Table 2.

Studies on echinocandin prophylaxis in patients with haematological malignancies or undergoing HCT

Study citation	Methodology	Setting, population, dates	Arms	Qualitya	Primary endpoint
Echinocandin versus fluconazole
131	RCT, blinded	US; multicentre (72 centres); mostly adult; allogeneic or autologous HCT; assessed neutropenic phase; 1999–2000	Micafungin, (50 mg iv daily; N = 425) vs fluconazole (400 mg iv daily; N = 457) starting ≤48 h after conditioning through engraftment, D + 42, IFI or drug cessation	High	Absence of IFI: micafungin superior, NNT 15
132	retrospective, cohort (historical control)	Japan; single-centre; mostly adult; allogeneic HCT; assessed through D + 49; micafungin patients recruited from 2004–07; unknown dates of historical cases; assessed neutropenic phase	Micafungin (100 mg iv daily; N = 41) vs historical control fluconazole (400 mg iv/po daily; N = 29); both started D − 14; both groups changed to fluconazole 200 mg po daily after engraftment and tolerating po intake	Low	Absence of proven, probable, or possible IFI: micafungin superior, NNT 5
133	RCT, open label	Japan; multicentre (6 centres); mostly adult; allogeneic or autologous HCT; assessed neutropenic phase; 2004–06	Micafungin (150 mg iv daily; N = 50) vs fluconazole (400 mg iv daily; N = 50) starting ≤48 h after conditioning through engraftment, D + 42, IFI or drug cessation	Medium	Absence of IFI: no significant difference
134	RCT, open label	Korea; single-centre; adult; allogeneic or autologous HCT; assessed neutropenic phase; 2010–15	Micafungin (50 mg iv daily; N = 165) vs fluconazole (400 mg po/iv daily; N = 85) starting ≤24 h after HCT infusion through engraftment, D + 21, IFI or drug cessation	Medium	Incidence of proven or probable IFI: no significant difference
Echinocandin versus itraconazole
137	RCT, open label	US (TX); single-centre; mostly adult; AML or MDS undergoing induction chemotherapy; 2001–03	Caspofungin (50 mg iv daily; N = 107) vs itraconazole (200 mg iv bid ×2 days then daily; N = 90) starting with induction through resolution of neutropenia, CR, death, change in therapy, IFI, toxicity or through 25 days	Medium	Completion of prophylaxis without IFI: no significant difference
135	RCT, open label	China; multicentre (10 centres); adults; allogeneic or autologous HCT; assessed neutropenic phase; 2008–09	Micafungin (50 mg iv daily; N = 136) vs itraconazole (5 mg/kg/day po in 2 divided doses; N = 147) starting within 48 h of beginning of conditioning regimen ending with engraftment, IFI, toxicity, death, withdrawal or other discontinuation	Medium	Absence of IFI: no significant difference
Echinocandin versus posaconazole
158	Retrospective, cohort (historical control)	Austria; single-centre; adults; mixed population (induction and consolidation for acute leukaemia, allo- and auto-HCT, GvHD, some others); 2011–12 (historical control 2008–10); notable differences in baseline characteristics	Micafungin (50 mg iv daily; N = 100) vs posaconazole suspension (200 mg po q8h; N = 202) during neutropenia or other immunosuppression	Low	IFI incidence: no significant difference
Echinocandin versus mixed azoles
92	RCT, open label	Italy; multicentre; adults; mostly AML with some ALL; 2007–09	Caspofungin (70, 50 mg iv daily; N = 93) vs standard prophylaxis (mostly itraconazole; some posaconazole; unclear others; N = 82)	Medium	Incidence of proven or probable IFIs: no significant difference
138	Retrospective, cohort	USA (TX); single centre; adults; AML newly diagnosed undergoing induction chemotherapy; 2009–11	Echinocandin (N = 38) vs voriconazole or posaconazole (N = 42), minimally described	Low	Development of IFI: azole superior

NNT, number needed to treat; bid, twice daily; po, by mouth; D, day; CR, complete remission.

^aDouble-blinded RCTs were rated as high quality. Other RCTS were rated as medium quality. Non-interventional studies were rated as low quality.