Comprehensive analysis on phenotype and genetic basis of Chinese Fanconi anemia patients: dismal outcomes call for nationwide studies

Table 4 Treatment and outcomes

Case No.	Therapeutics	Donor & HLA matching	Pre-HSCT conditioning regimen	Outcomes
1	HSCT	UUD; 8/10	Bu + CTX + Flu+Alemtuzumab	Dead (aGVHD, infections)
2	HSCT	UUD; 9/10	Bu + CTX + Flu+ATG + Me-CCNU	Alive
3	HSCT	MUD; 10/10	Bu + Flu+CTX + ATG	Alive
4	HSCT	HRD; 8/10	Bu + CTX + Flu+ATG + Me-CCNU	Alive
5	HSCT	HRD; 6/10		Dead (aGVHD, drug-induced encephalopathy)
			Decitabine+Ara-C + Bu + Flu+ATG + Me-CCNU	Dead (aGVHD, drug-induced encephalopathy)
6	Androgen and transfusion	–	–	Alive
7	HSCT	HRD; 7/10		Dead (aGVHD, MODS)
			Decitabine+Ara-C + Bu + Flu+ATG + Me-CCNU
8	Lost follow-up	–	–	–
9	HSCT	MRD; 10/12	Bu + Flu+CTX + ATG	Dead (aGVHD, septic shock)
10	HSCT	HRD; 5/10		Dead (aGVHD, septic shock)
			Decitabine+Ara-C + Bu + Flu+ATG + Me-CCNU
11	HSCT	UUC; 5/8	Bu + Flu+CTX + ATG	Dead (aGVHD, pulmonary infection, CMV infection)
12	Androgen, cytokine, transfusion	–	–	Alive
13	Androgen and cytokine	–	–	Alive
14	HSCT	HRD; 7/10	Bu + Flu+CTX + ATG	Dead (aGVHD, TMA, pulmonary infection)
15	HSCT	HRD; 7/10	Bu + Flu+CTX + ATG	Alive
16	Androgen and transfusion	–	–	Dead (pulmonary infection, septic shock)
17	HSCT	MUD; 10/10	Bu + Flu+CTX + ATG	Alive
18	HSCT	UUD; 8/10	Bu + Flu+CTX + ATG	Alive
19	HSCT	UUD; 9/10	TBI + CTX + Flu+ATG	Alive
20	HSCT	MUD; 10/10	Bu + Flu+CTX + ATG	Alive
21	Loss to follow-up	–	–	–

HSCT hematologic stem cell transplantation, UUD HLA-unmatched unrelated donor, MUD HLA-matched unrelated donor, HRD HLA-haploidentical related donor, MRD HLA-matched related donors, UUC HLA-unmatched unrelated cord blood, Bu Busulfan, CTX cyclophosphamide, Flu Fludarabine, ATG antithymocyte globulin, Me-CCNU Semustine, TBI total body irradiation, aGVHD acute graft-versus-host disease, CMV cytomegalovirus

ISSN: 1471-2350