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Table 4 Treatment and outcomes

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

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
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
  1. 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