Table 2.

Transplantation details, outcome, and clinical status post-HSCT. Key: AIHA, autoimmune hemolytic anemia; AUC, area under the curve; BM, bone marrow; CsA, ciclosporin A; MMF, mycophenolate mofetil; MSD, matched sibling donor; MUD, matched unrelated donor; PBSC, peripheral blood stem cell; PTCy, post-transplant cyclophosphamide

Patient	Age at HSCT (years)	Stem cell source and HLA match	Conditioning regimen Serotherapy	GvHD prophylaxis	GvHD and treatment	Follow-up duration (years)	Latest donor chimerism	Latest %DHR +	Outcome	Post-HSCT infection	Post-HSCT inflammation or autoimmunity
P1	15	10/10 MUD-PBSC	Treosulfan 42 g/m2 Fludarabine 150 mg/m2 Thiotepa 10 mg/kg Alemtuzumab 1 mg/kg	CsA/MMF	-	4	100%	100%	Survival	None	AIHA — resolved Rash and arthralgia in remission
P2	18	10/10 MUD-PBSC	Treosulfan 42 g/m2 Fludarabine 150 mg/m2 Thiotepa 10 mg/kg Alemtuzumab 1 mg/kg	CsA/MMF	-	-	100%	100%	Death D + 30 — toxic leukoencephalopathy from conditioning agents	-	-
P3	20	10/10 MUD-BM	Busulfan (AUC: 44 mg/L*h) Fludarabine 180 mg/m2 Alemtuzumab 0.6 mg/kg	CsA/MMF	Grade I skin — topical steroids	8	100%	100%	Survival	None	Clinical and histological remission from colitis
P4	56	10/10 MSD-PBSC	Busulfan (AUC: 49 mg/L*h) Alemtuzumab 1 mg/kg	PTCy Sirolimus	Grade I skin — topical steroids	3	60%	60%	Survival	None	Clinical remission from colitis and lupus Hypothyroidism continues
P5	22	Haplo-PBSC	Busulfan (AUC: 49 mg/L*h) Fludarabine 150 mg/m2 Cyclophosphamide 29 mg/kg	PTCy Sirolimus	Grade 3 GI/liver — corticosteroids, infliximab	-	100%	98%	Death D + 100 — multiorgan failure from disseminated adenovirus and GvHD	-	-
P6	7	10/10 MSD-BM	Busulfan (AUC: 91 mg/L*h) Fludarabine 160 mg/m2 Alemtuzumab 0.6 mg/kg	CsA	-	2	100%	100%	Survival	None	None
P7	1	TCRαβ/CD19-depleted 9/10 MUD-PBSC (single C-antigen HLA mismatch)	Busulfan (AUC: 85 mg/L*h) Fludarabine 150 mg/m2 Alemtuzumab 0.6 mg/kg	CsA/MMF	-	3	100%	100%	Survival	None	Remission of colitis