Twenty-five–year follow-up among survivors of childhood acute lymphoblastic leukemia: a report from the Childhood Cancer Survivor Study

Mody, Rajen; Li, Suwen; Dover, Douglas C.; Sallan, Stephen E.; Leisenring, Wendy M.; Oeffinger, Kevin C.; Yasui, Yutaka; Robison, Leslie L.; Neglia, Joseph P.

doi:10.1182/blood-2007-10-117150

Cited by 338 publications

(313 citation statements)

References 65 publications

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“…The relatively high proportion of hematological malignancies (particularly acute lymphocytic leukemia, as well as Hodgkin's and nonHodgkin's lymphoma) is well in accordance with literature data mentioning that these histiotypes are among the most prevalent malignant neoplasms in children [3,4,31]. And the substantially lower rates of acute myelogenous leukemia, chronic lymphocytic leukemia, and chronic myelogenous leukemia in the current study is supported by their substantially lower global prevalence in children than adults [31].…”

Section: Discussionsupporting

confidence: 78%

“…Childhood cancers can be treated successfully with multimodal therapies which result in average five-year survival rates of 80%, and for children with leukemia, Hodgkin's disease, or Wilms' tumor even close to 90% [4]. However, these improvements may occur at the cost of serious adverse effects later in life such as acute myeloid leukemia [4][5][6].…”

Section: Introductionmentioning

confidence: 99%

“…However, these improvements may occur at the cost of serious adverse effects later in life such as acute myeloid leukemia [4][5][6].…”

Section: Introductionmentioning

confidence: 99%

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Childhood Cancer in the Republic of Suriname (1980 Through 2008)

Mans¹,

Zijlmans²

2014

TOEPIJ

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Childhood cancer incidence in Suriname (South-America) was estimated using secondary data from 1980 to 2008, and these findings were stratified according to gender; age groups < 1, 1-4, 5-14, and 15-19 years; and the largest ethnic groups (Hindustanis, Maroons, Creoles, and Javanese). Data were expressed as total numbers, proportions, average yearly numbers, and/or crude incidence rates per 1,000,000 population per year. There were 290 malignancies in the period covered, i.e., about 10 new cases per year or 24 per 1,000,000 per year. The average yearly number of overall cancer increased from approximately 1 every two years in newborns to 3-4 per year in adolescents and young adults. Thirty to 35% of patients were Hindustani or Creole; the proportions of Javanese and Maroons patients were about twice and five times, respectively, lower. Leukemias and lymphomas comprised almost half of cases, each occurring 2 to 3 times per year. Bone tumors, soft-tissue sarcomas, and carcinomas were the most common non-hematological malignancies, occurring once or twice per year. Central nervous system tumors, neuroblastoma, retinoblastoma, renal tumors, primary hepatic tumors, and germ cell tumors were exceedingly rare. In conclusion, childhood cancer incidence in Suriname was relatively low; the individual histiotypes displayed an unusual ranking; and there were differences in the sex, age, and ethnic distribution of overall cancer as well as certain histiotypes. However, these observations might be biased by the use of crude rates, and underdiagnosis and incomplete registration of cases due to the absence of specialized (pediatric) cancer facilities in the country.

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Section: Discussionsupporting

confidence: 78%

Section: Introductionmentioning

confidence: 99%

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Childhood Cancer in the Republic of Suriname (1980 Through 2008)

Mans¹,

Zijlmans²

2014

TOEPIJ

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“…Numerous other studies found that, as in the BCCSS, males were more likely than females to be in employment and less likely to be unemployed due to health 4, 5, 18, 19, 20, 21. In the BCCSS, CNS neoplasm survivors were more likely than the majority of other types of childhood cancer to be unable to work due to illness or disability and they were less likely than survivors of leukaemia, Hodgkin's lymphoma, retinoblastoma or the ‘other’ neoplasm group to be in current employment.…”

Section: Discussionmentioning

confidence: 84%

Employment status and occupational level of adult survivors of childhood cancer in Great Britain: The British childhood cancer survivor study

Frobisher

Lancashire

Jenkinson

et al. 2017

Intl Journal of Cancer

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The British Childhood Cancer Survivor Study (BCCSS) provides the first detailed investigation of employment and occupation to be undertaken in a large population‐based cohort. Previous studies have been limited by design issues such as using small numbers of survivors with specific diagnoses, and involved limited assessment of employment status and occupational level. The BCCSS includes 17,981 5‐year survivors of childhood cancer. Employment status and occupational level were ascertained by questionnaire from eligible survivors (n = 14,836). Multivariate logistic regression was used to explore factors associated with employment and occupation, and to compare survivors to their demographic peers in the general population. Employment status was available for 10,257 survivors. Gender, current age, cancer type, radiotherapy, age at diagnosis and epilepsy were consistently associated with being: employed; unable to work; in managerial or non‐manual occupations. Overall, survivors were less likely to be working than expected (OR (99% CI): 0.89 (0.81–0.98)), and this deficit was greatest for irradiated CNS neoplasm survivors (0.34 (0.28–0.41)). Compared to the general population, survivors were fivefold more likely to be unable to work due to illness/disability; the excess was 15‐fold among CNS neoplasm survivors treated with radiotherapy. Overall survivors were less likely to be in managerial occupations than expected (0.85 (0.77–0.94)). However, bone sarcoma survivors were more likely to be in these occupations than expected (1.37 (1.01–1.85)) and also similarly for non‐manual occupations (1.90 (1.37–2.62)). Survivors of retinoblastoma (1.55 (1.20–2.01)) and ‘other’ neoplasm group (1.62 (1.30–2.03)) were also more likely to be in non‐manual occupations than expected.

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“…siblings). 190,191 In the model, the risk of mortality assigned to survivors of ALL was set equal to the general population background all-cause mortality risk profile, with an adjustment for an increased mortality risk among survivors of ALL.…”

Section: Input Assumptionmentioning

confidence: 99%

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

Hettle

Corbett

Hinde

et al. 2017

Health Technol Assess

104

143

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BackgroundThe National Institute for Health and Care Excellence (NICE) commissioned a ‘mock technology appraisal’ to assess whether changes to its methods and processes are needed. This report presents the findings of independent research commissioned to inform this appraisal and the deliberations of a panel convened by NICE to evaluate the mock appraisal.MethodsOur research included reviews to identify issues, analysis methods and conceptual differences and the relevance of alternative decision frameworks, alongside the development of an exemplar case study of chimeric antigen receptor (CAR) T-cell therapy for treating acute lymphoblastic leukaemia.ResultsAn assessment of previous evaluations of regenerative medicines found that, although there were a number of evidential challenges, none was unique to regenerative medicines or was beyond the scope of existing methods used to conceptualise decision uncertainty. Regarding the clinical evidence for regenerative medicines, the issues were those associated with a limited evidence base but were not unique to regenerative medicines: small non-randomised studies, high variation in response and the intervention subject to continuing development. The relative treatment effects generated from single-arm trials are likely to be optimistic unless it is certain that the historical data have accurately estimated the efficacy of the control agent. Pivotal trials may use surrogate end points, which, on average, overestimate treatment effects. To reduce overall uncertainty, multivariate meta-analysis of all available data should be considered. Incorporating indirectly relevant but more reliable (more mature) data into the analysis can also be considered; such data may become available as a result of the evolving regulatory pathways being developed by the European Medicines Agency. For the exemplar case of CAR T-cell therapy, target product profiles (TPPs) were developed, which considered the ‘curative’ and ‘bridging to stem-cell transplantation’ treatment approaches separately. Within each TPP, three ‘hypothetical’ evidence sets (minimum, intermediate and mature) were generated to simulate the impact of alternative levels of precision and maturity in the clinical evidence. Subsequent assessments of cost-effectiveness were undertaken, employing the existing NICE reference case alongside additional analyses suggested within alternative frameworks. The additional exploratory analyses were undertaken to demonstrate how assessments of cost-effectiveness and uncertainty could be impacted by alternative managed entry agreements (MEAs), including price discounts, performance-related schemes and technology leasing. The panel deliberated on the range of TPPs, evidence sets and MEAs, commenting on the likely recommendations for each scenario. The panel discussed the challenges associated with the exemplar and regenerative medicines more broadly, focusing on the need for a robust quantification of the level of uncertainty in the cost-effective estimates and the potential value of MEAs in limiting the exposure of the NHS to high upfront costs and loss associated with a wrong decision.ConclusionsIt is to be expected that there will be a significant level of uncertainty in determining the clinical effectiveness of regenerative medicines and their long-term costs and benefits, but the existing methods available to estimate the implications of this uncertainty are sufficient. The use of risk sharing and MEAs between the NHS and manufacturers of regenerative medicines should be investigated further.FundingThe National Institute for Health Research Health Technology Assessment programme.

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Twenty-five–year follow-up among survivors of childhood acute lymphoblastic leukemia: a report from the Childhood Cancer Survivor Study

Cited by 338 publications

References 65 publications

Childhood Cancer in the Republic of Suriname (1980 Through 2008)

Childhood Cancer in the Republic of Suriname (1980 Through 2008)

Employment status and occupational level of adult survivors of childhood cancer in Great Britain: The British childhood cancer survivor study

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

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