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Cancer. 2018 Mar 1;124(5):1016-1024. doi: 10.1002/cncr.31157. Epub 2017 Dec 6.

Prognostic relevance of early radiologic response to induction chemotherapy in pediatric rhabdomyosarcoma: A report from the International Society of Pediatric Oncology Malignant Mesenchymal Tumor 95 study.

Author information

1
Department of Pediatric Oncology, Emma Children's Hospital/Academic Medical Center, Amsterdam, the Netherlands.
2
Pediatric Clinical Research Office, Emma Children's Hospital/Academic Medical Center, Amsterdam, the Netherlands.
3
Department of Pediatric, Adolescent, and Young Adult Oncology, Curie Institute, Paris, France.
4
Children and Young People's Department, Royal Marsden Hospital, Sutton, United Kingdom.
5
Department of Biostatistics and Epidemiology, Gustave-Roussy, Villejuif, France.
6
Department of Pediatric Oncology, Children's Hospital for Wales, Cardiff, United Kingdom.
7
Pediatric Radiology, Emma Children's Hospital/Academic Medical Center, Amsterdam, the Netherlands.
8
Department of Radiology, Great Ormond Street Hospital for Children, London, United Kingdom.
9
Pediatric Oncology, Vall d'Hebron University Hospital, Barcelona, Spain.
10
Department of Pediatric and Adolescent Medicine, Oslo University Hospital, Oslo, Norway.
11
Pediatric Hematology Oncology Department, Children's University Hospital, Brussels, Belgium.
12
Department of Oncology, University College London Hospitals NHS Foundation Trust, London, United Kingdom.
13
Pathology Department, Royal Manchester Children's Hospital, Manchester, United Kingdom.
14
Department of Pediatric Oncology, Léon Bérard Center, Lyon, France.
15
Department of Pediatric Oncology, Bristol Royal Hospital for Children, Bristol, United Kingdom.
16
Department of Pediatric and Adolescent Oncology, Gustave-Roussy, Villejuif, France.

Abstract

BACKGROUND:

Early response to induction chemotherapy is used in current European guidelines to evaluate the efficacy of chemotherapy and subsequently to adapt treatment in pediatric patients with rhabdomyosarcoma (RMS). However, existing literature on the prognostic value of early radiologic response on survival is contradictory; here the prognostic value is analyzed with data from the International Society of Pediatric Oncology (SIOP) Malignant Mesenchymal Tumor 95 (MMT-95) study.

METHODS:

This study examined 432 Intergroup Rhabdomyosarcoma Study Grouping III (macroscopic residue) patients enrolled in the SIOP MMT-95 study with a response assessment after 3 courses of chemotherapy (a 2-dimensional assessment). Patients with progressive disease (PD) after 3 courses of chemotherapy were excluded (n = 7). Failure-free survival (FFS) and overall survival (OS), calculated with the Kaplan-Meier method, were compared for 3 groups (complete response [CR]/partial response [PR], objective response [OR], and no response [NR]). The prognostic impact of early response was assessed through the calculation of Cox proportional hazards.

RESULTS:

After 3 courses of chemotherapy, 85.2% of the patients had CR/PR, 8.6% had OR, and 6.3% had NR. For all patients, the 5-year FFS and OS rates were 60% (95% confidence interval [CI], 56%-65%) and 74% (95% CI, 70%-78%), respectively. However, a Cox proportional hazards regression analysis revealed no significant difference in FFS or OS between the response groups. The adjusted hazard ratios for an OR and NR were 1.09 (95% CI, 0.63-1.88) and 0.81 (95% CI, 0.39-1.67), respectively, for FFS and 0.91 (95% CI, 0.47-1.76) and 1.27 (95% CI, 0.61-2.64), respectively, for OS.

CONCLUSIONS:

No evidence was found for the idea that early radiologic response to chemotherapy is prognostic for survival for patients with RMS. Treatment adaptation based on early response (except for patients with PD) should, therefore, no longer be incorporated into future studies. Cancer 2018;124:1016-24. © 2017 American Cancer Society.

KEYWORDS:

chemotherapy; pediatric cancer; prognosis; rhabdomyosarcoma; tumor response

PMID:
29211298
DOI:
10.1002/cncr.31157
[Indexed for MEDLINE]
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