Table 4. Description of patients with... | Rockefeller University Press

Table 4.

Description of patients with autoimmunity after transplant

	P7	P8	P12		P15	P17	P20	P21		P29	P30	P31	P36	P37	P38	P41
CGD	gp91	gp91	p67		gp91	gp91	p47	gp91		gp91	gp91	gp91	p47	gp91	p47	p22
Clinical presentation at diagnosis	Liver and skin abscess CGD colitis Extrinsic allergic alveolitis	Invasive Aspergillosis BCG-osis	CGD colitis Osteomyelitis		CGD colitis Urethral granuloma	Lymphadenitis Pancreatitis Liver abscess Pneumonia Sepsis ITP	Skin and perianal abscesses Recurrent URTI/OMA Asthma	Recurrent LRTI		Colitis Perianal disease Lymphadenitis	Perianal disease Recurrent LRTI	Neonatal rash Recurrent LRTI Diarrhea Gastric obstruction Short stature	Liver abscess Fungal chest infection	Fungal chest infection	Skin granulomas Pseudomonas infection Rothia bacteremia Ground glass change	Colitis Liver abscess Lymphadenopathy Atypical mycobacteria infection
Age at HSCT (years)	3.4	0.9	3.1 (first)	4.4 (second)	5.4	6.3	8.7	4.4 (first)	8.2 (second)	12.4	10.6	2.2	12.3	1.5	5.4	13.2
Donor	MMUD	MUD	MUD	MMUD	MSD	MMUD	MUD	MUD	MUD	MUD	MMUD	MMUD	MSD	MUD	MMUD	MUD
Source	BM	PBSC	PBSC	PBSC	BM	Cord	BM	PBSC	PBSC	BM	BM	BM	BM	Cord	PBSC	PBSC
Conditioning	RIC	RIC	RIC	RIC	MAC	MAC	RIC	RIC	RIC	RIC	RIC	RIC	RIC	MAC	RIC	RIC
aGvHD	Yes	Yes	No	Yes	No	Yes	Yes	No	Yes	Yes	Yes	Yes	Yes	Yes	No	No
cGvHD	No	No	No	No	No	No	No	No	Yes	No	No	No	No	No	No	No
Autoimmunity1 (AI1)	Hashimoto	AHIA	GBS-like		ADEM	AIHA	AIT	Polyarthritis		Grave’s disease	GBS-like	AIHA	AIT	AIHA	AIHA	Thyroid swelling with +ab
Time to AI1 (m)π	80	6	28		14	13	9	51		58	4	4	3	14	8	49
Chimerism at AI1 onset	WB 100%	WB 100%	WB 100%		WB 100%	WB 100%	WB 100%	WB 100%		CD3 53%, CD15 100%	WB 98%	CD3 89%, CD15 93%	WB 100%	WB 100%	CD3 63%, CD15 100%	CD3 67%, CD15 9%
Treatment for AI1	Levothyroxine	Steroids-MMF-Rituximab-Sirolimus	Steroids-IVIG-Gabapentin		Steroids	Steroids-Rituximab	IVIG	Steroids		Propranolol-Carbimazole-Levothyroxine	Steroids-NCA-Acetazolamide-Amitriptyline-Gabapentin	Rituximab	IVIG-Steroids-Rituximab	Steroids-Rituximab	Steroids-Rituximab	None
Autoimmunity2 (AI2)						Immune complex glomerulonephritis						Nephrotic syndrome	Autoimmune neutropenia		ITP	/
Chimerism at AI2 onset						CD3 86%, CD15 100%						CD3 89%, CD15 93%	CD3 100%, CD15 100%		CD3 64%, CD15 79%
Time to AI2 (mo) π						97						5	3.7		29
Treatment for AI2						Steroids-MMF-Tacrolimus-Enalapril
Any AI in the first 2 years	No	Yes	No		Yes	Yes	Yes	No		No	Yes	Yes	Yes	Yes	Yes	No
Any AI >2 year	Yes	Yes	Yes		No	Yes	No	Yes		Yes	No	No	No	No	Yes	Yes
Second intervention	None	None	DLI + second HSCT		None	None	None	Gene therapy + second HSCT		None	None	None	None	None	None	DLI
IS at last FU	None	Sirolimus-Steroid	None		None	None	None	None		None	None	None	None	None	None	None
Outcome AI1-AI2	Stable on treatment	Persisting	Resolved		Resolved	Resolved, stable on treatments	Resolved	Resolved		Stable on treatments	Resolved	Resolved	Resolved	Resolved	Resolved	Monitoring

	P7	P8	P12		P15	P17	P20	P21		P29	P30	P31	P36	P37	P38	P41
CGD	gp91	gp91	p67		gp91	gp91	p47	gp91		gp91	gp91	gp91	p47	gp91	p47	p22
Clinical presentation at diagnosis	Liver and skin abscess CGD colitisExtrinsic allergic alveolitis	Invasive Aspergillosis BCG-osis	CGD colitisOsteomyelitis		CGD colitisUrethral granuloma	LymphadenitisPancreatitisLiver abscessPneumoniaSepsis ITP	Skin and perianal abscessesRecurrent URTI/OMAAsthma	Recurrent LRTI		ColitisPerianal diseaseLymphadenitis	Perianal diseaseRecurrent LRTI	Neonatal rashRecurrent LRTIDiarrheaGastric obstructionShort stature	Liver abscessFungal chest infection	Fungal chest infection	Skin granulomasPseudomonas infectionRothia bacteremiaGround glass change	ColitisLiver abscessLymphadenopathyAtypical mycobacteria infection
Age at HSCT (years)	3.4	0.9	3.1 (first)	4.4 (second)	5.4	6.3	8.7	4.4 (first)	8.2 (second)	12.4	10.6	2.2	12.3	1.5	5.4	13.2
Donor	MMUD	MUD	MUD	MMUD	MSD	MMUD	MUD	MUD	MUD	MUD	MMUD	MMUD	MSD	MUD	MMUD	MUD
Source	BM	PBSC	PBSC	PBSC	BM	Cord	BM	PBSC	PBSC	BM	BM	BM	BM	Cord	PBSC	PBSC
Conditioning	RIC	RIC	RIC	RIC	MAC	MAC	RIC	RIC	RIC	RIC	RIC	RIC	RIC	MAC	RIC	RIC
aGvHD	Yes	Yes	No	Yes	No	Yes	Yes	No	Yes	Yes	Yes	Yes	Yes	Yes	No	No
cGvHD	No	No	No	No	No	No	No	No	Yes	No	No	No	No	No	No	No
Autoimmunity1 (AI1)	Hashimoto	AHIA	GBS-like		ADEM	AIHA	AIT	Polyarthritis		Grave’s disease	GBS-like	AIHA	AIT	AIHA	AIHA	Thyroid swelling with +ab
Time to AI1 (m)π	80	6	28		14	13	9	51		58	4	4	3	14	8	49
Chimerism at AI1 onset	WB 100%	WB 100%	WB 100%		WB 100%	WB 100%	WB 100%	WB 100%		CD3 53%, CD15 100%	WB 98%	CD3 89%, CD15 93%	WB 100%	WB 100%	CD3 63%, CD15 100%	CD3 67%, CD15 9%
Treatment for AI1	Levothyroxine	Steroids-MMF-Rituximab-Sirolimus	Steroids-IVIG-Gabapentin		Steroids	Steroids-Rituximab	IVIG	Steroids		Propranolol-Carbimazole-Levothyroxine	Steroids-NCA-Acetazolamide-Amitriptyline-Gabapentin	Rituximab	IVIG-Steroids-Rituximab	Steroids-Rituximab	Steroids-Rituximab	None
Autoimmunity2 (AI2)						Immune complex glomerulonephritis						Nephrotic syndrome	Autoimmune neutropenia		ITP	/
Chimerism at AI2 onset						CD3 86%, CD15 100%						CD3 89%, CD15 93%	CD3 100%, CD15 100%		CD3 64%, CD15 79%
Time to AI2 (mo) π						97						5	3.7		29
Treatment for AI2						Steroids-MMF-Tacrolimus-Enalapril
Any AI in the first 2 years	No	Yes	No		Yes	Yes	Yes	No		No	Yes	Yes	Yes	Yes	Yes	No
Any AI >2 year	Yes	Yes	Yes		No	Yes	No	Yes		Yes	No	No	No	No	Yes	Yes
Second intervention	None	None	DLI + second HSCT		None	None	None	Gene therapy + second HSCT		None	None	None	None	None	None	DLI
IS at last FU	None	Sirolimus-Steroid	None		None	None	None	None		None	None	None	None	None	None	None
Outcome AI1-AI2	Stable on treatment	Persisting	Resolved		Resolved	Resolved, stable on treatments	Resolved	Resolved		Stable on treatments	Resolved	Resolved	Resolved	Resolved	Resolved	Monitoring

AI1, first autoimmunity episode; AI2, second autoimmunity episode; BCG, bacillus Calmette-Guerin; IS, on immunosuppressives; IVIG, intravenous immunoglobulin; LRTI, lower respiratory tract infection; MMF, mycophenolate mofetil; NCA, neurocirculatory asthenia; OMA, otitis media acute; PBSC, peripheral blood stem cell; URTI, upper respiratory tract infection. π: time to AI1 or time to AI2 is time from first HSCT to development of AI1 or AI2.

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