Progress towards Mechanism-Based Treatment for Diamond-Blackfan Anemia

Table 1

Compounds clinically tested as new DBA treatments and their respective outcome.

Therapy	Patients (𝑛)	Positive responders	Comments	Ref.
Cyclosporine A	19	22–50%	Good long-term response for some, but with side effects	[48–50]
Deferasirox	1	100%	Similar effect observed in MDS patients	[51]
Erythropoietin	10	0%	No response	[52, 53]
Interleukin 3	92	12–22%	Only partial response in some patients. Some severe side effects	[54–57]
IV IgG	2	0%	Brief or no response	[58, 59]
Metoclopramide	42	6–33%	Two studies with contradicting results	[60, 61]
Valproic acid	1	100%	Patient in sustained remission	[62]