A clinical randomized controlled trial (RCT) is an experimental research study in which subjects are randomly allocated to receive or not receive an experimental preventative, therapeutic or diagnostic procedure, and then are followed to determine the effect of the intervention. RCTs are considered the gold standard for clinical research design. However, in order for valid conclusions and appropriate recommendations to be made for clinical practice, the research needs to be methodologically sound and reported on in sufficient detail to allow reproducibility and critical appraisal. This article will review the fundamental components of a RCT for the prospective investigator as well as for those attempting to implement evidence-based therapy and rehabilitation practice based on reports of RCTs.
Background . Pulmonary function tests (PFTs) objectively measure the extent and progression of cystic fibrosis (CF) lung disease. The rate of lung function decline in developing countries has not previously been studied. Aim . To investigate the average annual rates of pulmonary function decline in South African children with CF from 1999 to 2006. Methodology : The medical records and best PFT over 3-monthly intervals of children attending the CF clinic at Red Cross War Memorial Children’s Hospital, Cape Town, were retrospectively reviewed and analysed using the mixed model regression method. Results . A total of 1 139 PFT were recorded on 79 patients, with a median (interquartile range) of 14 (6 - 21) PFTs per patient. The mean (standard error) forced expiratory volume in 1 second (FEV1) at age 6 years was estimated at 73.83 (3.34) per cent predicted with an FEV1 decline of 0.23 (0.43)% per annum. FEV1 at age 6 was affected by age at CF diagnosis, genotype, and year of birth. Rate of FEV1 decline was significantly affected by Pseudomonas aeruginosa colonisation and genotype. Conclusions . Although FEV1 at age 6 years was low compared with developed countries, the annual rate of FEV1 decline in South African children with CF was minimal, setting the scene for improved survival in this population.
To describe the clinical presentation and outcomes of hospitalised patients infected with human metapneumovirus (hMPV) and human respiratory syncytial virus (hRSV) in a tertiary hospital in Cape Town, South Africa.hMPV was identified in 17 respiratory specimens submitted for viral studies during the period 2001-2003. These patients' medical folders were retrospectively reviewed for clinical, radiological and laboratory data, together with a convenience sample of 20 hRSV-infected patients.hMPV-infected patients were older than those infected with hRSV (P = 0.04) and required a longer hospital stay (P = 0.02). Presenting clinical signs and symptoms were similar between groups. Fourteen (87.5%) hMPV- and 16 (80%) hRSV-infected patients presented with co-morbid and/or immunosuppressive conditions (P > or = 0.5). The most common abnormalities on chest radiographs in both groups were bronchial wall thickening, focal consolidation and atelectasis. Six (37.5%) hMPV- and 11 (55%) hRSV-infected patients required admission to the paediatric intensive care unit (P > 0.1) with five (31.3%) hMPV- and eight (40%) hRSV-infected patients requiring intubation and ventilation (P > 0.5). Three (18.7%) hMPV-patients and three (15%) hRSV-infected patients died during this admission (P > 0.5). All hMPV-infected patients who died had significant co-morbid conditions.These data confirm that hMPV is a significant respiratory pathogen in this setting, with similar presentation and outcome to hRSV infection. This is the largest report of hMPV infection causing significant morbidity, prolonged hospital stay and death, associated with underlying risk factors.
Introduction: Temporal trends in CF survival from low-middle-income settings are poorly reported. We describe changes in CF survival after diagnosis over 40 years from a South African (SA) CF center. Methods: An observational cohort study of people diagnosed with CF from 1974 to 2019. Changes in age-specific mortality rates from the year 2000 (versus before 2000) were estimated using multivariable Poisson regression. Data were stratified by current age < or ≥ 10 years and models controlled for diagnosis age, sex, ethnicity, genotype, and P. aeruginosa (PA) infection. A second analysis explored association of mortality with weight and FEV1z-scores at age 5-8 years. Results: 288 people (52% male; 57% Caucasian; 44% p.Phe508del homozygous) were included (median diagnosis age 0.5 years: Q1,Q3: 0.2, 2.5); 58 (35%) died and 30 (10%) lost to follow-up. Among age >10 years, age-specific mortality from year 2000 was significantly lower (adjusted hazard ratio aHR: 0.14; 95% CI: 0.06,0.29; p<0.001), but not among age <10 years (aHR: 0.67; 95% CI: 0.28,1.64; p=0.383). In children <10 years, Caucasian ethnicity was associated with lower mortality (aHR 0.17; 95% CI 0.05,0.63), and time since first PA infection with higher mortality (aHR 1.31; 95% CI 1.01,1.68). Mortality was 7-fold higher if FEV1z was < -2.0 at age 5-8 years (aHR 7.64; 95% CI 2.58,22.59). Conclusion: Overall, CF survival has significantly improved in SA from year 2000 in people older than 10 years. However, increased risk of mortality persists in young non-Caucasian children, and with FEV1z<-2.0 at age 5-8 years.
Abstract Background Kidney transplantation remains the treatment of choice for children with kidney failure (KF). In South Africa, kidney replacement therapy (KRT) is restricted to children eligible for transplantation. This study reports on the implementation of the Paediatric Feasibility Assessment for Transplantation (pFAT) tool, a psychosocial risk score developed in South Africa to support transparent transplant eligibility assessment in a low‐resource setting. Methods Single‐center retrospective descriptive analysis of children assessed for KRT using pFAT tool from 2015 to 2021. Results Using the pFAT form, 88 children (median [range] age 12.0 [1.1 to 19.0] years) were assessed for KRT. Thirty (34.1%) children were not listed for KRT, scoring poorly in all domains, and were referred for supportive palliative care. Fourteen of these 30 children (46.7%) died, with a median survival of 6 months without dialysis. Nine children were reassessed and two were subsequently listed. Residing >300 km from the hospital ( p = .009) and having adherence concerns ( p = .003) were independently associated with nonlisting. Of the 58 (65.9%) children listed for KRT, 40 (69.0%) were transplanted. One‐year patient and graft survival were 97.2% and 88.6%, respectively. Only one of the four grafts lost at 1‐year posttransplant was attributed to psychosocial issues. Conclusions Short‐term outcomes among children listed using the pFAT form are good. Among those nonlisted, the pFAT highlights specific psychosocial/socioeconomic barriers, over which most children themselves have no power to change, which should be systemically addressed to permit eligibility of more children and save lives.
Trauma is one of the leading causes of mortality and morbidity world wide, affecting both adult and paediatric populations. The objective of this book is to provide physiotherapists at all stages of their careers with current evidencebased information to guide their physiotherapeutic management of both adults and children with traumarelated injuries. Information has been drawn from published research as well as the clinical expertise of the contributors to provide readers with a comprehensive book that details the physiotherapist’s role and management of trauma related injuries. Chapter 1 examines the physiological response to trauma, deepening understanding of the physiological basis of the problems trauma patients often face. The following chapter highlights the important anatomical differences between children and adults and the clinical implications thereof. Of particular relevance to the South African setting is the chapter on immunosuppressive diseases and trauma. This comprehensive chapter covers physiotherapy modalities, exercise prescription and both subjective and objective outcome measures that may be used in the management of trauma patients. Commonly encountered traumatic injuries such as burn, head, spinal cord, multiple orthopaedic and blunt penetrating injuries are presented in the following chapters. The final chapter examines the quality of life of trauma (and critical illness) survivors and its management. The clinical chapters provide a concise but thorough review of the mechanism of injury, and medical and surgical management. The contributors present the specific objectives of physiotherapy, general precautions and contraindications, recommended treatment and outcome measures and, importantly, specific paediatric considerations. Each chapter also contains a clinical case study for each of adults and children, providing the reader with an example of the clinical application of the theory presented. The paediatric considerations detailed in each chapter are useful, especially to those working in this field. Key take home points are highlighted throughout in ‘Key Message’ boxes and the use of summary tables for detailed information helps the reader with quick referencing. While the focus of this book is primarily on the acute management of traumarelated injuries, where information is beyond the scope of the book, the reader is directed to additional resources. The information provided in this book is practical and presented in such a manner that physiotherapists, whether qualified for one or many years or undergraduate students, will come away with comprehensive, evidencedbased understanding of the physiotherapist’s role and management in adults and children with trauma. It can also be used as a reference source for physiotherapists who have to treat patients with injuries that they do not normally encounter, and who require a quick refresh on how to approach and implement treatment. This wellwritten book provides with reader with comprehensive, practical information for a holistic, evidencebased approach to the management of both children and adults with traumatic injuries. It is a worthwhile investment for anyone working in the trauma and hospital setting and with an interest in cardiopulmonary physiotherapy.
Aim and objectives: Temporal trends in CF survival from low-middle-income (LMIC) settings are poorly reported. We describe changes in CF survival over 40 years from a South African (SA) CF center. Methods: A cohort study of people with CF from 1974 to 2019. Changes in age-specific mortality rates (MR) before/after year 2000 were estimated using multivariable Poisson regression. Adjusted models were stratified for age 10 years and controlled for diagnosis age, sex, ancestry, genotype, and P.aeruginosa(PA) infection. Adjusted sub-analysis explored association between MRratio (aMRr) and FEV1z-scores at age 5-8 years. Results: 288 people (52% male;57% Caucasian;44% p.Phe508del homozygous) were included (median diagnosis age 0.5 years, IQR 0.2-2.5); 58(35%) died and 30(10%) lost to follow-up. Age-specific MR reduced by 64% (figure 1) after year 2000, with significant reduction age>10 (aMRr 0.25; 95% CI 0.14,0.46;p<0.01) compared to <10 years (aMRr 0.67; 95% CI 0.28,1.64).In children<10 years, Caucasian ancestry was associated with lower mortality (aMRr 0.17; 95% CI 0.04,0.63), and time since first PA infection with higher mortality (aMRr 1.30; 95% CI 1.01,1.68). Mortality was 6-fold higher if FEV1z was<-2.0 at age 5-8 years (aMRr 6.56; 95% CI 2.40,17.9). Conclusion: Overall, CF survival has significantly improved in SA after year 2000. However, increased risk of mortality persists in non-Caucasians and children with FEV1z<-2.0 at age 5-8 years.
The Society of Critical Care Medicine (SCCM) Reviewer Academy seeks to train and establish a community of trusted, reliable, and skilled peer reviewers with diverse backgrounds and interests to promote high-quality reviews for each of the SCCM journals. Goals of the Academy include building accessible resources to highlight qualities of excellent manuscript reviews; educating and mentoring a diverse group of healthcare professionals; and establishing and upholding standards for insightful and informative reviews. This manuscript will map the mission of the Reviewer Academy with a succinct summary of the importance of peer review, process of reviewing a manuscript, and the expected ethical standards of reviewers. We will equip readers to target concise, thoughtful feedback as peer reviewers, advance their understanding of the editorial process and inspire readers to integrate medical journalism into diverse professional careers.
'%3e%3cpath fill='%23001489' d='M0 0v6h9V0z'/%3e%3cpath fill='%23e03c31' d='M0 0v3h9V0z'/%3e%3cg stroke='%23fff' stroke-width='2'%3e%3cpath id='d' d='m0 0 4.5 3L0 6m4.5-3H9'/%3e%3cuse xlink:href='%23b' stroke='%23ffb81c' clip-path='url(%23c)'/%3e%3c/g%3e%3cuse xlink:href='%23d' fill='none' stroke='%23007749' stroke-width='1.2'/%3e%3c/g%3e%3c/svg%3e)
