Table 2. Individual characteristics of... | Rockefeller University Press

Table 2.

Individual characteristics of patients undergoing HSCT

Patients	P1	P2	P3	P4	P5	P6	P7	P8	P9	P10	P11	P12	P13	P14
Gender	Male	Male	Male	Male	Male	Male	Male	Male	Female	Male	Male	Male	Male	Male
CGD
Age at diagnosis (years)	1	3	3	1	3	1	1	8	0.1	3	0.1	0.1	0.1	27
Genetic pattern	X-linked CGD	X-linked CGD	X-linked CGD	X-linked CGD	X-linked CGD	X-linked CGD	X-linked CGD	X-linked CGD	AR	X-linked CGD	X-linked CGD	X-linked CGD	X-linked CGD	X-linked CGD
Comorbidity	None	Alloimmunization	Alloimmunization	Alloimmunization	Alloimmunization	No	No	CKD	No	No	No	No	CKD	No
History of inflammatory diseases	None	Liver	CNS, colitis	No	No	No data	No	No: Lung	Yes: Colitis	Yes: Colitis	No	No	No	No
History of previous infections	Aspergillus spp	Staphylococcus aureus	S. aureus, A. fumigatus	S. aureus	A. fumigatus	A. fumigatus	A. fumigatus, Serratia marcescens	S. aureus	S. aureus	Aspergillus sydowii	S. aureus	No	Burkholderia multivorans	S. aureus, A. fumigatus
Immunosuppressor treatment ^{^a}	No	Rituximab	Jak inhibitors	Rituximab	Rituximab	CTC	No	No	No data	Adalimumab, AZA, CTC	No	No	No	No
IMI
Age at diagnosis (years)	14	19	38	13	14	14	29	23	0.1	9	0.2	0.1	38	28
Antifungal primary prophylaxis	Itraconazole	Posaconazole	Itraconazole	No data	Itraconazole	Itraconazole	No itraconazole	Posaconazole	No	Posaconazole	No	No	Posaconazole	Posaconazole
Delay between IMI and HSCT (months)^{^b}	28	12	3	23	4	3	7	19	5	7	25	7	14	22
Fungal species ^{^c}	A. fumigatus	A. nidulans	Azole-R A. fumigatus	S. apiospermum	A. nidulans	L. prolificans	A. fumigatus	Azole-R A. fumigatus	Lichtheimia spp	Mucorales spp	A. fumigatus	A. fumigatus	Aspergillus spp	A. fumigatus
Site of infection	Brain^{^d}	Disseminated: Lung, cardiac, and mediastinal	Lung	Disseminated: Brain^{^d} and bones	Disseminated: Lung and bones	Disseminated: Lung, fungemia, and skin	Lung	Disseminated: Lung and thyroid	Lung	Lung	Lung	Lung	Lung	Disseminated: Brain^{^d} and lung
Antifungal treatment	Voriconazole and caspofungin	Isavuconazole and caspofungin	LAmb and caspofungin	Voriconazole and caspofungin	Isavuconazole and caspofungin	Voriconazole, LAmB, and terbinafine	Posaconazole	LAmB and caspofungin	LAmB	LAmB	LAmB	LAmB and caspofungin	Isavuconazole	Voriconazole
Additional treatment	Interferon	Granulocyte transfusion^{^e}	Arterial embolization	Granulocyte transfusion^{^e}	Granulocyte transfusion^{^e}	Granulocyte transfusion^{^e}	No	No	No	No	No	No	No	No
HSCT
Age at HCST	18	20	38	15	14	14	30	25	0.6	10	2	0.8	39	30
HCT-CI score^{^f}	1	3	1	2	1	1	3	1	2	1	1	1	1	3
Donor type	MUD	MUD	MMUD	MSD	MSD	MMUD	MSD	MMUD	MSD	MMRD	MSD	MUD	MUD	MSD
Stem cell source	BM	PBSC	PBSC	BM	BM	BM	BM	PBSC	BM	BM	BM	BM	PBSC	BM
HLA matching	10/10	10/10	9/10	10/10	10/10	9/10	10/10	No data	10/10	No data	10/10	12/12	10/10	10/10
CD34 cells (cells per kg bodyweight)	3.5 × 10⁶	5.7 × 10⁶	4 × 10⁶	7.2 × 10⁶	9.2 × 10⁶	6.1 × 10⁶	1.7 × 10⁶	5.6 × 10⁶	12 × 10⁶	9.06 × 10⁶	17 × 10⁶	10.9 × 10⁶	12 × 10⁶	4.1 × 10⁶
Conditioning intensity^{^g}	RIC	RIC	RIC	RIC	RIC	RIC	RIC	RIC	RIC	RIC	RIC	RIC	RIC	RTC
Serotherapy	Alemtuzumab	Alemtuzumab	Alemtuzumab	Alemtuzumab	ATG	Alemtuzumab	ATG	Alemtuzumab	ATG	Alemtuzumab	ATG	Alemtuzumab	Alemtuzumab	ATG
GVHD prophylaxis^{^h}	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin
Engraftment^ⁱ	Yes	Yes	Yes	No	Yes	No	Yes	Yes	Yes	Yes	Yes	Yes	Yes	Yes
Time of resolution of aplasia (days post-HSC infusion)	27	14	24	–	13	–	23	15	17	21	16	15	20	15
Time of platelet recovery (days post-HSC infusion)	No data	44	10	–	18	–	17	14	11	22	19	39	19	12
Post-HSCT antifungal treatment time (mo) (% donor)	51	14	4		90	–	12	2	1	3	5	2	3	No data
Post-HSCT infection	Klebsiella bacteremia	CMV reactivation	BK virus	HSV reactivation	No	Fungemia	No	No	Klebsiella bacteremia	No	No	No	Rhodotorula	Clostridium difficile
Outcome
1-year OS	Alive	Alive	Alive	Dead (day +10 after HSCT)	Alive	Dead (day +50 after HSCT)	Alive	Alive	Alive	Alive	Alive	Alive	Alive	Alive
DHR at 1 mo after HSCT (%)	92	78	96	–	40	–	99	89	94	81	87	95	67	No data
3-mo chimerism status (% donor)	94	99	99	–	94	–	99	98	98	91	96	97	99	99
3-mo clinical and radiological response	Yes	Yes	Yes	No	Yes	No	Yes	Yes	Yes	Yes	–	–	–	–
1-year persistent clinical and radiological cure	Yes	Yes	Yes	No	Yes	No	Yes	Yes	Yes	Yes	Yes	Yes	Yes	Yes

Patients	P1	P2	P3	P4	P5	P6	P7	P8	P9	P10	P11	P12	P13	P14
Gender	Male	Male	Male	Male	Male	Male	Male	Male	Female	Male	Male	Male	Male	Male
CGD
Age at diagnosis (years)	1	3	3	1	3	1	1	8	0.1	3	0.1	0.1	0.1	27
Genetic pattern	X-linked CGD	X-linked CGD	X-linked CGD	X-linked CGD	X-linked CGD	X-linked CGD	X-linked CGD	X-linked CGD	AR	X-linked CGD	X-linked CGD	X-linked CGD	X-linked CGD	X-linked CGD
Comorbidity	None	Alloimmunization	Alloimmunization	Alloimmunization	Alloimmunization	No	No	CKD	No	No	No	No	CKD	No
History of inflammatory diseases	None	Liver	CNS, colitis	No	No	No data	No	No: Lung	Yes: Colitis	Yes: Colitis	No	No	No	No
History of previous infections	Aspergillus spp	Staphylococcus aureus	S. aureus, A. fumigatus	S. aureus	A. fumigatus	A. fumigatus	A. fumigatus, Serratia marcescens	S. aureus	S. aureus	Aspergillus sydowii	S. aureus	No	Burkholderia multivorans	S. aureus, A. fumigatus
Immunosuppressor treatment ^{^a}	No	Rituximab	Jak inhibitors	Rituximab	Rituximab	CTC	No	No	No data	Adalimumab, AZA, CTC	No	No	No	No
IMI
Age at diagnosis (years)	14	19	38	13	14	14	29	23	0.1	9	0.2	0.1	38	28
Antifungal primary prophylaxis	Itraconazole	Posaconazole	Itraconazole	No data	Itraconazole	Itraconazole	No itraconazole	Posaconazole	No	Posaconazole	No	No	Posaconazole	Posaconazole
Delay between IMI and HSCT (months)^{^b}	28	12	3	23	4	3	7	19	5	7	25	7	14	22
Fungal species ^{^c}	A. fumigatus	A. nidulans	Azole-R A. fumigatus	S. apiospermum	A. nidulans	L. prolificans	A. fumigatus	Azole-R A. fumigatus	Lichtheimia spp	Mucorales spp	A. fumigatus	A. fumigatus	Aspergillus spp	A. fumigatus
Site of infection	Brain^{^d}	Disseminated: Lung, cardiac, and mediastinal	Lung	Disseminated: Brain^{^d} and bones	Disseminated: Lung and bones	Disseminated: Lung, fungemia, and skin	Lung	Disseminated: Lung and thyroid	Lung	Lung	Lung	Lung	Lung	Disseminated: Brain^{^d} and lung
Antifungal treatment	Voriconazole and caspofungin	Isavuconazole and caspofungin	LAmb and caspofungin	Voriconazole and caspofungin	Isavuconazole and caspofungin	Voriconazole, LAmB, and terbinafine	Posaconazole	LAmB and caspofungin	LAmB	LAmB	LAmB	LAmB and caspofungin	Isavuconazole	Voriconazole
Additional treatment	Interferon	Granulocyte transfusion^{^e}	Arterial embolization	Granulocyte transfusion^{^e}	Granulocyte transfusion^{^e}	Granulocyte transfusion^{^e}	No	No	No	No	No	No	No	No
HSCT
Age at HCST	18	20	38	15	14	14	30	25	0.6	10	2	0.8	39	30
HCT-CI score^{^f}	1	3	1	2	1	1	3	1	2	1	1	1	1	3
Donor type	MUD	MUD	MMUD	MSD	MSD	MMUD	MSD	MMUD	MSD	MMRD	MSD	MUD	MUD	MSD
Stem cell source	BM	PBSC	PBSC	BM	BM	BM	BM	PBSC	BM	BM	BM	BM	PBSC	BM
HLA matching	10/10	10/10	9/10	10/10	10/10	9/10	10/10	No data	10/10	No data	10/10	12/12	10/10	10/10
CD34 cells (cells per kg bodyweight)	3.5 × 10⁶	5.7 × 10⁶	4 × 10⁶	7.2 × 10⁶	9.2 × 10⁶	6.1 × 10⁶	1.7 × 10⁶	5.6 × 10⁶	12 × 10⁶	9.06 × 10⁶	17 × 10⁶	10.9 × 10⁶	12 × 10⁶	4.1 × 10⁶
Conditioning intensity^{^g}	RIC	RIC	RIC	RIC	RIC	RIC	RIC	RIC	RIC	RIC	RIC	RIC	RIC	RTC
Serotherapy	Alemtuzumab	Alemtuzumab	Alemtuzumab	Alemtuzumab	ATG	Alemtuzumab	ATG	Alemtuzumab	ATG	Alemtuzumab	ATG	Alemtuzumab	Alemtuzumab	ATG
GVHD prophylaxis^{^h}	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin + MMF	Ciclosporin
Engraftment^ⁱ	Yes	Yes	Yes	No	Yes	No	Yes	Yes	Yes	Yes	Yes	Yes	Yes	Yes
Time of resolution of aplasia (days post-HSC infusion)	27	14	24	–	13	–	23	15	17	21	16	15	20	15
Time of platelet recovery (days post-HSC infusion)	No data	44	10	–	18	–	17	14	11	22	19	39	19	12
Post-HSCT antifungal treatment time (mo) (% donor)	51	14	4		90	–	12	2	1	3	5	2	3	No data
Post-HSCT infection	Klebsiella bacteremia	CMV reactivation	BK virus	HSV reactivation	No	Fungemia	No	No	Klebsiella bacteremia	No	No	No	Rhodotorula	Clostridium difficile
Outcome
1-year OS	Alive	Alive	Alive	Dead (day +10 after HSCT)	Alive	Dead (day +50 after HSCT)	Alive	Alive	Alive	Alive	Alive	Alive	Alive	Alive
DHR at 1 mo after HSCT (%)	92	78	96	–	40	–	99	89	94	81	87	95	67	No data
3-mo chimerism status (% donor)	94	99	99	–	94	–	99	98	98	91	96	97	99	99
3-mo clinical and radiological response	Yes	Yes	Yes	No	Yes	No	Yes	Yes	Yes	Yes	–	–	–	–
1-year persistent clinical and radiological cure	Yes	Yes	Yes	No	Yes	No	Yes	Yes	Yes	Yes	Yes	Yes	Yes	Yes

This table includes individual characteristics of patients undergoing HSCT with DP of IMI (patients 1–5), patients undergoing HSCT with SR (patient 6) and PR (patients 7–10) of IMI, and patients undergoing HSCT with CR of IMIs (patients 11–14). CKD, chronic kidney disease; CNS, central nervous system; Azole-R, azole resistant; BM, bone marrow; MMRD, HLA-mismatched related donor; ATG, antithymocyte globulin (thymoglobuline); LAmB, liposomal amphotericin B; MMF, mycophenolate mofetil; GT, granulocyte transfusion; AZA, azathioprine; CTC, corticosteroids; HCT-CI, hematopoietic cell transplantation–specific comorbidity index; RTC, myeloablative reduced toxicity conditioning, BK: Human Polyomavirus 1, ARDS: acute respiratory distress syndrome.

In the year before the diagnosis of invasive fungal infection.

Delay between the first microbiological evidence of IMI and the last HSCT.

Azole-R A.fumigatus was defined by a documented resistance to all the triazole.

Patient 1 underwent a brain biopsy at the onset of the infection to identify the fungal species. After discussion with the neurosurgeons, the brain lesions of patient 4 and patient 14 were deemed inaccessible for biopsy.

Patient 2 received a granulocytes transfusion three times a wk from month three before HSCT until 3 wk after HCST. Patient 4 received a granulocytes transfusion at the time of IFI diagnosis, but we did not find any information about either frequency of administration or the duration of treatment. Patient 5 received a granulocytes transfusion from 2 mo before HCST until 2 wk before HCST. Patient 6 received a granulocytes transfusion from 2 wk after HCST until death.

HCT-CI is a validated scoring system that assesses the risks of patients undergoing HSCT.

Patients received a fludarabine-busulfan based platform including RIC dose (busulfan = 9.6 mg/kg i.v., n = 12) and RTC dose (busulfan = 12.8 mg/kg i.v.).

Ciclosporin was initiated from day 1 before reinfusion and stopped on day 180 after reinfusion. MMF was initiated from the day of reinfusion and stopped on day 100 after reinfusion.

Graft enhancement was defined by a resolution of aplasia and a bone marrow composition including >50% of cell donor in the chimerism analysis.

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