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Amustaline‐glutathione pathogen‐reduced red blood cell concentrates for transfusion‐dependent thalassaemia
Author(s) -
Aydinok Yesim,
Piga Antonio,
Origa Raffaella,
Mufti Nina,
Erickson Anna,
North Anne,
Waldhaus Katie,
Ernst Christine,
Lin JinSying,
Huang Norman,
Benjamin Richard J.,
Corash Laurence
Publication year - 2019
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.15963
Subject(s) - glutathione , medicine , confidence interval , red blood cell , gastroenterology , adverse effect , crossover study , immunology , pharmacology , surgery , biology , pathology , biochemistry , placebo , enzyme , alternative medicine
Summary Transfusion‐dependent thalassaemia ( TDT ) requires red blood cell concentrates ( RBCC ) to prevent complications of anaemia, but carries risk of infection. Pathogen reduction of RBCC offers potential to reduce infectious risk. We evaluated the efficacy and safety of pathogen‐reduced ( PR ) Amustaline‐Glutathione (A‐ GSH ) RBCC for TDT . Patients were randomized to a blinded 2‐period crossover treatment sequence for six transfusions over 8–10 months with Control and A‐ GSH ‐ RBCC . The efficacy outcome utilized non‐inferiority analysis with 90% power to detect a 15% difference in transfused haemoglobin (Hb), and the safety outcome was the incidence of antibodies to A‐ GSH ‐ PR ‐ RBCC . By intent to treat (80 patients), 12·5 ± 1·9 RBCC were transfused in each period. Storage durations of A‐ GSH and C‐ RBCC were similar (8·9 days). Mean A‐ GSH ‐ RBCC transfused Hb (g/kg/day) was not inferior to Control (0·113 ± 0·04 vs. 0·111 ± 0·04, P = 0·373, paired t ‐test). The upper bound of the one‐sided 95% confidence interval for the treatment difference from the mixed effects model was 0·005 g/kg/day, within a non‐inferiority margin of 0·017 g/kg/day. A‐ GSH ‐ RBCC mean pre‐transfusion Hb levels declined by 6·0 g/l. No antibodies to A‐ GSH ‐ RBCC were detected, and there were no differences in adverse events. A‐ GSH ‐ RBCC s offer potential to reduce infectious risk in TDT with a tolerable safety profile.