Table 1

Summary of response, OS, and clonal evolution in refractory AA patients treated with second-line IST using ATG

ReferenceNATG first courseATG second courseResponseOSClonal evolution
Scheinberg et al18  27 hATG + CSA rATG + CSA 33% 60% at 3 y 2 with −7, 1 with t(6;14), 1 with trisomy 6 
Scheinberg et al14  22 hATG + CSA ± MMF or sirolimus rATG + CSA ± MMF or sirolimus 30% 70% at d +1000 2 (MDS: +6, −7), 2 (AML) 
Di Bona et al13  30 hATG + CSA + G-CSF (until d 90) rATG + CSA + G-CSF d +1-90 77% 93% at d +914 2 (MDS), 1 (MGUS) 
Tichelli et al42  25 hATG + androgens + CSA after 1991 hATG + androgens + CSA after 1991 64% 55% at 10 y NK 
Marsh et al43  18 hATG + androgens rATG + androgens 22% NK NK 
ReferenceNATG first courseATG second courseResponseOSClonal evolution
Scheinberg et al18  27 hATG + CSA rATG + CSA 33% 60% at 3 y 2 with −7, 1 with t(6;14), 1 with trisomy 6 
Scheinberg et al14  22 hATG + CSA ± MMF or sirolimus rATG + CSA ± MMF or sirolimus 30% 70% at d +1000 2 (MDS: +6, −7), 2 (AML) 
Di Bona et al13  30 hATG + CSA + G-CSF (until d 90) rATG + CSA + G-CSF d +1-90 77% 93% at d +914 2 (MDS), 1 (MGUS) 
Tichelli et al42  25 hATG + androgens + CSA after 1991 hATG + androgens + CSA after 1991 64% 55% at 10 y NK 
Marsh et al43  18 hATG + androgens rATG + androgens 22% NK NK 

hATG indicates horse ATG; rATG, rabbit ATG; MMF, mycophenolate mofetil; MGUS, monoclonal gammopathy of unknown significance; AML, acute myeloid leukemia; and NK, not known.

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