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3d
‌Clinical Observation of Oral Pirfenidone in the Treatment of Stiff skin syndrome (ChiCTR2600117147)
P=N/A, N=10, Not yet recruiting, Beijing Childrens Hospital,Capital Medical University; Beijing Childrens Hospital,Capital Medical University
New trial
3d
Luspatercept for Refractory Poor Platelet Engraftment after Allogeneic Hematopoietic Stem Cell Transplantation: A Single-Center, Single-Arm, Prospective Clinical Study (ChiCTR2500111793)
P2, N=20, Not yet recruiting, The First Affiliated Hospital of Zhejiang Chinese Medical University; The First Affiliated Hospital of Zhejiang Chinese Medical University
New P2 trial
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Reblozyl (luspatercept-aamt)
11d
New P1/2 trial
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Reblozyl (luspatercept-aamt)
11d
New P1/2 trial
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Winrevair (sotatercept-csrk)
11d
Erythropoiesis in health and disease: Distinguishing defective and ineffective erythropoiesis. (PubMed, Hemasphere)
Advances in therapy now directly target these mechanisms: Luspatercept, a TGF-β ligand trap, has transformed care in β-thalassemia and MDS by restoring late-stage differentiation, while anti-inflammatory strategies, metabolic modulators, and gene editing approaches are being actively explored. Together, these discoveries reframe IE as a modifiable process rather than an inevitable consequence of disease. By distinguishing defective from IE and mapping their mechanistic underpinnings, this review outlines how novel therapeutic strategies can improve anemia, reduce systemic complications, and ultimately enhance outcomes in patients with erythroid disorders.
Review • Journal
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TGFB1 (Transforming Growth Factor Beta 1) • GATA1 (GATA Binding Protein 1)
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Reblozyl (luspatercept-aamt)
13d
Enrollment closed • Enrollment change
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Winrevair (sotatercept-csrk)
15d
Impact of Mutational Landscape and Burden on RBC Transfusion Response in Patients With Lower-Risk Myelodysplastic Syndromes (LR-MDS) in the COMMANDS Study. (PubMed, Am J Hematol)
Here we report red blood cell (RBC) transfusion response analysis based on somatic mutations profile and disease risk for patients treated with luspatercept or epoetin alfa in the COMMANDS trial. Luspatercept represents an effective treatment option in various mutational backgrounds in LR MDS. Trial Registration: ClinicalTrials.gov Identifier: NCT03682536.
Journal
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SF3B1 (Splicing Factor 3b Subunit 1)
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SF3B1 mutation
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Reblozyl (luspatercept-aamt)
19d
A Study of Sotatercept in Japanese Pulmonary Arterial Hypertension (PAH) Participants (MK-7962-020) (clinicaltrials.gov)
P3, N=46, Completed, Merck Sharp & Dohme LLC | Active, not recruiting --> Completed | Trial completion date: Feb 2026 --> Nov 2025
Trial completion • Trial completion date
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Winrevair (sotatercept-csrk)