Efficacy Of Specific Interventions Research Articles

Prescription for change: Unveiling burnout perspectives among pharmacy leaders

Background: Burnout among pharmacists is increasingly pertinent, with growing demand for effective interventions. Burnout can lead to reduced productivity, increased job turnover, medical errors, poor patient satisfaction, and other negative outcomes for patients and providers. Growing attention to burnout in the pharmacy profession highlights the need for personal, organizational, and systemic solutions. However, the uptake and relative efficacy of different approaches remain unclear, particularly within community pharmacy practice. This study sought the viewpoint of community pharmacy leaders (i.e., community pharmacy managers, district managers, franchisees, owners, and executives from various pharmacy organizations) to characterize burnout from their perspectives. Methods: This qualitative study followed a grounded theory approach. Community pharmacy leaders were interviewed using a semistructured format to gather in-depth insights into their experiences and perspectives on burnout and engagement. Results: Sixteen people were interviewed; interviews lasted 30 to 65 minutes, averaging 51 minutes long. Six themes were identified: perceived disconnection between front-line staff and pharmacy decision-makers, overwhelming work demands, cautious optimism toward the expanding scope of pharmacy practice, the importance of employee recognition and appreciation, appropriateness and use of existing work resources, and multimodal, systemic responsibility and solutions to burnout. Conclusion: Addressing burnout requires a multifaceted approach involving personal, organizational, and systemic interventions. Evidence from this study provides valuable insights into the feasibility and efficacy of specific interventions, informing future strategies to enhance workplace well-being and engagement. The study highlights the importance of managing job demands and maximizing resources, emphasizing that personal approaches alone are insufficient and that organizational and systemic interventions are crucial.

#1038 Progressive multifocal leukoencephalopathy in kidney transplant recipients: a retrospective multicenter nationwide cohort study

Abstract Background and Aims Progressive multifocal leukoencephalopathy (PML) is a serious opportunistic infectious disease affecting the central nervous system caused by JC virus. While extensively studied in patients with HIV infection, haematological malignancies, or undergoing immunotherapies, there is a lack of data concerning kidney transplant recipients (KTRs). Method We performed a retrospective, multicentric, nationwide study. We contacted all kidney transplantation centres in France to gather all cases of PML. We obtained the count of patients with a functional kidney allograft using the registry of the French regulatory agency. Results We collected 24 cases of PML. Between 2012 and 2021, the estimated minimal nationwide incidence of PML was 4.7/100 000 patient-years. The median duration from kidney transplantation to PML was 88 months (interquartile range: 9 to 355). Eight patients (33%) had been exposed to belatacept (median exposure: 18.5 months). Almost all patients underwent reduction or withdrawal of immunosuppressive drug doses and 13 (54%) received a specific treatment. Fifteen patients (65%) died within one year after diagnosis. Conclusion In this nationwide study, PML incidence rate in KTRs was at least 4.7/100 000 patient-years resulting in an alarming 65% mortality rate within a year despite immunosuppressants doses decrease or withdrawal. Further investigations are warranted to demonstrate the efficacy of specific treatments and confirm a potential association between belatacept exposure and PML occurrence, as suggested by our study.

Competing risks data in clinical oncology.

Competing risks data analysis plays a critical role in the evaluation of clinical utility of specific cancer treatments and can inform the development of future treatment approaches. Although competing risks data are ubiquitous in cancer studies, competing risks data are infrequently recognized and competing risks data analysis is not commonly performed. Consequently, efficacy of specific treatments is often incompletely and inaccurately presented and thus study results may be interpreted improperly. In the present article, we aim to enhance awareness of competing risks data and provide a general overview and guidance on competing risks data and its analysis using cancer clinical studies.

Cardiac Involvement in Patients With Multisystem Inflammatory Syndrome in Adults.

Multisystemic inflammatory syndrome in adults is a hyperinflammatory condition following (within 4-12 weeks) SARS-CoV-2 infection. Here, the dysregulation of the immune system leads to a multiorgan involvement often affecting the heart. Cardiac involvement in multisystemic inflammatory syndrome in adults has been described mainly in young men without other comorbidities and may present with different clinical scenarios, including acute heart failure, life-threatening arrhythmias, pericarditis, and myocarditis, with a nonnegligible risk of mortality (up to 7% of all cases). The heterogeneity of its clinical features and the absence of a clear case definition make the differential diagnosis with other postinfectious (eg, infective myocarditis) and hyperinflammatory diseases (eg, adult Still disease and macrophage activation syndrome) challenging. Moreover, the evidence on the efficacy of specific treatments targeting the hyperinflammatory response underlying this clinical condition (eg, glucocorticoids, immunoglobulins, and other immunomodulatory agents) is sparse and not supported by randomized clinical trials. In this review article, we aim to provide an overview of the clinical features and the diagnostic workup of multisystemic inflammatory syndrome in adults with cardiac involvement, highlighting the possible pathogenetic mechanisms and the therapeutic management, along with remaining knowledge gaps in this field.

Early life stress and the role of environmental and molecular moderators in the ontology of pathological and resilient behavioral phenotypes

Early life stress (ELS) in the form of trauma or caregiver abuse and neglect is often associated with psychopathology. However, not everyone exposed to ELS develops a pathology; others display resilience, or the ability to adapt and persevere despite ongoing adversity. Several molecular moderator variables between ELS and behavioral phenotypes have been proposed, including single nucleotide polymorphisms (SNPs) and epigenetic markers. Specifically, several SNPs and aberrant methylation or expression of genes associated with neurotransmitter systems and brain-derived neurotrophic factor have been associated with anxiety, depression or schizophrenia. The present review seeks to explore the relationship between SNPs, epigenomics and disease, and offer data to suggest several SNPs may also predict specific treatment efficacy and psychological resilience. Due to these different mental health outcomes as a function of ELS, it is critical that environmental moderators be equally considered in determining the ontology of resilient or pathological phenotypes; this includes the infant-caregiver relationship, and the degree of control, magnitude, and type of the stressor experienced. Finally, we will offer evidence to suggest that several intervention strategies, including drug treatment, environmental enrichment, or exercise can ameliorate many of the psychological, biological, and molecular consequences of ELS exposure, and help shift one toward a resilient phenotype.

Multi-omics analysis of multiple myeloma patients with differential response to first-line treatment.

The genome backgrounds of multiple myeloma (MM) would affect the efficacy of specific treatment. However, the mutational and transcriptional landscapes in MM patients with differential response to first-line treatment remains unclear. We collected paired whole-exome sequencing (WES) and transcriptomic data of over 200 MM cases from MMRF-COMPASS project. R package, maftools was applied to analyze the somatic mutations and mutational signatures across MM samples. Differential expressed genes (DEG) was calculated using R package, DESeq2. The feature selection of the predictive model was determined by LASSO regression. In silico analysis revealed newly discovered recurrent mutated genes such as TTN, MUC16. TP53 mutation was observed more frequent in nonCR (complete remission) group with poor prognosis. DNA repair-associated mutational signatures were enriched in CR patients. Transcriptomic profiling showed that the activity of NF-kappa B and TGF-β pathways was suppressed in CR patients. A transcriptome-based response predictive model was constructed and showed promising predictive accuracy in MM patients receiving first-line treatment. Our study delineated distinctive mutational and transcriptional landscapes in MM patients with differential response to first-line treatment. Furthermore, we constructed a 20-gene predictive model which showed promising accuracy in predicting treatment response in newly diagnosed MM patients.

Occupational Therapy and COVID-19: How the Transition to Telehealth Has Affected Outcomes for Children: A Systematic Review

ABSTRACT This systematic review aimed to evaluate the evidence for the outcomes of telehealth occupational therapy service delivery versus traditional in-person service delivery for children during the peak of the COVID-19 pandemic. Using three academic databases, a search string including the following keywords, telehealth, occupational, and child, returned 825 articles. Five articles were selected for full-text review after screening and employing the inclusion/exclusion criteria. A quality appraisal was completed, leaving two articles in this systematic review. Researchers found that moderate evidence supports the use of telehealth to disseminate occupational therapy service delivery. Researchers also found substantial evidence that supports the use of telehealth in overall satisfaction with services compared to traditional in-person therapy services. This systematic review confirms that telehealth has improved access to occupational therapy services. Further research needs to be conducted on the efficacy of specific interventions in the telehealth setting.

Early life stress and the role of environmental and molecular moderators in the ontology of pathological and resilient behavioral phenotypes

Early life stress (ELS) in the form of trauma or caregiver abuse and neglect is often associated with psychopathology. However, not everyone exposed to ELS develops a pathology; others display resilience, or the ability to adapt and persevere despite ongoing adversity. Several molecular moderator variables between ELS and behavioral phenotypes have been proposed, including single nucleotide polymorphisms (SNPs) and epigenetic markers. Specifically, several SNPs and aberrant methylation or expression of genes associated with neurotransmitter systems and brain-derived neurotrophic factor have been associated with anxiety, depression or schizophrenia. The present review seeks to explore the relationship between SNPs, epigenomics and disease, and offer data to suggest several SNPs may also predict specific treatment efficacy and psychological resilience. Due to this discrepancy in the literature, it is critical that environmental moderators be equally considered in determining the ontology of resilient or pathological phenotypes; this includes the infant-caregiver relationship, and the degree of control, magnitude, and type of the stressor experienced. Finally, we will offer evidence to suggest that several intervention strategies, including drug treatment, environmental enrichment, or exercise can ameliorate many of the psychological, biological, and molecular consequences of ELS exposure, and help shift one toward a resilient phenotype.

Научно обоснованные методы психотерапии для детей дошкольного возраста: краткий обзор для клиницистов

<p>Despite growing consensus that clinical interventions must be supported by empirical evidence, preschool-aged children are more likely than older children to receive services with little to no empirical support. The dissemination of research findings on the efficacy of specific interventions is constrained by clinicians’ limited access to peer-reviewed research journals and treatment manuals. The current paper provides a synthesis on the literature of psychological treatment for children with externalizing or internalizing problems and their parents or families. The review highlights key principles and treatment approaches that are supported by the evidence so that clinicians may readily implement these evidence-based treatments. The approaches that have the most empirical support for externalizing problems are parent behavior management training (PBMT), parent-child interaction therapy (PCIT), and cognitive behavioral therapy (CBT; in individual or group format, including social skills groups). For internalizing problems, research suggests that family-based CBT, group parent CBT (alone or in combination with group child CBT), trauma-focused CBT, and adapted versions of PCIT are the most evidence-supported treatments in preschool children. Common principles and techniques that are shared by a number of evidence-based treatments for preschool-aged children, and recommendations for clinicians and for the research community are discussed.</p>

Cardiac Magnetic Resonance Features of Fabry Disease: From Early Diagnosis to Prognostic Stratification.

In the past few years, the wide application of cardiac magnetic resonance (CMR) significantly changed the approach to the study of cardiac involvement in Fabry Disease (FD). The possibility to perform non-invasive tissue characterization, including new sequences such as T1/T2 mapping, offered a powerful tool for differential diagnosis with other forms of left ventricular hypertrophy. In patients with confirmed diagnosis of FD, CMR is the most sensitive non-invasive technique for early detection of cardiac involvement and it provides new insight into the evolution of cardiac damage, including gender-specific features. Finally, CMR multiparametric detection of subtle changes in cardiac morphology, function and tissue composition is potentially useful for monitoring the efficacy of specific treatment over time. This paper aims to provide a comprehensive review of current knowledge regarding the application of CMR in FD cardiac involvement and its clinical implication.

Efficacy of treatments for neurotrophic keratopathy: a systematic review and meta-analysis.

Management of NK can be difficult, involving a range of treatments with variable efficacy. We conducted a systematic review and meta-analysis to evaluate the efficacy of medical and surgical treatments for neurotrophic keratitis (NK). PubMed, Cochrane Library, Embase, ClinicalTrial.gov, and ScienceDirect were searched for studies assessing efficacy of NK treatments. We computed random-effect meta-analyses on corneal healing, time to complete healing, and visual acuity changes between baselines and after treatment, stratified on treatment classes. We followed the PRISMA guidelines (registration number CRD42021225721). We included 20 studies: 571 patients and 5 treatment classes (2 surgical and 3 non-surgical). The percentage of patients with complete corneal healing did not differ between specific treatments (nerve growth factor eyedrops (NGF), 75%, 95CI 46 to 104%; autologous serum (AS), 92%, 86 to 98%; neurotization, 99%, 95 to 103%; amniotic membrane transplantation (AMT), 86%, 78 to 94%). All specific treatments had better percentage of complete healing (p < 0.001) than non-specific treatment groups, i.e., mainly lubricants (23%, 14 to 32). Time to complete healing was 24.2days (5.4 to 43.1) with NGF, 27.6days (15.2 to 40.0) with AS, 117days (28.8 to 205.2) with neurotization, and 16.4days (11.1 to 21.7) with AMT. Only NGF and AMT improved visual acuity. Efficacy outcomes were not affected by sociodemographic (age, sex) nor severity of disease (Mackie stages). We confirmed the efficacy of specific treatments in NK. Further comparative trials are needed to investigate the medical and economic benefits of innovative therapies.

Preventing persistent postsurgical pain: A systematic review and component network meta-analysis.

Evidence for perioperative methods to prevent persistent postsurgical pain (PPP) is uncertain, in part because few treatments have been directly compared. Here we have used component network meta-analysis (cNMA) to incorporate both direct and indirect evidence in the evaluation of the efficacy and tolerability of pharmacological and neural block treatments. We searched the Cochrane Central Registry of Controlled Trials, Embase, MEDLINE, ClinicalTrials.gov and the World Health Organization International Clinical Trials Registry up to January 2021 for randomized, double-masked, controlled trials that reported the prevalence of PPP. We assessed trial quality with the Cochrane risk of bias tool (RoB 2.0). We analysed the results with frequentist cNMA models. The primary outcome was the relative risk (RR) of PPP. We assessed efficacy in relation to a clinically important effect size of RR=0.9, which is a 10% improvement with treatment. The analysis included 107 trials (13,553 participants) of 13 treatments. The effects of complex interventions were the multiplicative effects of their components. Compared with placebo, serotonin-norepinephrine reuptake inhibitors (SNRIs), neural block alone, or in combination with NMDA receptor blockers or gabapentanoids were effective. Treatments with benefit in the immediate post-operative period predicted a reduced risk of PPP. Several treatments and treatment combinations effectively reduce PPP prevalence. Pain outcomes in the immediate postoperative period are an important mediator of PPP. Multimodal interventions can be analysed using cNMA. Systematic reviews of PPP prevention usually focus on the efficacy of specific treatments in comparison with control interventions. In this study we used component network meta-analysis to compare interventions to each other, including both pharmacological and neural block techniques, and multimodal interventions. Interventions that are not effective alone may improve the efficacy of multimodal interventions that include neural block techniques. Immediate postoperative benefit was an important mediator for reduction of PPP. PROSPERO: CRD42018085570 https://www.crd.york.ac.uk/prospero/.

Addressing Burnout in Urology: A Qualitative Assessment of Interventions.

We characterized physician burnout among urologists to determine the prevalence and efficacy of specific burnout interventions utilized and to determine involvement of workplaces in effective burnout interventions. The Western Section of the American Urological Association created an electronic, 29 question workforce survey. Several questions focused on assessing the level of urologist burnout, prevalence of work sponsored burnout interventions and efficacy of specific interventions. A total of 440 responses were received (25.9% response rate); 82.2% of responders were male. The majority of urologists noted some level of burnout (79.5%) with no significant difference between those who reported no burnout vs some level of burnout (p=0.30). The most commonly tried interventions to reduce burnout were participating in regular physical exercise (76.6%), reading nonmedical literature (67.1%) and decreasing or modifying work hours (52.3%). The interventions most frequently cited as "very effective" were hiring a scribe (62.5%), regular exercise (56.1%) and participating in 1-on-1 gatherings with colleagues outside of work (44.6%). There were no significant differences noted when comparing "very effective" interventions by gender. The interventions most frequently cited as not effective were stress or burnout seminars (26.9%) and meditation/mindfulness training (11.5%); 42.5% reported workplace interventions to help prevent or reduce burnout. Certain practice-changing and personal burnout interventions were noted to be "very effective" in decreasing burnout. Fewer than half of responders noted workplace sponsorship of interventions. Organizational support may lead to increased participation and effectiveness of burnout interventions.

Omega-3 fatty acids as component of nutritional and metabolic treatment of patients with COVID-19 and other viral diseases (literature review)

Nutritional deficit in patients with viral diseases, including COVID-19, can reduce the efficacy of specific treatment, decrease the survival rate, and increase medical expenses. According to international guidelines, timely correction of nutritional status with supplemental enteral and parenteral nutrition containing omega-3 polyunsaturated fatty acid from fish oil (omega-3 PUFAs: ЕРА and DHA) is able to improve clinical outcomes of specific antiviral treatment. The authors conducted an analytical review to assess the results from the study of clinical use of omega-3 PUFAs for the prevention and treatment of COVID-19 and other viral infections. Supplementation of clinical (enteral and parenteral) nutrition with omega-3 PUFAs allows for symptom reduction, shorter stay in hospital, and quicker recovery of patients infected with SARS-CoV-2 and other viruses. When used in combination with adequate macronutrient support, it can reverse nutritional deficit and improve clinical outcomes.

COVID-19 Pandemic and Neurological Disease: A Critical Review of the Existing Literature

Most of what we know about the coronavirus disease 2019 (COVID-19) is limited to what we know about severe acute respiratory syndrome (SARS) and COVID-19's epidemiology, fatality, and acute care. However, infection with COVID-19 may also involve the central nervous system (CNS), which may or may not be due to a multi-organ injury. Our aim in this paper is to briefly summarize the main aspects of the growing literature on neurological manifestations of the COVID-19 infection. As such, after mentioning some general background on the economic and medical implications of the pandemic on individuals, the healthcare system, and the society, we summarize some common aspects of the published literature on neurological manifestations of the COVID-19 infection. We also highlight the existing gaps in the literature, which requires additional work. The most common neurological manifestation of COVID-19 infection is an olfactory deficit. However, it is still unknown if it is inflammatory or degenerative in nature. Still, the incidence of neurological complications, and also their mechanisms and treatments are unknown. This literature is predominantly composed of opinions and reviews rather than original articles, so the patients' data are not used for a majority of the studies. Multi-center studies that not only conduct chest CT or MRI but also brain CT or MRI are needed. Randomized trials are still required on the management of acute and chronic neurological conditions due to COVID-19 infection. Cohort studies may also determine the natural history of the conditions and factors that are prognostic. Furthermore, while disparities in COVID-19 infections are known, inequalities in neurological manifestations are unknown. Besides this, the efficacy of specific treatments on CNS involvement is still unknown. We will discuss the health care needs of patients with chronic neurological conditions. We Included a few recommendations for practice and further research at the end of this paper.

Comparison of baseline patient characteristics in immunotherapy versus targeted therapy trials for advanced melanoma.

e22012 Background: Immune checkpoint inhibitors (ICI) or BRAF/MEK targeted therapy are both FDA approved first line options for BRAF mutant advanced melanoma. However, the optimal sequencing of these approaches and the profile of patients who benefit the most from one treatment over the other is suboptimally defined. Methods: We compared the baseline characteristics of the participants enrolled in the immunotherapy (IT) and targeted therapy (TT) trials quoted in the NCCN guidelines as the guidelines are used by many oncologists who do not subspecialize in melanoma to formulate treatment plans. Data was gathered about age, sex, race, ECOG score, LDH levels, stage, BRAF mutation status, PD-L1 expression and prior lines of therapy. Results: Median age range was younger in the TT population (48-57 y) as compared to the IT population (56-65 y); &gt; 50% were males. 54-82% patients in the IT trials had an ECOG score of 0. Relatively more patients (pts) with ECOG score of 1 were included in Dabrafenib/ Trametinib and Vemurafenib/Cobimetinib trials (62-65%). Most pts had stage IV M1c disease. LDH levels were similar in IT and TT trials. V600E mutation was uniformly more common in the TT trials as expected. PD-L1 expression varied widely; from 23% to 81% patients with a high PD-L1 expression. Trials evaluating pembrolizumab included patients (7-26%) who were previously treated with BRAF/MEK inhibitor, however TT trials did not include as many pts exposed to ICI. Conclusions: Age range and sex ratio of the pts included in the trials were representative of the epidemiology of melanoma, but race was underreported. TT studies trended towards a younger median age range than patients treated in the IT studies. However optimal biomarkers for initial treatment choice cannot be determined by the patient demographics and characteristics presented in the studies discussed in the NCCN guidelines. Studies randomizing treatment sequence and identification of biomarkers prognostic of patient specific treatment efficacy are needed. Comparison of the IT and TT studies support the choice of either approach as initial treatment in pts with BRAF mutant melanoma lacking contraindications.

What Does the Australian General Public Know About Treatments for Dementia? A Population Survey.

Objective: To identify the Australian general population’s awareness regarding the presence and effectiveness of treatments for dementia. Method: An online survey administered through a consumer panel provider (PureProfile). Included were people aged 18 years or above living in Australia. The survey asked participants about their knowledge of treatments for dementia and attitudes toward the effectiveness of evidence-based treatments. Results: Of the 1,001 participants, more than half (63.5%) could not spontaneously name any treatments that improved outcomes for people with dementia. When asked about the efficacy of specific treatments, “brain training” was considered to be “very likely” to be effective by approximately half (49.4%) of the participants followed by “education for caregivers” (46.2%) and “healthy diet” (43.4%). Discussion: Knowledge of treatments for dementia among the Australian public is poor. There is a need to better educate the public about treatments that have demonstrated effectiveness to improve their uptake and use.

Effect of a multinutrient intervention after ischemic stroke in female C57Bl/6 mice.

Stroke can affect females very differently from males, and therefore preclinical research on underlying mechanisms and the effects of interventions should not be restricted to male subjects, and treatment strategies for stroke should be tailored to benefit both sexes. Previously, we demonstrated that a multinutrient intervention (Fortasyn) improved impairments after ischemic stroke induction in male C57Bl/6 mice, but the therapeutic potential of this dietary treatment remained to be investigated in females. We now induced a transient middle cerebral artery occlusion (tMCAo) in C57Bl/6 female mice and immediately after surgery switched to either Fortasyn or an isocaloric Control diet. The stroke females performed several behavioral and motor tasks before and after tMCAo and were scanned in an 11.7 Tesla magnetic resonance imaging (MRI) scanner to assess brain perfusion, integrity, and functional connectivity. To assess brain plasticity, inflammation, and vascular integrity, immunohistochemistry was performed after killing of the mice. We found that the multinutrient intervention had diverse effects on the stroke-induced impairments in females. Similar to previous observations in male stroke mice, brain integrity, sensorimotor integration and neurogenesis benefitted from Fortasyn, but impairments in activity and motor skills were not improved in female stroke mice. Overall, Fortasyn effects in the female stroke mice seem more modest in comparison to previously investigated male stroke mice. We suggest that with further optimization of treatment protocols more information on the efficacy of specific interventions in stroked females can be gathered. This in turn will help with the development of (gender-specific) treatment regimens for cerebrovascular diseases such as stroke. This article is part of the Special Issue "Vascular Dementia".

Validation of an Instrument to Measure Patients' Intentions and Ability to Change Attitudes and Behavior

Background: The efficacy of specific interventions also of mind-body medicine is also dependent on the patients' ability to engage in these interventions and to perceive and change health-affecting attitudes and behaviors. The aim was to validate a 13-item instrument to measure (1) the patients' perception of specific attitudes and behaviors that are assumed to have a negative influence on the health situation, (2) their intention to change them, and (3) the implementation of the intended changes in their life affairs. Patients and Methods: Anonymous cross-sectional survey among 512 patients with chronic pain conditions (mean age 42.6 ± 11.4 years; 58% women) using standardized instruments. Results: After the elimination of 4 items, an explorative factor analysis of the 9 remaining items indicated 2 factors that would explain 57% of the variance: Perceptions/Intentions (5 items; Cronbach's alpha = 0.75) and Ability/ Implementation (4 items; alpha = 0.77). Factor 2 correlated moderately to strongly with internal adaptive coping strategies (AKU) and situational awareness (CPSC), and weakly with mental health (SF-36), life satisfaction (BMLSS), and low depressive symptoms (BDI) and escape from illness (Escape). Factor 1 correlated weakly with age and adaptive coping strategies (AKU). Conclusions: The final 9-item Perception, Intention and Ability to Change (PIAC) scale was approved as a short, practicable and promising instrument, which should be further evaluated in the context of training and intervention programs, particularly with respect to its predictive relevance.

Clinical trials to improve childhood cancer care and survival in sub-Saharan Africa

Over 80% of children with cancer live in low and middle-income countries where survival rates are much lower than high-income countries. Challenges to successful treatment of paediatric cancers in these countries include late presentation, malnutrition, failure to complete treatment and less-intense supportive care leading to increased treatment-related mortality and the need to reduce the intensity of treatment. Clinical trials can contribute to improved care and survival by providing objective information on the number of patients treated, accuracy of diagnosis, causes of treatment failure and the efficacy of specific interventions. Clinical trials can also help to build capacity (salary support and training), improve facilities (equipment) and fund treatment or essential associated costs (social support, nutritional support and follow-up care). In this article, we discuss our experience with clinical trials in Malawi and sub-Saharan Africa with emphasis on the treatment of children with Wilms tumour.