Table 2.

HSCT for Patients with GATA2 Deficiency 14 jan

Donor	Pt	Donor Source	HLA Match	BU AUC	BU dose/d # of days	CD34+/kg	CD3+/kg	Myeloid chimerism Day 100	CD3+ chimerism Day 100	Time after Transplant (months)	GVHD prophylaxis	aGVHD	cGVHD	Current Status
MRD
	1 RM	PBSC	10/10	4800	15.8 mg/kg 4	7.67 x 106/kg	1.3 x 108/kg	99	90	45	Tacro/MTX	Grade 1 skin	None	Alive, off Immunosupp.
	2 AV	BM	10/10	4800	16 mg/kg 4	6.75 x 106/kg	4.9 x 107/kg	99	29	24	Tacro/MTX	Grade 3 GI	Vaginal	Alive, off immunosupp.
URD
	1 SS	PBSC	10/10	4800	14.6 mg/kg 4	8 x 106/kg	1.87 x 108/kg	100	83	43	Tacro/MTX	Grade 2 skin and GI	Moderate/severe cGVHD	Alive, on Immunosupp
	2 AT	PBSC	10/10	4800	15.8 mg/kg 4	5.0 x 106/kg	4.79 x 107/kg	100	100	24	Tacro/MTX	Grade 4 3 system	Moderate/severe cGVHD	Died, GVHD, 24 mo.
	3 EB	PBSC	10/10	4800	13.6 mg/kg 4	4.45 x 106/kg	2.13 x 108/kg	42	98	6	Tacro/MTX	None	None	Died, ref AML
	4 JD	PBSC	10/10	4200	11.6 mg/kg 4	6.39 x 106/kg	1.45 x 108/kg	100	89	21	Tacro/MTX	None	Eye, mouth cGVHD	Alive, on Immunosupp
	5 AK	BM	10/10	4137	10.3 mg/kg 4	1.33 x 106/kg	2.5 x 107/kg	99	91	20	Tacro/MTX	None	None	Alive, off Immunosupp
	6 CS	BM	10/10	4800	13.8 mg/kg 4	3.19 x 106/kg	2.1 x 107/kg	100	93	18	Tacro/MTX	No	Moderate/severe cGVHD	Alive, on Immunosupp
	7 MM	PBSC	10/10	4600	12.8 mg/kg 4	6.02 x 106/kg	1.79 x 108/kg	100	100	16	Tacro/MTX	Grade 2 colon	Skin	Alive, on Immunosupp
	8 EG	BM	10/10	4400	11.6 mg/kg 4	5.24 x 106/kg	3.2 x 107/kg	100	97	15	Tacro/MTX	Grade 3 skin	Skin	Alive, on Immunosupp
	9 JM	BM	10/10	4000	12 mg/kg 4	2.67 x 106/kg	2.48 x 107/kg	100	56	14	Tacro/MTX	Grade 3 skin	Skin	Alive, on Immunosupp
	10 AP	BM	10/10	3900	12.8 mg/kg 4	1.86 x 106/kg	3.0 x 107/kg	100	98	8	Tacro/MTX	Grade 3 skin and GI	GI	Died, sepsis 8 mo
	11 SC	PBSC	10/10	4800	13.2 mg/kg 4	8.69 x 106/kg	4.51 x 107/kg	100	71	12	Tacro/MTX	Grade 3 3-system	Skin/GI	Alive, on Immunosupp
	12 SD	BM	10/10	4300	12 mg/kg 4	1.5 x 106/kg	2.16 x 107/kg	100	100	6	Tacro/MTX	None	None	Alive, on immunosupp
	13 MB	PBSC	10/10	4200	10 mg/kg 4	7 x 106/kg	1.57 x 108/kg	82	100	3	Tacro/MTX	None	None	Alive, on immunosupp
Haplo
	1 JP	BM	5/10	4800	7.0 mg/kg 2	2.5 x 106/kg	4.4 x 107/kg	100	99	38	PT/CY	Grade 1 skin	None	Alive, off Immunosupp
	2 KM	BM	8/10	4800	6.6 mg/kg 2	5.5 x 106/kg	5.3 x 107/kg	100	100	30	PT/CY	Grade 1 skin Grade 2 GI	None	Alive, off Immunosupp
	3 LM	BM	8/10	4700	6.3 mg/kg 2	6.1 x 106/kg	5.9 x 107/kg	100	100	25	PT/CY	Grade 2 GI	Vaginal	Alive, off Immunosupp
	4 AT	BM	5/10	3800	7.4 mg/kg 2	6.1 x 106/kg	3.5 x 107/kg	100	100	22	PT/CY	None	None	Alive, off Immunosupp
	5 WSA	BM	6/10	4400	6.8 mg/kg 2	2.6 x 106/kg	3.2 x 107/kg	100	100	18	PT/CY	Grade 3 GI	Moderate/severe cGVHD	Alive, on Immunosupp
	6 GC	BM	8/10	4000	9.3 mg/kg 2	5.9 x 106/kg	3.0 x 107/kg	100	100	14	PT/CY	Grade 1 skin	None	Alive, off Immunosupp
	7 NLO	BM	5/10	4400	6.6 mg/kg 3	7.2 x 106/kg	5.2 x 107/kg	100	100	12	PT/CY	Grade 2 upper GI	None	Alive, off Immunosupp

Abbreviations: MRD, matched related donor; URD, matched unrelated donor; Haplo, haploidentical related donor; PBSC, peripheral blood stem cells; BM, bone marrow; Pt, patient; HLA, human leukocyte antigen; BU, busulfan; AUC, area under curve; aGVHD, acute graft-versus-host disease; cGVHD, chronic graft-versus-host disease; Tacro, tacrolimus, MTX, methotrexate; PT/CY, post-transplant cyclophosphamide;