Matched-paired analysis of patients treated for invasive mucormycosis: standard treatment versus posaconazole new formulations (MoveOn)
Authors: Salmanton-García Jon , Seidel Danila , Koehler Philipp , Mellinghoff Sibylle C , Herbrecht Raoul , Klimko Nikolai , Ráčil Zdeněk , Falces-Romero Iker , Ingram Paul , Benítez-Peñuela Miguel-Ángel , Rodríguez José Yesid , Desoubeaux Guillaume , Barać Aleksandra , García-Vidal Carolina , Hoenigl Martin , Mehta Sanjay R , Cheng Matthew P , Klyasova Galina , Heinz Werner J , Iqbal Nousheen , Krause Robert , Ostermann Helmut , Penack Olaf , Schalk Enrico , Sheppard Donald C , Willinger Birgit , Wisplinghoff Hilmar , Vehreschild J Janne , Cornely Oliver A , Vehreschild Maria J G T ,   The FungiScope® ECMM/ISHAM Working Group, Khedr Reham Abdelaziz , Arencibia-Núñez Alberto , Avilés-Robles Martha , Banke Ingo , Basher Ariful , Benachinamardi Keertilaxmi , Bertz Harmut , Chakrabarti Arunaloke , Drgona Lubos , García-Martínez Jesús , García-Rodríguez Julio , Gräber Sandra , Härter Georg , Klein Michael , Kouba Michal , Lee Dong-Gun , Le Govic Yohann , Leo Fabian , Maertens Johan , Maschmeyer Georg , Meintker Lisa , Mo Xiao-Dong , Müller Lena-Katharina , Müller Nicolas , Nel Jeremy Stephen , Novák Jan , Patel Atul , Pfäfflin Frieder , Pozo-Laderas Juan-Carlos , Puerta-Alcalde Pedro , Rodríguez-Guardado Azucena , Schroers Roland , Shekar Vandana , Shenoi Susan , Silling Gerda , Vinh Donald , Waizel-Haiat Salomón , Yee Yee Mandy Yap , Prakash Peralam Yegneswaran , Žák Pavel 
Published: July 2019

Abstract

Background
First-line antifungal treatment for invasive mucormycosis (IM) consists of liposomal amphotericin B. Salvage treatment options are limited and often based on posaconazole oral suspension. With the approval of posaconazole new formulations, patients could benefit from improved pharmacokinetics, safety and tolerability.

Objectives
Our aim was to assess the effectiveness of posaconazole new formulations for IM treatment.

Methods
We performed a case-matched analysis with proven or probable IM patients from the FungiScope® Registry. First-line posaconazole new formulations (1st-POSnew) and first-line amphotericin B plus posaconazole new formulations (1st-AMB+POSnew) cases were matched with first-line amphotericin B-based (1st-AMB) treatment controls. Salvage posaconazole new formulations (SAL-POSnew) cases were matched with salvage posaconazole oral suspension (SAL-POSsusp) controls. Each case was matched with up to three controls (based on severity, haematological/oncological malignancy, surgery and/or renal dysfunction).

Results
Five patients receiving 1st-POSnew, 18 receiving 1st-AMB+POSnew and 22 receiving SAL-POSnew were identified. By day 42, a favourable response was reported for 80.0% (n = 4/5) of patients receiving 1st-POSnew, for 27.8% (n = 5/18) receiving 1st-AMB+POSnew and for 50.0% (n = 11/22) receiving SAL-POSnew. Day 42 all-cause mortality of patients receiving posaconazole new formulations was lower compared with controls [20.0% (n = 1/5) in 1st-POSnew versus 53.3% (n = 8/15) in 1st-AMB; 33.3% (n = 6/18) in 1st-AMB+POSnew versus 52.0% (n = 26/50) in 1st-AMB; and 0.0% (n = 0/22) in SAL-POSnew versus 4.4% (n = 2/45) in SAL-POSsusp].

Conclusions
Posaconazole new formulations were effective in terms of treatment response and associated mortality of IM. While posaconazole new formulations may be an alternative for treatment of IM, the limited sample size of our study calls for a cautious interpretation of these observations.