Isavuconazole treatment for mucormycosis: a single-arm
open-label trial and case-control analysis

J 2016

Isavuconazole treatment for mucormycosis: a single-arm open-label trial and case-control analysis

MARTY, Francisco M; Luis OSTROSKY-ZEICHNER; Oliver A CORNELY; Kathleen M MULLANE; John R PERFECT et al.

Základní údaje

Originální název

Isavuconazole treatment for mucormycosis: a single-arm open-label trial and case-control analysis

Autoři

Vydání

Lancet Infectious Diseases, Oxford, Elsevier SCI LTD, 2016, 1473-3099

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30300 3.3 Health sciences

Stát vydavatele

Velká Británie a Severní Irsko

Utajení

není předmětem státního či obchodního tajemství

Impakt faktor

Impact factor: 19.864

Označené pro přenos do RIV

Ano

Kód RIV

RIV/00216224:14110/16:00089550

Organizační jednotka

Lékařská fakulta

Klíčová slova anglicky

LIPOSOMAL AMPHOTERICIN-B; MYCOSES STUDY-GROUP; FUNGAL-INFECTIONS; INVASIVE ASPERGILLOSIS; TRANSPLANT RECIPIENTS; EUROPEAN-ORGANIZATION; SALVAGE THERAPY; ZYGOMYCOSIS; POSACONAZOLE; VORICONAZOLE

Štítky

EL OK

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 4. 8. 2016 08:49, Ing. Mgr. Věra Pospíšilíková

Anotace

V originále

Background Mucormycosis is an uncommon invasive fungal disease with high mortality and few treatment options. Isavuconazole is a triazole active in vitro and in animal models against moulds of the order Mucorales. We assessed the effi cacy and safety of isavuconazole for treatment of mucormycosis and compared its effi cacy with amphotericin B in a matched case-control analysis. Methods In a single-arm open-label trial (VITAL study), adult patients (>= 18 years) with invasive fungal disease caused by rare fungi, including mucormycosis, were recruited from 34 centres worldwide. Patients were given isavuconazole 200 mg (as its intravenous or oral water-soluble prodrug, isavuconazonium sulfate) three times daily for six doses, followed by 200 mg/day until invasive fungal disease resolution, failure, or for 180 days or more. The primary endpoint was independent data review committee-determined overall response—ie, complete or partial response (treatment success) or stable or progressive disease (treatment failure)—according to prespecifi ed criteria. Mucormycosis cases treated with isavuconazole as primary treatment were matched with controls from the FungiScope Registry, recruited from 17 centres worldwide, who received primary amphotericin B-based treatment, and were analysed for day-42 allcause mortality. VITAL is registered with ClinicalTrials.gov, number NCT00634049. FungiScope is registered with ClinicalTrials.gov, number NCT01731353. Findings Within the VITAL study, from April 22, 2008, to June 21, 2013, 37 patients with mucormycosis received isavuconazole for a median of 84 days (IQR 19–179, range 2–882). By day 42, four patients (11%) had a partial response, 16 (43%) had stable invasive fungal disease, one (3%) had invasive fungal disease progression, three (8%) had missing assessments, and 13 (35%) had died. 35 patients (95%) had adverse events (28 [76%] serious). Day-42 crude all-cause mortality in seven (33%) of 21 primary-treatment isavuconazole cases was similar to 13 (39%) of 33 amphotericin B-treated matched controls (weighted all-cause mortality: 33% vs 41%; p=0·595). Interpretation Isavuconazole showed activity against mucormycosis with effi cacy similar to amphotericin B. Isavuconazole can be used for treatment of mucormycosis and is well tolerated.

Citovat

MARTY, Francisco M; Luis OSTROSKY-ZEICHNER; Oliver A CORNELY; Kathleen M MULLANE; John R PERFECT; George R THOMPSON III; George J ALANGADEN; Janice M BROWN; David N FREDRICKS; Werner J HEINZ; Raoul HERBRECHT; Nikolai KLIMKO; Galina KLYASOVA; Johan A MAERTENS; Sameer R MELINKERI; Ilana OREN; Peter G PAPPAS; Zdeněk RÁČIL; Galia RAHAV; Rodrigo SANTOS; Stefan SCHWARTZ; J Janne VEHRESCHILD; Jo-Anne H YOUNG; Ploenchan CHETCHOTISAKD; Sutep JARURATANASIRIKUL; Souha S KANJ; Marc ENGELHARDT; Achim KAUFHOLD; Masanori ITO; Misun LEE; Carolyn SASSE; Rochelle M MAHER; Bernhardt ZEIHER a Maria J G T VEHRESCHILD. Isavuconazole treatment for mucormycosis: a single-arm open-label trial and case-control analysis. Lancet Infectious Diseases. Oxford: Elsevier SCI LTD, 2016, roč. 16, č. 7, s. 828-837. ISSN 1473-3099. Dostupné z: https://doi.org/10.1016/S1473-3099(16)00071-2.

@article{1339539,
   author = {Marty, Francisco M and OstroskyandZeichner, Luis and Cornely, Oliver A and Mullane, Kathleen M and Perfect, John R and Thompson III, George R and Alangaden, George J and Brown, Janice M and Fredricks, David N and Heinz, Werner J and Herbrecht, Raoul and Klimko, Nikolai and Klyasova, Galina and Maertens, Johan A and Melinkeri, Sameer R and Oren, Ilana and Pappas, Peter G and Ráčil, Zdeněk and Rahav, Galia and Santos, Rodrigo and Schwartz, Stefan and Vehreschild, J Janne and Young, JoandAnne H and Chetchotisakd, Ploenchan and Jaruratanasirikul, Sutep and Kanj, Souha S and Engelhardt, Marc and Kaufhold, Achim and Ito, Masanori and Lee, Misun and Sasse, Carolyn and Maher, Rochelle M and Zeiher, Bernhardt and Vehreschild, Maria J G T},
   article_location = {Oxford},
   article_number = {7},
   doi = {https://doi.org/10.1016/S1473-3099(16)00071-2},
   keywords = {LIPOSOMAL AMPHOTERICIN-B; MYCOSES STUDY-GROUP; FUNGAL-INFECTIONS; INVASIVE ASPERGILLOSIS; TRANSPLANT RECIPIENTS; EUROPEAN-ORGANIZATION; SALVAGE THERAPY; ZYGOMYCOSIS; POSACONAZOLE; VORICONAZOLE},
   language = {eng},
   issn = {1473-3099},
   journal = {Lancet Infectious Diseases},
   title = {Isavuconazole treatment for mucormycosis: a single-arm open-label trial and case-control analysis},
   volume = {16},
   year = {2016}
}

TY  - JOUR
ID  - 1339539
AU  - Marty, Francisco M - Ostrosky-Zeichner, Luis - Cornely, Oliver A - Mullane, Kathleen M - Perfect, John R - Thompson III, George R - Alangaden, George J - Brown, Janice M - Fredricks, David N - Heinz, Werner J - Herbrecht, Raoul - Klimko, Nikolai - Klyasova, Galina - Maertens, Johan A - Melinkeri, Sameer R - Oren, Ilana - Pappas, Peter G - Ráčil, Zdeněk - Rahav, Galia - Santos, Rodrigo - Schwartz, Stefan - Vehreschild, J Janne - Young, Jo-Anne H - Chetchotisakd, Ploenchan - Jaruratanasirikul, Sutep - Kanj, Souha S - Engelhardt, Marc - Kaufhold, Achim - Ito, Masanori - Lee, Misun - Sasse, Carolyn - Maher, Rochelle M - Zeiher, Bernhardt - Vehreschild, Maria J G T
PY  - 2016
TI  - Isavuconazole treatment for mucormycosis: a single-arm open-label trial and case-control analysis
JF  - Lancet Infectious Diseases
VL  - 16
IS  - 7
SP  - 828-837
EP  - 828-837
PB  - Elsevier SCI LTD
SN  - 14733099
KW  - LIPOSOMAL AMPHOTERICIN-B
KW  - MYCOSES STUDY-GROUP
KW  - FUNGAL-INFECTIONS
KW  - INVASIVE ASPERGILLOSIS
KW  - TRANSPLANT RECIPIENTS
KW  - EUROPEAN-ORGANIZATION
KW  - SALVAGE THERAPY
KW  - ZYGOMYCOSIS
KW  - POSACONAZOLE
KW  - VORICONAZOLE
N2  - Background Mucormycosis is an uncommon invasive fungal disease with high mortality and few treatment options. Isavuconazole is a triazole active in vitro and in animal models against moulds of the order Mucorales. We assessed the effi cacy and safety of isavuconazole for treatment of mucormycosis and compared its effi cacy with amphotericin B in a matched case-control analysis. Methods In a single-arm open-label trial (VITAL study), adult patients (>= 18 years) with invasive fungal disease caused by rare fungi, including mucormycosis, were recruited from 34 centres worldwide. Patients were given isavuconazole 200 mg (as its intravenous or oral water-soluble prodrug, isavuconazonium sulfate) three times daily for six doses, followed by 200 mg/day until invasive fungal disease resolution, failure, or for 180 days or more. The primary endpoint was independent data review committee-determined overall response—ie, complete or partial response (treatment success) or stable or progressive disease (treatment failure)—according to prespecifi ed criteria. Mucormycosis cases treated with isavuconazole as primary treatment were matched with controls from the FungiScope Registry, recruited from 17 centres worldwide, who received primary amphotericin B-based treatment, and were analysed for day-42 allcause mortality. VITAL is registered with ClinicalTrials.gov, number NCT00634049. FungiScope is registered with ClinicalTrials.gov, number NCT01731353. Findings Within the VITAL study, from April 22, 2008, to June 21, 2013, 37 patients with mucormycosis received isavuconazole for a median of 84 days (IQR 19–179, range 2–882). By day 42, four patients (11%) had a partial response, 16 (43%) had stable invasive fungal disease, one (3%) had invasive fungal disease progression, three (8%) had missing assessments, and 13 (35%) had died. 35 patients (95%) had adverse events (28 [76%] serious). Day-42 crude all-cause mortality in seven (33%) of 21 primary-treatment isavuconazole cases was similar to 13 (39%) of 33 amphotericin B-treated matched controls (weighted all-cause mortality: 33% vs 41%; p=0·595). Interpretation Isavuconazole showed activity against mucormycosis with effi cacy similar to amphotericin B. Isavuconazole can be used for treatment of mucormycosis and is well tolerated.
ER  -

MARTY, Francisco M; Luis OSTROSKY-ZEICHNER; Oliver A CORNELY; Kathleen M MULLANE; John R PERFECT; George R THOMPSON III; George J ALANGADEN; Janice M BROWN; David N FREDRICKS; Werner J HEINZ; Raoul HERBRECHT; Nikolai KLIMKO; Galina KLYASOVA; Johan A MAERTENS; Sameer R MELINKERI; Ilana OREN; Peter G PAPPAS; Zdeněk RÁČIL; Galia RAHAV; Rodrigo SANTOS; Stefan SCHWARTZ; J Janne VEHRESCHILD; Jo-Anne H YOUNG; Ploenchan CHETCHOTISAKD; Sutep JARURATANASIRIKUL; Souha S KANJ; Marc ENGELHARDT; Achim KAUFHOLD; Masanori ITO; Misun LEE; Carolyn SASSE; Rochelle M MAHER; Bernhardt ZEIHER a Maria J G T VEHRESCHILD. Isavuconazole treatment for mucormycosis: a single-arm open-label trial and case-control analysis. \textit{Lancet Infectious Diseases}. Oxford: Elsevier SCI LTD, 2016, roč.~16, č.~7, s.~828-837. ISSN~1473-3099. Dostupné z: https://doi.org/10.1016/S1473-3099(16)00071-2.

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