Improved osteosarcoma survival with addition of mifamurtide to
conventional chemotherapy-Observational prospective single
institution analysis

J 2021

Improved osteosarcoma survival with addition of mifamurtide to conventional chemotherapy-Observational prospective single institution analysis

MÚDRY, Peter, Michal KÝR, Ondřej ROHLEDER, Michal MAHDAL, Iva ZAMBO et. al.

Základní údaje

Originální název

Improved osteosarcoma survival with addition of mifamurtide to conventional chemotherapy-Observational prospective single institution analysis

Autoři

MÚDRY, Peter (203 Česká republika, garant, domácí), Michal KÝR (203 Česká republika, domácí), Ondřej ROHLEDER (203 Česká republika, domácí), Michal MAHDAL (203 Česká republika, domácí), Iva ZAMBO (203 Česká republika, domácí), Marta JEŽOVÁ (203 Česká republika, domácí), Tomáš TOMÁŠ (203 Česká republika, domácí) a Jaroslav ŠTĚRBA (203 Česká republika, domácí)

Vydání

JOURNAL OF BONE ONCOLOGY, AMSTERDAM, ELSEVIER, 2021, 2212-1374

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30204 Oncology

Stát vydavatele

Nizozemské království

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 4.491

Kód RIV

RIV/00216224:14110/21:00123645

Organizační jednotka

Lékařská fakulta

DOI

http://dx.doi.org/10.1016/j.jbo.2021.100362

UT WoS

000665786400007

Klíčová slova anglicky

Osteosarcoma; Mifamurtide; Survival; Single institution analysis; Comparative analysis

Štítky

14110112, 14110123, 14110230, 14110321, rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 21. 2. 2022 09:09, Mgr. Tereza Miškechová

Anotace

V originále

Purpose: Conventional osteosarcoma is an orphan disease. Current treatment approaches include combining a three drug chemotherapy schedule and surgery. The 3-and 5-year event-free survival (EFS) in localized disease is roughly 65 and 60%, respectively. The registration study of mifamurtide reported survival benefit, but some methodological controversies have been insufficient for FDA market authorization in contrast to EMA. Methods: prospective single centre survival analysis of a mifamurtide addition to conventional therapy in 23 patients over a 5.5 year enrolment period is reported and compared to a historical control of 26 patient with localized disease. Bias arising from observational methodology was addressed using Landmark analysis and time-dependent Cox models. Blood count dynamics were analysed during the treatment. Results: The adverse event profile was as expected with no dose limiting toxicities. There were no local relapses observed, one patient died in the first complete remission due to doxorubicin cardiotoxicity, one patient had pulmonary metastatic relapse. The observed 3-and 5-year EFS was 87.4% (CI 72.4-100%) and 87.4% (CI 72.4-100%), progression free survival (PFS) was 92.9% (CI 80.3-100%) and 92.9% (CI 80.3-100%), overall survival was 94.1% (CI 83.6-100) and 80.7% (CI 58.3-100), respectively. Comparison to the historical control showed statistically significant better PFS for mifamurtide patients (Landmark analysis; p = 0.044). Risk of progression was 5-times lower for the mifamurtide group (Cox model; HR 0.21, p = 0.136). Only subtle differences in lymphocyte counts were observed across treatment. Conclusion: the PFS benefit of mifamurtide is reported herein. The addition of mifamurtide could be considered as a best treatment option for localized osteosarcoma. (c) 2021 The Authors. Published by Elsevier GmbH. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).

Návaznosti

MUNI/A/1409/2019, interní kód MU

Název: Personalizovaná léčba v dětské onkologii: na cestě k "liquid dynamic medicine" a "N-of-1 clinical trials"

Investor: Masarykova univerzita, Personalizovaná léčba v dětské onkologii: na cestě k "liquid dynamic medicine" a "N-of-1 clinical trials", DO R. 2020_Kategorie A - Specifický výzkum - Studentské výzkumné projekty

MUNI/A/1701/2020, interní kód MU

Název: Personalizovaná léčba v dětské onkologii: na cestě k „liquid dynamic medicine“ a „N-of-1 clinical trials“

Investor: Masarykova univerzita, Personalizovaná léčba v dětské onkologii: na cestě k „liquid dynamic medicine“ a „N-of-1 clinical trials“

90128, velká výzkumná infrastruktura

Název: CZECRIN III

Citovat

MÚDRY, Peter, Michal KÝR, Ondřej ROHLEDER, Michal MAHDAL, Iva ZAMBO, Marta JEŽOVÁ, Tomáš TOMÁŠ a Jaroslav ŠTĚRBA. Improved osteosarcoma survival with addition of mifamurtide to conventional chemotherapy-Observational prospective single institution analysis. JOURNAL OF BONE ONCOLOGY. AMSTERDAM: ELSEVIER, 2021, roč. 28, June 2021, s. 1-6. ISSN 2212-1374. Dostupné z: https://dx.doi.org/10.1016/j.jbo.2021.100362.

@article{1819498,
   author = {Múdry, Peter and Kýr, Michal and Rohleder, Ondřej and Mahdal, Michal and Zambo, Iva and Ježová, Marta and Tomáš, Tomáš and Štěrba, Jaroslav},
   article_location = {AMSTERDAM},
   article_number = {June 2021},
   doi = {http://dx.doi.org/10.1016/j.jbo.2021.100362},
   keywords = {Osteosarcoma; Mifamurtide; Survival; Single institution analysis; Comparative analysis},
   language = {eng},
   issn = {2212-1374},
   journal = {JOURNAL OF BONE ONCOLOGY},
   title = {Improved osteosarcoma survival with addition of mifamurtide to conventional chemotherapy-Observational prospective single institution analysis},
   url = {https://www.sciencedirect.com/science/article/pii/S2212137421000166?via%3Dihub},
   volume = {28},
   year = {2021}
}

TY  - JOUR
ID  - 1819498
AU  - Múdry, Peter - Kýr, Michal - Rohleder, Ondřej - Mahdal, Michal - Zambo, Iva - Ježová, Marta - Tomáš, Tomáš - Štěrba, Jaroslav
PY  - 2021
TI  - Improved osteosarcoma survival with addition of mifamurtide to conventional chemotherapy-Observational prospective single institution analysis
JF  - JOURNAL OF BONE ONCOLOGY
VL  - 28
IS  - June 2021
SP  - 1-6
EP  - 1-6
PB  - ELSEVIER
SN  - 22121374
KW  - Osteosarcoma
KW  - Mifamurtide
KW  - Survival
KW  - Single institution analysis
KW  - Comparative analysis
UR  - https://www.sciencedirect.com/science/article/pii/S2212137421000166?via%3Dihub
N2  - Purpose: Conventional osteosarcoma is an orphan disease. Current treatment approaches include combining a three drug chemotherapy schedule and surgery. The 3-and 5-year event-free survival (EFS) in localized disease is roughly 65 and 60%, respectively. The registration study of mifamurtide reported survival benefit, but some methodological controversies have been insufficient for FDA market authorization in contrast to EMA. Methods: prospective single centre survival analysis of a mifamurtide addition to conventional therapy in 23 patients over a 5.5 year enrolment period is reported and compared to a historical control of 26 patient with localized disease. Bias arising from observational methodology was addressed using Landmark analysis and time-dependent Cox models. Blood count dynamics were analysed during the treatment. Results: The adverse event profile was as expected with no dose limiting toxicities. There were no local relapses observed, one patient died in the first complete remission due to doxorubicin cardiotoxicity, one patient had pulmonary metastatic relapse. The observed 3-and 5-year EFS was 87.4% (CI 72.4-100%) and 87.4% (CI 72.4-100%), progression free survival (PFS) was 92.9% (CI 80.3-100%) and 92.9% (CI 80.3-100%), overall survival was 94.1% (CI 83.6-100) and 80.7% (CI 58.3-100), respectively. Comparison to the historical control showed statistically significant better PFS for mifamurtide patients (Landmark analysis; p = 0.044). Risk of progression was 5-times lower for the mifamurtide group (Cox model; HR 0.21, p = 0.136). Only subtle differences in lymphocyte counts were observed across treatment. Conclusion: the PFS benefit of mifamurtide is reported herein. The addition of mifamurtide could be considered as a best treatment option for localized osteosarcoma. (c) 2021 The Authors. Published by Elsevier GmbH. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
ER  -

MÚDRY, Peter, Michal KÝR, Ondřej ROHLEDER, Michal MAHDAL, Iva ZAMBO, Marta JEŽOVÁ, Tomáš TOMÁŠ a Jaroslav ŠTĚRBA. Improved osteosarcoma survival with addition of mifamurtide to conventional chemotherapy-Observational prospective single institution analysis. \textit{JOURNAL OF BONE ONCOLOGY}. AMSTERDAM: ELSEVIER, 2021, roč.~28, June 2021, s.~1-6. ISSN~2212-1374. Dostupné z: https://dx.doi.org/10.1016/j.jbo.2021.100362.

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