RESUMEN
The study analyzes the current status of personalized medicine in pediatric oncology in Spain. It gathers national data on the tumor molecular studies and genomic sequencing carried out at diagnosis and at relapse, the centers that perform these studies, the technology used and the interpretation and clinical applicability of the results. Current challenges and future directions to achieve a coordinated national personalized medicine strategy in pediatric oncology are also discussed. Next generation sequencing-based (NGS) gene panels are the technology used in the majority of centers and financial limitations are the main reason for not incorporating these studies into routine care. Nowadays, the application of precision medicine in pediatric oncology is a reality in a great number of Spanish centers. However, its implementation is uneven and lacks standardization of protocols; therefore, national coordination to overcome the inequalities is required. Collaborative work within the Personalized Medicine Group of SEHOP is an adequate framework for encouraging a step forward in the effort to move precision medicine into the national healthcare system.
Asunto(s)
Hematología , Neoplasias , Niño , Consenso , Secuenciación de Nucleótidos de Alto Rendimiento , Humanos , Neoplasias/genética , Neoplasias/patología , Neoplasias/terapia , Medicina de Precisión/métodos , EspañaRESUMEN
Rhabdomyosarcoma (RMS) is the most frequent soft tissue sarcoma (STS) in children and adolescents. In Spain the annual incidence is 4.4 cases per million children < 14 years. It is an uncommon neoplasm in adults, but 40% of RMS are diagnosed in patients over 20 years of age, representing 1% of all STS in this age group. RMS can appear anywhere in the body, with some sites more frequently affected including head and neck, genitourinary system and limbs. Assessment of a patient with suspicion of RMS includes imaging studies (MRI, CT, PET-CT) and biopsy. All patients with RMS should receive chemotherapy, either at diagnosis in advanced or metastatic stages, or after initial resection in early local stages. Local control includes surgery and/or radiotherapy depending on site, stage, histology and response to chemotherapy. This guide provides recommendations for diagnosis, staging and treatment of this neoplasm.
Asunto(s)
Tomografía Computarizada por Tomografía de Emisión de Positrones/métodos , Guías de Práctica Clínica como Asunto/normas , Rabdomiosarcoma/terapia , Niño , Terapia Combinada , Humanos , Incidencia , Rabdomiosarcoma/diagnóstico por imagen , Rabdomiosarcoma/epidemiología , Rabdomiosarcoma/patología , España/epidemiologíaRESUMEN
PURPOSE: Early phase trials are crucial in developing innovative effective agents for childhood malignancies. We report the activity in early phase paediatric oncology trials in Spain from its beginning to the present time and incorporate longitudinal data to evaluate the trends in trial characteristics and recruitment rates. METHODS: Members of SEHOP were contacted to obtain information about the open trials at their institutions. The study period was split into two equal periods for analysis: 2007-2013 and 2014-2020. RESULTS: Eighty-one trials and two molecular platforms have been initiated. The number of trials has increased over the time of the study for all tumour types, with a predominance of trials available for solid tumours (66%). The number of trials addressed to tumours harbouring specific molecular alterations has doubled during the second period. The proportion of industry-sponsored compared to academic trials has increased over the same years. A total of 565 children and adolescents were included, with an increasing trend over the study period. For international trials, the median time between the first country study approval and the Spanish competent authority approval was 2 months (IQR 0-6.5). Fourteen out of 81 trials were sponsored by Spanish academic institutions. CONCLUSIONS: The number of available trials, and the number of participating patients, has increased in Spain from 2007. Studies focused on molecular-specific targets are now being implemented. Barriers to accessing new drugs for all ranges of age and cancer diseases remain. Additionally, opportunities to improve academic research are still required in Spain.
Asunto(s)
Ensayos Clínicos como Asunto/estadística & datos numéricos , Oncología Médica/tendencias , Neoplasias/terapia , Pediatría/tendencias , Adolescente , Adulto , Niño , Estudios de Seguimiento , Humanos , Estudios Longitudinales , Neoplasias/patología , Sociedades Médicas , Adulto JovenRESUMEN
BACKGROUND: Neuroblastoma (NB) is a heterogeneous tumor with extremely diverse prognosis according to clinical and genetic factors such as specific combinations of chromosomal imbalances. METHODS: Molecular karyotyping data from a national neuroblastic tumor database of 155 NB samples were analyzed and related to clinical data. RESULTS: Segmental chromosomal alterations (SCA) were detected in 102 NB, whereas 45 only displayed numerical alterations. Incidence of SCA was higher in stage M (92%) and MYCN amplified (MNA) NB (96%). Presence of SCA was associated with older age, especially 1q gain and 3p deletion. 96% of the deaths were observed in the SCA group and 85% of the relapsed NB contained SCA. The alteration most commonly associated with a higher number of other segmental rearrangements was 11q deletion, followed by 4p deletion. Whole-chromosome 19 gain was associated with lower stages, absence of SCA and better outcome. CONCLUSIONS: SCA are not randomly distributed and are concentrated on recurrent chromosomes. The most frequently affected chromosomes identify prognostic factors in specific risk groups. SCA are associated with older age and MNA. We have identified a small subset of patients with better outcome that share whole-chromosome 19 numeric gain, suggesting its use as a prognostic biomarker in NB.
Asunto(s)
Aberraciones Cromosómicas , Neuroblastoma/genética , Adolescente , Adulto , Niño , Preescolar , Humanos , Lactante , Estudios Retrospectivos , Adulto JovenRESUMEN
PURPOSE: The COVID-19 pandemic has forced healthcare stakeholders towards challenging decisions. We analyse the impact of the pandemic on the conduct of phase I-II trials for paediatric cancer during the first month of state of alarm in Spain. METHODS: A questionnaire was sent to all five ITCC-accredited Spanish Paediatric Oncology Early Phase Clinical Trial Units, including questions about impact on staff activities, recruitment, patient care, supply of investigational products, and legal aspects. RESULTS: All units suffered personnel shortages and difficulties in enrolling patients, treatment continuity, or performing trial assessments. Monitoring activity was frequently postponed (73%), and 49% of on-going trials interrupted recruitment. Only two patients could be recruited during this period (75% reduction in the expected rate). CONCLUSIONS: The COVID-19 crisis has significantly impacted clinical research practice and access to innovation for children with cancer. Structural and functional changes are under way to better cope with the expected future restrictions.
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COVID-19/epidemiología , Ensayos Clínicos como Asunto , Neoplasias/terapia , COVID-19/prevención & control , Niño , Humanos , Oncología Médica/organización & administración , Oncología Médica/estadística & datos numéricos , Cuerpo Médico de Hospitales/provisión & distribución , Neoplasias/epidemiología , Atención al Paciente , Selección de Paciente , SARS-CoV-2 , España/epidemiología , Encuestas y CuestionariosRESUMEN
BACKGROUND: Under the ExPO-r-NeT project (European Expert Paediatric Oncology Reference Network for Diagnostics and Treatment), we aimed to identify paediatric oncology tumour boards in Europe to investigate the kind of technologies and logistics that are in place in different countries and to explore current differences between regions. METHODS: A 20-question survey regarding several features of tumor boards was designed. Data collected included infrastructure, organization, and clinical decision-making information from the centres. The survey was distributed to the National Paediatric Haematology and Oncology Societies that forwarded the survey to the sites. For comparative analysis, respondents were grouped into four geographical regions. RESULTS: The questionnaire was distributed amongst 30 countries. Response was obtained from 23 (77%) that altogether have 212 paediatric oncology treating centres. A total of 121 institutions answered (57%). Ninety-one percent of the centres hold multidisciplinary boards; however, international second consultations are performed in 36% and only 15% participate on virtual tumor boards. Videoconferencing facilities and standard operational procedures (SOPs) are available in 49 and 43% of the centres, respectively. There were statistically significant differences between European regions concerning meeting infrastructure and organization/logistics: specific room, projecting equipment, access to medical records, videoconferencing facilities, and existence of SOPs. CONCLUSION: Paediatric tumor boards are a common feature in Europe. To reduce inequalities and have equal access to healthcare, a virtual network is needed. Important differences on the functioning and access to technology between regions in Europe have been observed and need to be addressed.
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Atención a la Salud/normas , Accesibilidad a los Servicios de Salud/normas , Oncología Médica/normas , Neoplasias/terapia , Grupo de Atención al Paciente/normas , Pediatría/normas , Niño , Atención a la Salud/organización & administración , Europa (Continente) , Humanos , Oncología Médica/organización & administración , Neoplasias/diagnóstico , Grupo de Atención al Paciente/organización & administración , Pediatría/organización & administración , Encuestas y CuestionariosRESUMEN
PURPOSE: Multidisciplinary tumour boards (MDTs) are conducted worldwide for the management of patients with cancer, and they deliver a higher standard of care by simultaneously involving different specialists in diagnosis and treatment planning. However, information of paediatric MDTs functioning is scarce. A pilot study was conducted in Spain in the frame of the European Expert Paediatric Oncology Reference Network for Diagnostics and Treatment (ExPO-r-Net). METHODS: A specific questionnaire was designed regarding various features of MDT practice. Data collected included information on the centres and the team, infrastructure for meetings, MDT organization/logistics and clinical decision-making. The survey was distributed to all Paediatric Oncology Units that register patients in the Spanish Registry of Childhood Tumours (RETI-SEHOP). RESULTS: 32 out of 43 contacted centres responded the questionnaire (74 % response rate; 88 % response rate for centres with >25 new patients/year). All units with >25 new patients/year have a dedicated Paediatric MDT compared to 76 % of units with ≤25 new patients/year. MDTs should be improved at institutional level by clear protected time in service planning for all specialists involved, incentives for attendance and attendance registration. Clinical decision-making process and follow-up of recommendation adherence should be assessed and potential legal responsibilities for physicians participating in Tumour Board defined. Network collaboration through virtual MDTs, using available videoconferencing tools, is an opportunity to share expertise among centres.