Table 2. Summary of the clinical... | American Society of Hematology

Table 2.

Summary of the clinical experience of hematopoietic stem cell (HSC)-gene therapy for adenosine deaminase-deficient severe combined immunodeficiencies (ADA-SCID) in the last decade.

Study	No. pts treated	Conditioning	Longest follow-up	Efficacy	Toxicity
GOSH indicates Great Ormond Street Hospital; CHLA, Children’s Hospital Los Angeles.
HSR-TIGET¹¹^,¹⁴	15	Busulfan (4 mg/kg)	8 y	Yes	No
GOSH¹¹^,¹⁹	5	Melphalan (140 mg/m²)	5.5 y	Yes	No
CHLA/NIH¹⁷	4	No	8 y	No	No
CHLA/NIH¹¹^,¹⁷^,¹⁸	6	Busulfan (75–90 mg/m²)	2 y	Yes	Pancytopenia due to pre-existing cytogenetic abnormality (1 pt)

Study	No. pts treated	Conditioning	Longest follow-up	Efficacy	Toxicity
GOSH indicates Great Ormond Street Hospital; CHLA, Children’s Hospital Los Angeles.
HSR-TIGET¹¹^,¹⁴	15	Busulfan (4 mg/kg)	8 y	Yes	No
GOSH¹¹^,¹⁹	5	Melphalan (140 mg/m²)	5.5 y	Yes	No
CHLA/NIH¹⁷	4	No	8 y	No	No
CHLA/NIH¹¹^,¹⁷^,¹⁸	6	Busulfan (75–90 mg/m²)	2 y	Yes	Pancytopenia due to pre-existing cytogenetic abnormality (1 pt)