comprehensively compared before and after intervention. A total of five MDS patients with elevated baseline PASP were enrolled in the final analysis. Although formal statistical comparisons failed to reach significance, which was largely attributable to the limited sample size, consistent clinically beneficial improvements were observed after luspatercept treatment. The median PASP decreased from 62.0 (IQR 14) mmHg at baseline to 33.0 (IQR 18.5) mmHg following treatment. Parallel reductions in N-terminal pro-brain natriuretic peptide (NT-proBNP) and uric acid levels were identified, accompanied by a mild yet favorable elevation in hemoglobin concentration. In this small cohort of MDS patients with elevated PASP, luspatercept treatment was associated with decreased pulmonary artery pressure, reduced NT-proBNP and uric acid levels, and improved hemoglobin levels. These preliminary findings suggest novel pleiotropic benefits of luspatercept in improving pulmonary arterial pressure, cardiac function, and anemia status, providing a compelling rationale for further prospective clinical studies to verify its therapeutic value in PH populations.
SN - 2045-8932
SN - 2045-8940
ER -