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1.
BMJ Open ; 14(10): e084477, 2024 Oct 11.
Artigo em Inglês | MEDLINE | ID: mdl-39395824

RESUMO

BACKGROUND: Metronomic chemotherapy ('less is more, regularly') could be an alternative to the maximum tolerated dose ('the more, the better') in the chemotherapeutic cancer treatment of high-risk malignant solid extracranial tumours in children or young adults. OBJECTIVE: To evaluate the efficacy of metronomic chemotherapy compared with placebo or stop treatment in paediatric patients with extracranial malignant solid tumours. METHODS: We searched the databases MEDLINE and CENTRAL on 8 September 2023 and included randomised clinical trials (RCTs). Primary outcome was overall survival, and the main outcome measure was the HR. RESULTS: We identified three RCTs with parallel assignment and intention-to-treat analyses of data from 775 people. The studies primarily reported on participants with rhabdomyosarcoma, neuroblastoma and osteosarcoma. The HR favoured the metronomic chemotherapy group (0.75 (95% CI 0.56 to 0.98)). CONCLUSIONS: The evidence base is compatible with a favourable effect of metronomic chemotherapy on children and young adults with high-risk extracranial malignant solid tumours, especially other than bone tumours, when compared with placebo or stop treatment. Statistical heterogeneity is low while clinical heterogeneity is substantial. Thus, the results must be interpreted with caution and applicability of the results is limited. Future RCTs could provide more data on individual tumour entities and subsequently add information on tumour-specific responses. PROSPERO REGISTRATION NUMBER: CRD42023457195.


Assuntos
Administração Metronômica , Neoplasias , Ensaios Clínicos Controlados Aleatórios como Assunto , Humanos , Criança , Neoplasias/tratamento farmacológico , Antineoplásicos/administração & dosagem , Antineoplásicos/uso terapêutico , Osteossarcoma/tratamento farmacológico , Adolescente
2.
JMIR Pediatr Parent ; 7: e58101, 2024 Oct 01.
Artigo em Inglês | MEDLINE | ID: mdl-39352720

RESUMO

Background: The substantial increase in smartphone ownership has led to a rise in mobile health (mHealth) app use. Developing tailored features through mHealth apps creates a pathway to address the health care needs of pediatric patients with cancer and their families who have complex care needs. However, few apps are designed specifically to integrate with pediatric cancer care. Objective: This study reports a systematic search and analysis of mHealth apps available on the Apple App (iOS) and Google Play (Android) stores designed for pediatric cancer through a list of features that serve (1) patients, (2) caregivers, or (3) both audiences. Methods: Following PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines, we reviewed apps for pediatric patients with cancer and caregivers available as of January 30, 2024. We searched the Apple App and Google Play stores with a list of keyword combinations focusing on pediatric cancer care. The inclusion criteria were (1) specifically apps targeted toward pediatric patients with cancer, their families, or both; (2) available in either app store; and (3) available in English. Apps were assessed using the Mobile Application Rating Scale (MARS). The MARS is a quality assessment for mHealth apps, including components of engagement, functionality, aesthetics, and informational quality (5-point Likert scale items-1: low and 5: high quality). Results: In total, 22 apps were identified and 17 of those apps were available on both platforms. The most popular features (n=12) were resource sharing, symptom tracking, reminders, care team connections, journaling, community support, medication tracking, data visualizations, and appointment tracking. Features and interfaces were designed for caregivers (n=9) more frequently than the patients (n=7) while a subset of apps created options for both users (n=6). A total of 16 apps received positive reviews (mean 4.4, SD 0.59; Min=3.1, Max=5.0). A small subset (n=3) achieved over 5000 downloads; however, the majority (n=15) had fewer than 500. More than half (n=12) of the apps were not available in English. Apps requested access to a range of device functionalities to operate (mean 2.72, SD 3.13; Min=0, Max=10). Out of 22, a total of 17 apps were publicly accessible. The mean MARS scores for the apps ranged from 1.71 (SD 0.75) to 4.33 (SD 0.82). Overall, apps scored high on functionality (mean 3.72, SD 0.54) but low on engagement (mean 3.02, SD 0.93). Conclusions: Our review highlights the promising yet underdeveloped potential of mHealth apps in pediatric oncology care, underscoring the need for more inclusive, comprehensive, and integrative digital health solutions. Future developments should actively involve key stakeholders from the pediatric oncology community, including patients, families, and health care professionals, to ensure the apps meet specific needs while addressing linguistic and cultural barriers.

3.
Infect Drug Resist ; 17: 4327-4332, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-39399884

RESUMO

Background: This case report underscores the intricate challenges in managing paediatric patients with acute myeloid leukaemia (AML) undergoing intensive chemotherapy, particularly when complicated by the emergence of multidrug-resistant pathogens such as Carbapenem-Resistant Pseudomonas aeruginosa (CRPA). Case Presentation: An 11-year-old male with AML presented with skin purpura and persistent cough. Clinical and laboratory assessments revealed a high-risk AML profile with genetic mutations, leading to the initiation of intensive chemotherapy per the C-HUANA-AML-2015 protocol. Despite successful disease remission after initial chemotherapy courses, the patient experienced unexpected complications. Notably, septic shock, bone marrow failure, and the emergence of CRPA were encountered during the clinical course. Septic shock occurred following Course B3 chemotherapy, marked by a fever unresponsive to initial antibiotic therapy. Despite negative blood cultures, meropenem and vancomycin were initiated, successfully normalizing temperature. Subsequent challenges included persistent bone marrow suppression, perianal dermatitis, and the identification of CRPA in stool cultures, leading to altered antibiotic therapy guided by minimum inhibitory concentration (MIC) considerations. Whole-genome sequencing (WGS) of the CRPA strain revealed a highly virulent clone (ST-970) with numerous resistance and virulence genes. Conclusion: This case report offers new insights into the complexities of pediatric AML management, with a focus on the emergence of CRPA. The discovery of a high-risk CRPA clone with detailed genomic data underscores the growing challenge of antimicrobial resistance in pediatric oncology. The persistent presence of CRPA and ongoing bone marrow failure highlight the difficulties in managing these complications. This case calls for a reassessment of treatment strategies and encourages further research to improve outcomes in pediatric AML, emphasizing the need for a multidisciplinary approach to address infectious complications and antimicrobial resistance.

4.
BMJ Case Rep ; 17(9)2024 Sep 30.
Artigo em Inglês | MEDLINE | ID: mdl-39349300

RESUMO

Retroperitoneal neuroendocrine tumours are exceptionally rare. The excision of tumours located in the renal hilum near the renal vessels can be challenging. We report a case of a paraganglioma located at the renal hilum which was excised successfully in a child who presented with abdominal pain, breathlessness, left varicocele and hypertension.


Assuntos
Neoplasias Renais , Paraganglioma , Humanos , Neoplasias Renais/cirurgia , Neoplasias Renais/diagnóstico , Neoplasias Renais/diagnóstico por imagem , Paraganglioma/cirurgia , Paraganglioma/diagnóstico por imagem , Paraganglioma/diagnóstico , Paraganglioma/complicações , Masculino , Criança , Dor Abdominal/etiologia , Tomografia Computadorizada por Raios X , Hipertensão , Varicocele/cirurgia , Varicocele/diagnóstico
5.
Pharmaceutics ; 16(9)2024 Sep 14.
Artigo em Inglês | MEDLINE | ID: mdl-39339248

RESUMO

Personalised medicine, facilitated by advancements like 3D printing, may offer promise in oncology. This scoping review aims to explore the applicability of 3D printing for personalised pharmaceutical dosage forms in paediatric cancer care, focusing on treatment outcomes and patient experiences. Following the Joanna Briggs Institute (JBI) methodology, a comprehensive search strategy was implemented to identify the relevant literature across databases including PubMed, Embase, and Web of Science. Three independent reviewers conducted study selection and data extraction, focusing on studies involving paediatric patients under 18 years old and pharmaceutical dosage forms manufactured using 3D printing technology. From 2752 records screened, only six studies met the inclusion criteria, none of which specifically targeted paediatric cancer patients. These studies examined aspects of acceptability, including swallowability, taste, and feasibility of 3D-printed formulations for children. While the studies demonstrated the potential benefits of 3D printing in paediatric medication, particularly in personalised dosing, there is a notable lack of evidence addressing its acceptability in paediatric cancer patients. Further interdisciplinary collaborative research is needed in this area to fully assess preferences and acceptability among children with cancer and their parents or caregivers.

6.
Eur J Oncol Nurs ; 72: 102683, 2024 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-39305738

RESUMO

PURPOSE: Throughout a child's cancer treatment, health professionals (HPs) constitute an important source of support for the entire family. However, the understanding of their presence and essential attributes is unclear. This study explored HPs' presence and attributes in connecting with parents and identified facilitators and barriers for connectedness. METHODS: This qualitative study was undertaken in a compassion paradigm, designed and guided by Heidegger's and Gadamer's philosophical concepts, and employed compassionate methods. Data were generated through ethnographic fieldwork (144 h), parent interviews (n = 16), and focus group interviews with parents of cancer survivors (n = 2) and HPs (n = 3). Inductive content analysis was utilised to analyse data. RESULTS: Many HP-parent contacts developed into close, genuine connections based on HPs' great commitment and ability to balance the act of closeness and distance. This involved HPs' sensitivity, humanity, humility, honest communication, genuine interest, and high clinical competencies; all promoting trust. Adapting and ending close relationships when approaching the end of treatment had little attention and was difficult for families, making some find ways of keeping contact on a personal level. Barriers disclosed were structural work changes, busyness, dishonest, poor, or lack of communication, and poor or lack of interpersonal chemistry. CONCLUSION: Human interconnectedness is powerful in long-term professional relationships and strengthens the parents. More research and clinical attention are needed to develop the understanding and help target actions toward building, maintaining, and ending relationships. Further, cultivating being present in the moment, through mindfulness and compassion, may support HPs in maintaining a receptive mind and a caring role.


Assuntos
Empatia , Neoplasias , Pais , Relações Profissional-Família , Pesquisa Qualitativa , Humanos , Feminino , Masculino , Criança , Pais/psicologia , Neoplasias/psicologia , Neoplasias/terapia , Adulto , Grupos Focais , Pessoa de Meia-Idade , Adolescente , Pré-Escolar , Sobreviventes de Câncer/psicologia
7.
BMJ Case Rep ; 17(8)2024 Aug 31.
Artigo em Inglês | MEDLINE | ID: mdl-39216889

RESUMO

22q11.2 deletion syndrome is a condition with complex multisystem involvement, and many clinicians will encounter patients living with the condition. 22q11.2 deletion syndrome is known to significantly increase the risk of psychosis, and there is some emerging evidence that 22q11.2 deletion syndrome may be associated with an increased risk of malignancy. We report on a case of an adolescent female who had a delayed diagnosis of 22q11.2 deletion syndrome after she developed severe psychosis at an early age. She was subsequently diagnosed in late adolescence with papillary thyroid carcinoma. This case contributes to the limited body of evidence regarding the treatment of psychosis secondary to 22q11.2 deletion syndrome and the potential increased risk of malignancy associated with the genetic condition.


Assuntos
Síndrome de DiGeorge , Esquizofrenia , Câncer Papilífero da Tireoide , Neoplasias da Glândula Tireoide , Humanos , Feminino , Câncer Papilífero da Tireoide/diagnóstico , Câncer Papilífero da Tireoide/genética , Câncer Papilífero da Tireoide/complicações , Neoplasias da Glândula Tireoide/diagnóstico , Neoplasias da Glândula Tireoide/genética , Síndrome de DiGeorge/complicações , Síndrome de DiGeorge/diagnóstico , Adolescente , Esquizofrenia/complicações , Carcinoma Papilar/diagnóstico , Diagnóstico Tardio
8.
BMJ Case Rep ; 17(8)2024 Aug 31.
Artigo em Inglês | MEDLINE | ID: mdl-39216892

RESUMO

Hepatocellular carcinoma (HCC) is an extremely rare long-term complication of Budd-Chiari syndrome (BCS) which may occur due to long-term venous congestion causing fibrosis, cirrhosis and subsequent hepatocellular dysplasia or anaplasia. This complication is even rarer in paediatric BCS and warrants early diagnosis for a favourable prognosis. Benign regenerative nodules seen with BCS are difficult to differentiate from malignant nodular lesion of HCC, thereby making serial imaging less sensitive for early diagnosis of HCC in BCS. Surveillance guidelines like adults do not exist in monitoring chronic paediatric BCS due to rarity of this complication. Six monthly serum alpha-fetoprotein monitoring in addition to radiological surveillance improves the sensitivity of early detection of HCC transformation in BCS and should be the way ahead in paediatric BCS as well. We describe a paediatric patient who presented with advanced HCC after 25-month follow-up for BCS.


Assuntos
Síndrome de Budd-Chiari , Carcinoma Hepatocelular , Neoplasias Hepáticas , Humanos , Síndrome de Budd-Chiari/etiologia , Síndrome de Budd-Chiari/diagnóstico , Carcinoma Hepatocelular/diagnóstico , Carcinoma Hepatocelular/complicações , Carcinoma Hepatocelular/diagnóstico por imagem , Neoplasias Hepáticas/complicações , Neoplasias Hepáticas/diagnóstico , Neoplasias Hepáticas/diagnóstico por imagem , Masculino , Criança , alfa-Fetoproteínas/análise , alfa-Fetoproteínas/metabolismo , Tomografia Computadorizada por Raios X
10.
Pediatr Blood Cancer ; 71(11): e31147, 2024 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-39185694

RESUMO

INTRODUCTION: Accessing compassionate access schemes to obtain novel therapeutic agents for children with hard-to-treat cancers can be fraught with challenges such as regulatory barriers and limited resources. This study aimed to explore clinician perspectives on the barriers, impacts and ethical considerations of accessing novel therapeutic agents within the context of a paediatric oncology precision medicine trial. METHODS: We gathered data from 37 semi-structured interviews with paediatric oncologists participating in the PRecISion Medicine for Children with Cancer (PRISM) study, a precision medicine clinical trial in Australia. The interviews, conducted over 2 years, focused on paediatric oncologist's experiences with the PRISM trial. Interviews were re-analysed to identify themes related to access pathways and any challenges in obtaining novel agents through thematic analysis. The resulting thematic framework was discussed and refined by a multidisciplinary team. RESULTS: Three main themes were identified: (i) barriers to access, including poor drug availability, lack of evidence and the time burden of the application process; (ii) impacts of inaccessibility, encompassing medical consequences and financial burden on families; and (iii) ethical considerations, centred around balancing realistic expectations and providing compassionate care to patients and families. Paediatric oncologists expressed frustration with the complex regulatory landscape and the lack of systematic reporting on applications and outcomes of obtaining novel agents. Lengthy wait times for decision notifications were also highlighted, raising concerns about missed therapeutic opportunities for patients. CONCLUSION: This study provides insight to the challenges faced when seeking access to novel therapies for paediatric oncology patients. There is a clear need for improved communication, streamlining processes and increased resources to facilitate access to novel agents. Further resource development is necessary to address these complexities in accessing novel therapy agents to ultimately ensure equitable and timely access.


Assuntos
Acessibilidade aos Serviços de Saúde , Neoplasias , Medicina de Precisão , Humanos , Neoplasias/terapia , Criança , Feminino , Masculino , Oncologistas , Austrália
11.
Pediatr Blood Cancer ; 71(11): e31299, 2024 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-39189659

RESUMO

Childhood cancer treatment in Africa has a dramatically increasing patient population resulting in greater rehabilitation needs. The International Society of Paediatric Oncology (SIOP) mapped childhood cancer services in Africa including access to physiotherapy. Irrespective of income classification, just over two-thirds of countries in Africa reported having access to physiotherapy services in paediatric oncology sites. There is a lack of knowledge about African childhood physiotherapy services. Research is needed to understand the rehabilitation needs of these children/adolescents and how to meet their needs in a globally equitable and sustainable way.


Assuntos
Oncologia , Neoplasias , Modalidades de Fisioterapia , Humanos , Criança , África , Neoplasias/terapia , Neoplasias/reabilitação , Acessibilidade aos Serviços de Saúde , Sociedades Médicas , Pediatria , Adolescente
12.
BMJ Open ; 14(8): e087343, 2024 Aug 19.
Artigo em Inglês | MEDLINE | ID: mdl-39160096

RESUMO

OBJECTIVES: Childhood cancer survivors may experience complex health issues during transition and long-term follow-up (LTFU); therefore, high-quality healthcare is warranted. Care coordination is one of the essential concepts in advanced healthcare. Care coordination models vary among childhood cancer survivors in transition and LTFU. This study aimed to identify care coordination models for childhood cancer survivors in transition and LTFU and synthesise essential components of the models. DESIGN: This scoping review was guided by the methodological framework from Arksey and O'Malley and was reported with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews. A systematic literature search was conducted on six databases using possible combinations of terms relevant to childhood cancer survivors, transition/LTFU and care coordination model. Data were analysed by descriptive and content analysis. DATA SOURCES: The literature search was first conducted in May 2023 and updated in May 2024. Six databases including Medline, PubMed, Embase, Web of Science, CINAHL and Cochrane Library were searched; meanwhile, a hand search was also conducted. ELIGIBILITY CRITERIA FOR SELECTING STUDIES: Studies relevant to describing any models, interventions or strategies about care coordination of transition or LTFU healthcare services among childhood cancer survivors were included. DATA EXTRACTION AND SYNTHESIS: Two reviewers independently screened and included studies. Basic information as well as care coordination model-related data in the included studies were extracted. Descriptive summary and content analysis were used for data analysis. RESULTS: In the 20 545 citations generated by the search strategy, seven studies were identified. The critical determinants of the models in the included studies were the collaboration of the multidisciplinary team, integration of the navigator role and the provision of patient-centred, family-involved, needs-oriented clinical services. The main functions of the models included risk screening and management, primary care-based services, psychosocial support, health education and counselling, and financial assistance. Models of care coordination were evaluated at patient and clinical levels. Based on this review, core concepts of successful care coordination models for childhood cancer survivors in transition or LTFU were synthesised and proposed as the '3 I' framework: individualisation, interaction and integration. CONCLUSION: This scoping review summarised core elements of care coordination models for childhood cancer survivors' transition and LTFU. A proposed conceptual framework to support and guide the development of care coordination strategies for childhood cancer survivors' transition and LTFU care was developed. Future research is needed to test the proposed model and develop appropriate care coordination strategies for providing high-quality healthcare for childhood cancer survivors' transition and LTFU.


Assuntos
Sobreviventes de Câncer , Humanos , Criança , Continuidade da Assistência ao Paciente/organização & administração , Neoplasias/terapia , Transição para Assistência do Adulto/organização & administração
13.
Cancers (Basel) ; 16(15)2024 Jul 29.
Artigo em Inglês | MEDLINE | ID: mdl-39123424

RESUMO

Large datasets in paediatric oncology are inherently rare. Therefore, it is paramount to fully exploit all available data, which are distributed over several resources, including biomaterials, images, clinical trials, and registries. With privacy-preserving record linkage (PPRL), personalised or pseudonymised datasets can be merged, without disclosing the patients' identities. Although PPRL is implemented in various settings, use case descriptions are currently fragmented and incomplete. The present paper provides a comprehensive overview of current and future use cases for PPRL in paediatric oncology. We analysed the literature, projects, and trial protocols, identified use cases along a hypothetical patient journey, and discussed use cases with paediatric oncology experts. To structure PPRL use cases, we defined six key dimensions: distributed personalised records, pseudonymisation, distributed pseudonymised records, record linkage, linked data, and data analysis. Selected use cases were described (a) per dimension and (b) on a multi-dimensional level. While focusing on paediatric oncology, most aspects are also applicable to other (particularly rare) diseases. We conclude that PPRL is a key concept in paediatric oncology. Therefore, PPRL strategies should already be considered when starting research projects, to avoid distributed data silos, to maximise the knowledge derived from collected data, and, ultimately, to improve outcomes for children with cancer.

14.
J Pediatr Nurs ; 78: e187-e198, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-39025711

RESUMO

PROBLEM: The terminal phase of childhood cancer poses profound physical and mental challenges for children, simultaneously influencing parents and rendering them particularly susceptible to psychosocial issues. ELIGIBILITY CRITERIA: This review included studies exploring the experiences of either: (1) paediatric terminal oncology patients aged under 18 years, (2) parents with a child facing terminal cancer undergoing palliative care, or (3) parents with a child who had undergone palliative care and died. English language, qualitative journal studies or grey literature of any care settings, geographical locations and publication years were included. Studies exploring the experiences of (1) paediatric terminal oncology not receiving palliative care from qualified healthcare professionals, and (3) non-biological parents or non-parental family members, were excluded. SAMPLE: A total of 22 studies were included, published between January 2000 and December 2023. Seventy-two children (aged between 5 and 18 years old) and 236 parents (aged between 24 and 57 years old) participated across all studies. Palliative care settings mostly comprised oncology centres, hospitals and homes. RESULTS: Two themes were identified from the 22 included studies: (1) Navigating rough waters and enduring hardships, and (2) Preparing for end-of-life amidst the looming threat of death. CONCLUSIONS: This review underscored the importance of integrating palliative childhood cancer care in a holistic, age-specific, family-centred, person-centred and timely manner. IMPLICATIONS: Paediatric oncology nurses should attend to physical and psychosocial needs of children and parents, fostering familial and social ties while recognising cultural and spiritual needs. Future research could recruit participants of varying ages, genders, and cultures.


Assuntos
Neoplasias , Cuidados Paliativos , Pais , Pesquisa Qualitativa , Adolescente , Adulto , Criança , Pré-Escolar , Feminino , Humanos , Masculino , Neoplasias/enfermagem , Neoplasias/psicologia , Cuidados Paliativos/psicologia , Pais/psicologia , Assistência Terminal/psicologia , Pessoa de Meia-Idade
15.
BMJ Open ; 14(7): e081823, 2024 Jul 11.
Artigo em Inglês | MEDLINE | ID: mdl-38991689

RESUMO

OBJECTIVES: In our study, we aimed to characterise adult childhood cancer survivors (ACCS), assess their health issues, gauge health-related quality of life (HRQOL) and evaluate visit satisfaction. DESIGN: Prospective cohort study using data from clinical visits and questionnaires. SETTING: Interdisciplinary follow-up programme for ACCS based on the long-term follow-up (LTFU) guidelines of the Children's Oncology Group and overseen by internists in two Swiss hospitals. PARTICIPANTS: ACCS attending our LTFU clinics between April 2017 and January 2022 were eligible. INTERVENTIONS: We documented medical history, current health status and assessed HRQOL using Short Form-36 V.2, comparing it with Swiss general population (SGP) norms (T mean=50, SD=10; age stratified). 3 months post visit, a feedback questionnaire was distributed. MAIN RESULTS: Among 102 ACCS (mean age: 32 years (range: 18-62 years), 68% women), 43 had no prior follow-up (36 ACCS>28 years, 7 ACCS≤28 years). A notable 94% had health issues, affecting an average of 6.1 (SD=3.3) organ systems. HRQOL was lower in ACCS>28 years than the SGP>28 years (physical: 44.8 (SD=11.65) vs 49.3 (SD=10.29), p=0.016; mental: 44.4 (SD=13.78) vs 50.53 (SD=9.92), p=0.004). Older ACCS (>28 years) reported inferior physical (44.8 vs 50.1 (SD=9.30), p=0.017) and mental HRQOL (44.4 vs 50.3 (SD=7.20), p=0.009) than younger ACCS. The majority of respondents reported high levels of satisfaction with the consultation, exceeding 90%. CONCLUSION: ACCS attending LTFU clinics face diverse health issues impacting multiple organ systems and exhibit lower HRQOL compared with the SGP. Thus, internist-led LTFU clinics are crucial for optimising follow-up care.


Assuntos
Sobreviventes de Câncer , Neoplasias , Qualidade de Vida , Humanos , Feminino , Masculino , Sobreviventes de Câncer/psicologia , Estudos Prospectivos , Adulto , Suíça , Pessoa de Meia-Idade , Adolescente , Adulto Jovem , Neoplasias/psicologia , Neoplasias/terapia , Inquéritos e Questionários , Satisfação do Paciente , Seguimentos , Nível de Saúde
17.
J Hosp Infect ; 152: 56-65, 2024 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-39004189

RESUMO

BACKGROUND: Taurolidine-citrate(-heparin) lock solutions (TCHL) are suggested as a promising and safe method for the prevention of central-line-associated bloodstream infections (CLABSI). AIM: To investigate the efficacy of TCHL for the prevention of CLABSI in paediatric oncology patients. METHODS: An assessor-blinded randomized controlled trial at the Princess Máxima Centre for paediatric oncology, the Netherlands, was performed from 2020 to 2023. Paediatric oncology patients receiving a tunnelled central venous access device (CVAD) were eligible. A total of 462 patients were required to compare the TCHL to the heparin-only lock (HL). Patients were followed-up for the first 90 days after CVAD insertion. The primary outcome was the incidence of the first CLABSI from CVAD insertion until the end of follow-up. Intention-to-treat and per-protocol analyses were performed. FINDINGS: In total, 232 were randomized in the HL and 231 in the TCHL group. A total of 47 CLABSIs were observed. The intention-to-treat analysis showed that a CLABSI was observed in 26 (11.2%) of the HL group patients versus 21 (9.1%) of the TCHL group patients; incidence rate ratio (IRR) of 0.81 (95% confidence interval (CI): 0.46-1.45) in favour of the TCHL group. The per-protocol analysis showed that a CLABSI was observed in 10 (7.9%) of the HL group patients versus 6 (4.8%) of the TCHL group patients; IRR of 0.59 (95% CI: 0.21-1.62) in favour of the TCHL group. Adverse events were more common in the TCHL group but rarely reported. CONCLUSION: No difference was detected between the TCHL and HL in the incidence of CLABSI in paediatric oncology patients.


Assuntos
Infecções Relacionadas a Cateter , Cateterismo Venoso Central , Heparina , Taurina , Tiadiazinas , Humanos , Infecções Relacionadas a Cateter/prevenção & controle , Infecções Relacionadas a Cateter/epidemiologia , Tiadiazinas/administração & dosagem , Tiadiazinas/uso terapêutico , Taurina/análogos & derivados , Taurina/administração & dosagem , Masculino , Feminino , Criança , Heparina/administração & dosagem , Pré-Escolar , Países Baixos , Lactente , Cateterismo Venoso Central/efeitos adversos , Neoplasias/complicações , Ácido Cítrico/administração & dosagem , Adolescente , Incidência , Cateteres Venosos Centrais/efeitos adversos , Resultado do Tratamento
18.
Eur J Cancer ; 207: 114145, 2024 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-38936103

RESUMO

Phosphatidylinositol 3-kinase (PI3-K) signalling pathway is a crucial path in cancer for cell survival and thus represents an intriguing target for new paediatric anti-cancer drugs. However, the unique clinical toxicities of targeting this pathway (resulting in hyperglycaemia) difficulties combining with chemotherapy, rarity of mutations in childhood tumours and concomitant mutations have resulted in major barriers to clinical translation of these inhibitors in treating both adults and children. Mutations in PIK3CA predict response to PI3-K inhibitors in adult cancers. The same mutations occur in children as in adults, but they are significantly less frequent in paediatrics. In children, high-grade gliomas, especially diffuse midline gliomas (DMG), have the highest incidence of PIK3CA mutations. New mutation-specific PI3-K inhibitors reduce toxicity from on-target PI3-Kα wild-type activity. The mTOR inhibitor everolimus is approved for subependymal giant cell astrocytomas. In paediatric cancers, mTOR inhibitors have been predominantly evaluated by academia, without an overall strategy, in empiric, mutation-agnostic clinical trials with very low response rates to monotherapy. Therefore, future trials of single agent or combination strategies of mTOR inhibitors in childhood cancer should be supported by very strong biological rationale and preclinical data. Further preclinical evaluation of glycogen synthase kinase-3 beta inhibitors is required. Similarly, even where there is an AKT mutation (∼0.1 %), the role of AKT inhibitors in paediatric cancers remains unclear. Patient advocates strongly urged analysing and conserving data from every child participating in a clinical trial. A priority is to evaluate mutation-specific, central nervous system-penetrant PI3-K inhibitors in children with DMG in a rational biological combination. The choice of combination, should be based on the genomic landscape e.g. PTEN loss and resistance mechanisms supported by preclinical data. However, in view of the very rare populations involved, innovative regulatory approaches are needed to generate data for an indication.


Assuntos
Glicogênio Sintase Quinase 3 beta , Neoplasias , Proteínas Proto-Oncogênicas c-akt , Serina-Treonina Quinases TOR , Humanos , Criança , Adolescente , Neoplasias/tratamento farmacológico , Neoplasias/genética , Glicogênio Sintase Quinase 3 beta/antagonistas & inibidores , Glicogênio Sintase Quinase 3 beta/metabolismo , Proteínas Proto-Oncogênicas c-akt/antagonistas & inibidores , Proteínas Proto-Oncogênicas c-akt/metabolismo , Serina-Treonina Quinases TOR/antagonistas & inibidores , Inibidores de Fosfoinositídeo-3 Quinase/uso terapêutico , Inibidores de Fosfoinositídeo-3 Quinase/farmacologia , Inibidores de MTOR/uso terapêutico , Inibidores de MTOR/farmacologia , Inibidores de Proteínas Quinases/uso terapêutico , Inibidores de Proteínas Quinases/farmacologia , Antineoplásicos/uso terapêutico , Antineoplásicos/farmacologia , Transdução de Sinais/efeitos dos fármacos
19.
BMJ Case Rep ; 17(6)2024 Jun 04.
Artigo em Inglês | MEDLINE | ID: mdl-38834309

RESUMO

Rhabdomyosarcomas are the most common soft-tissue sarcomas, found usually in the younger age group. Histologically, they are subdivided into embryonal, alveolar, pleomorphic and not otherwise specified. They have a heterogenous appearance on imaging with few additional characteristic features based on the subtype. Botryoid variant of embryonal rhabdomyosarcoma commonly involves the genitourinary and the biliary system. They can be multifocal. Most of these lesions have a heterogenous appearance on imaging with areas of necrosis and haemorrhage. On ultrasound, they are polypoidal with cystic areas and are vascular. The lesions are hyperintense on T2 sequences, isointense to the skeletal muscle on T1 sequences and show heterogenous enhancement. Surgery is the mainstay of treatment along with radiotherapy or chemotherapy depending on the site and the stage of the tumour. We report a case of botryoid variant of rhabdomyosarcoma involving the vagina and the urinary bladder.


Assuntos
Rabdomiossarcoma Embrionário , Neoplasias da Bexiga Urinária , Neoplasias Vaginais , Feminino , Humanos , Imageamento por Ressonância Magnética , Rabdomiossarcoma Embrionário/patologia , Rabdomiossarcoma Embrionário/diagnóstico , Rabdomiossarcoma Embrionário/diagnóstico por imagem , Rabdomiossarcoma Embrionário/cirurgia , Ultrassonografia , Neoplasias da Bexiga Urinária/diagnóstico por imagem , Neoplasias da Bexiga Urinária/patologia , Neoplasias Vaginais/patologia , Neoplasias Vaginais/diagnóstico por imagem , Neoplasias Vaginais/cirurgia , Neoplasias Vaginais/diagnóstico , Pré-Escolar
20.
Indian J Palliat Care ; 30(2): 163-167, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-38846133

RESUMO

Objectives: The burden of advanced and metastatic cancer is high among children in developing countries, and palliative care (PC) services for children are sparsely available and poorly accessed. To estimate the burden of PC requirements in children with metastatic neuroblastoma (NB), and to evaluate the PC services offered. Materials and Methods: Retrospective analysis of case records of children 1-14 years diagnosed with metastatic NB from 1 January 2008 to 31 December 2017. Results: One hundred and nineteen patients with metastatic NB were included, of which 87 patients received PC consultation. Early PC referral occurred only in 13 patients (14.9%), and pain was the most prominent symptom. Shifting of care from oncology to PC occurred at disease relapse in 58 patients (66.6%) and at end-of-life in 16 patients (18.3%). Nausea/vomiting, constipation and abdominal distension were the most common symptoms during end-of-life. Seventy-one patients (85%) died of disease, median time to death being 9 months from diagnosis and 4 months from relapse. The mean time from initiation of PC to death was 4.2 months. Conclusion: Timely integration of PC and shared care incorporating the oncology team, PC team and local paediatricians can ease out transition in care, ensure a continuum of care and improve the quality of treatment delivered to children with metastatic cancer.

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