Risk stratification of high-risk metastatic neuroblastoma: A report from the HR-NBL-1/SIOPEN study

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11130%2F18%3A10381336" target="_blank" >RIV/00216208:11130/18:10381336 - isvavai.cz</a>

Nalezeny alternativní kódy

RIV/00064203:_____/18:10381336

Výsledek na webu

<a href="https://doi.org/10.1002/pbc.27363" target="_blank" >https://doi.org/10.1002/pbc.27363</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1002/pbc.27363" target="_blank" >10.1002/pbc.27363</a>

Jazyk výsledku

angličtina

Název v původním jazyce

Risk stratification of high-risk metastatic neuroblastoma: A report from the HR-NBL-1/SIOPEN study

Popis výsledku v původním jazyce

BackgroundRisk stratification is crucial to treatment decision-making in neuroblastoma. This study aimed to explore factors present at diagnosis affecting outcome in patients aged18 months with metastatic neuroblastoma and to develop a simple risk score for prognostication. ProcedureData were derived from the European high-risk neuroblastoma 1 (HR-NBL1)/International Society for Paediatric Oncology European Neuroblastoma (SIOPEN) trial with analysis restricted to patients aged 18 months with metastatic disease and treated prior to the introduction of immunotherapy. Primary endpoint was 5-year event-free survival (EFS). Prognostic factors assessed were sex, age, tumour MYCN amplification (MNA) status, serum lactate dehydrogenase (LDH)/ferritin, primary tumour and metastatic sites. Factors significant in univariate analysis were incorporated into a multi-variable model and an additive scoring system developed based on estimated log-cumulative hazard ratios. ResultsThe cohort included 1053 patients with median follow-up 5.5years and EFS 271%. In univariate analyses, age; serum LDH and ferritin; involvement of bone marrow, bone, liver or lung; and &gt;1 metastatic system/compartment were associated with worse EFS. Tumour MNA was not associated with worse EFS. A multi-variable model and risk score incorporating age (&gt;5 years, 2 points), serum LDH (&gt;1250U/L, 1 point) and number of metastatic systems (&gt;1, 2 points) were developed. EFS was significantly correlated with risk score: EFS 52 +/- 9% for score=0versus 6 +/- 3% for score=5 (P&lt;0.0001). ConclusionsA simple score can identify an ultra-high risk (UHR) cohort (score=5) comprising 8% of patients with 5-year EFS&lt;10%. These patients appear not to benefit from induction therapy and could potentially be directed earlier to alternative experimental therapies in future trials.

Název v anglickém jazyce

Risk stratification of high-risk metastatic neuroblastoma: A report from the HR-NBL-1/SIOPEN study

Popis výsledku anglicky

BackgroundRisk stratification is crucial to treatment decision-making in neuroblastoma. This study aimed to explore factors present at diagnosis affecting outcome in patients aged18 months with metastatic neuroblastoma and to develop a simple risk score for prognostication. ProcedureData were derived from the European high-risk neuroblastoma 1 (HR-NBL1)/International Society for Paediatric Oncology European Neuroblastoma (SIOPEN) trial with analysis restricted to patients aged 18 months with metastatic disease and treated prior to the introduction of immunotherapy. Primary endpoint was 5-year event-free survival (EFS). Prognostic factors assessed were sex, age, tumour MYCN amplification (MNA) status, serum lactate dehydrogenase (LDH)/ferritin, primary tumour and metastatic sites. Factors significant in univariate analysis were incorporated into a multi-variable model and an additive scoring system developed based on estimated log-cumulative hazard ratios. ResultsThe cohort included 1053 patients with median follow-up 5.5years and EFS 271%. In univariate analyses, age; serum LDH and ferritin; involvement of bone marrow, bone, liver or lung; and &gt;1 metastatic system/compartment were associated with worse EFS. Tumour MNA was not associated with worse EFS. A multi-variable model and risk score incorporating age (&gt;5 years, 2 points), serum LDH (&gt;1250U/L, 1 point) and number of metastatic systems (&gt;1, 2 points) were developed. EFS was significantly correlated with risk score: EFS 52 +/- 9% for score=0versus 6 +/- 3% for score=5 (P&lt;0.0001). ConclusionsA simple score can identify an ultra-high risk (UHR) cohort (score=5) comprising 8% of patients with 5-year EFS&lt;10%. These patients appear not to benefit from induction therapy and could potentially be directed earlier to alternative experimental therapies in future trials.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30204 - Oncology

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2018

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

Pediatric Blood and Cancer

ISSN

1545-5009

e-ISSN

—

Svazek periodika

65

Číslo periodika v rámci svazku

11

Stát vydavatele periodika

US - Spojené státy americké

Počet stran výsledku

9

Strana od-do

—

Kód UT WoS článku

000445194700032

EID výsledku v databázi Scopus

2-s2.0-85050502637