Table 4.

Summary of case report results and adverse events.

Citation	Patient case reports included	Mean follow-up time	% pts survived	% able to complete treatment, once started	Among those who completed treatment, % who are symptom free from SCD	% patients who became transfusion independent	# of patients with severe AEs (list)
NCT02151526	1	15 months	1	1	1	1	1 grade 4 neutropenia grade 3 anemia grade 3 thrombocytopenia grade 3 infection with Staphylococcus epidermidis (with positive results on blood culture)
NCT03745287	1	16.6 months	1	1	1	1	1 sepsis in the presence of neutropenia cholelithiasis abdominal pain
NCT03282656	6	18 months	6	6	6	5	3 hospital admission for less than 24 hours for fever and influenza infection recurrent priapism leading to two hospital admissions leg pain leading to a readmission for less than 24 hours
NCT02140554	2		2	2			2 myelodysplastic syndrome acute myeloid leukemia
NCT04293185	1		1	1			1 myelodysplastic syndrome