Table 1.

Clinical studies using agents that interfere with TGF-β signaling in MDS and β-thalassemia. * p < 0.001.

	Drug	Patients	Primary Endpoint	Outcomes	Ref
MDS	Sotatercept	Total = 74	Hematological response (HI-E)	49%	[36]
	Luspatercept	Total = 58	Hematological response (HI-E)	63%	[37]
	Luspatercept	Total = 229	Transfusion independence for ≥8 weeks during weeks 1–24		[38]
		Drug = 153 Placebo = 76		$\begin{array}{l}38\%\\ 13\%\end{array}\}$ *
β-thalassemia	Luspatercept	Total = 64			[39]
		NTDT = 33	Mean hemoglobin increase ≥1.5 g/dL for 14 days	58%
		TDT = 31	Transfusion burden reduction	81%
	Luspatercept	Total = 336	Reduction in the transfusion burden during 13–24 weeks		[40]
		Drug = 224 Placebo = 112		$\begin{array}{c}21.4\%\\ 4.5\%\end{array}\}$ *