Prophylaxis is the new standard of care in patients with haemophilia.

Randomised controlled clinical trial evidence on prophylaxis as optimal care for patients with haemophilia was generated more than a decade ago. However, this knowledge has not translated into clinical practice in South Africa (SA) owing to many barriers to prophylaxis. These include the high treatment burden imposed by prophylaxis (frequent injections two to four times a week), the need for intravenous access to administer replacement clotting factor therapies, and the higher volume of clotting factor required compared with episodic treatment. The recently introduced non-factor therapies in haemophilia care have addressed many of these barriers. For example, emicizumab, which is currently the only globally approved non-factor therapy, can be administered subcutaneously less frequently (weekly, fortnightly or every 4 weeks) and has led to global adoption of prophylaxis as the standard of care in haemophilia by the bleeding disorders community. Haemophilia A is the most prevalent clotting factor deficiency in SA, with >2 000 people diagnosed to date. However, only a few of these patients are currently on prophylaxis. In this 'In Practice' article, we review the rationale for prophylaxis, outline its goals and benefits, and provide evidence-based guidance on which haemophilia patients should be prioritised for emicizumab prophylaxis. This consensus guidance facilitates the adoption of prophylaxis as a national policy and the new standard of care in haemophilia in SA.

PEACE V - Salvage Treatment of OligoRecurrent nodal prostate cancer Metastases (STORM): a study protocol for a randomized controlled phase II trial.

BACKGROUND: Pelvic nodal recurrences are being increasingly diagnosed with the introduction of new molecular imaging techniques, like choline and PSMA PET-CT, in the restaging of recurrent prostate cancer (PCa). At this moment, there are no specific treatment recommendations for patients with limited nodal recurrences and different locoregional treatment approaches are currently being used, mostly by means of metastasis-directed therapies (MDT): salvage lymph node dissection (sLND) or stereotactic body radiotherapy (SBRT). Since the majority of patients treated with MDT relapse within 2 years in adjacent lymph node regions, with an estimated median time to progression of 12-18 months, combining MDT with whole pelvic radiotherapy (WPRT) may improve oncological outcomes in these patients. The aim of this prospective multicentre randomized controlled phase II trial is to assess the impact of the addition of WPRT to MDT and short-term androgen deprivation therapy (ADT) on metastasis-free survival (MFS) in the setting of oligorecurrent pelvic nodal recurrence. METHODS & DESIGN: Patients diagnosed with PET-detected pelvic nodal oligorecurrence (≤5 nodes) following radical local treatment for PCa, will be randomized in a 1:1 ratio between arm A: MDT and 6 months of ADT, or arm B: WPRT added to MDT and 6 months of ADT. Patients will be stratified by type of PET-tracer (choline, FACBC or PSMA) and by type of MDT (sLND or SBRT). The primary endpoint is MFS and the secondary endpoints include clinical and biochemical progression-free survival (PFS), prostate cancer specific survival, quality of life (QoL), toxicity and time to castration-resistant prostate cancer (CRPC) and to palliative ADT. Estimated study completion: December 31, 2023. DISCUSSION: This is the first prospective multicentre randomized phase II trial assessing the potential of combined WPRT and MDT as compared to MDT alone on MFS for patients with nodal oligorecurrent PCa. TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT03569241, registered June 14, 2018, ; Identifier on Swiss National Clinical Trials Portal (SNCTP): SNCTP000002947, registered June 14, 2018.

Tumours of the Chest in Children: A Review

Tumours of the chest in children constitute an array of pathology and clinical symptomatology. These tumours can be benign or malignant; cystic or solid; primary or as a result of secondary metastases. Collectively; tumours of the chest in children are very rare; the exact incidence of which is largely unknown globally. Non neoplastic lesions of the lung including bronchogenic cysts; sequestrations; congenital pulmonary airway malformations as well as infective and inflammatory disorders are 60 times more common than neoplastic causes.1 A tumour of the chest is considerably difficult to diagnose since patients can be asymptomatic for many years before symptoms evolve. Even more so; the symptoms are non-specific and can suggest more common and less sinister pathology. Clinically patients present with a variety of symptoms that depend largely on the location of the tumour.Airway tumours can be symptomatic or can present with chronic cough; wheeze; haemoptysis; atelectasis or persistent pneumonia. Secondary malignant parenchymal tumours are likely to be symptomatic from the primary lesion. Anterior mediastinal tumours can cause compression of the large airways or superior vena caval structures.It stands to reason that the physician needs to have a very high index of suspicion when dealing with these non-specific signs and symptoms. This article provides an approach to tumours of chest and reviews the common aetiology in the different compartments of the chest. The article will focus on common tumours of the airway; lung parenchyma; mediastinum; cardiac and chest wall pathology

Retinoblastoma outcome at a single institution in South Africa.

INTRODUCTION: Retinoblastoma (RB) is the most common eye cancer in children. Early detection is necessary for cure. OBJECTIVE: To compare stage and outcome of children with RB treated at Kalafong Hospital, Pretoria, South Africa (SA), during two time periods (1993 - 2000 and 2001 - 2008, after outreach interventions in 2000 and introduction of compulsory community service for doctors in 1998). METHODS: Data collected included demography (age, gender, date of birth), stage and treatment received. The main outcome measure was disease-free survival and the study end-point was 60 months after diagnosis. RESULTS: There were 51 patients during the time period 1993 - 2000 (group 1) and 73 during 2001 - 2008 (group 2), with median ages of 32 and 26 months, respectively (marginally significantly younger in group 2; p = 0.046). In group 1, the majority (57%) presented with advanced disease (stages III and IV), with a decline in this proportion in group 2 (40%) indicating a downward but not significant trend (p = 0.075). Bilateral disease was diagnosed in 22% of patients in group 1 and 33% in group 2. Overall survival was 33% and 43% for groups 1 and 2, respectively. Excluding absconding patients, event-free survival was 50% in group 1, improving to 68% in group 2 (not statistically significant; p = 0.18). Fewer patients needed radiotherapy during the second period (statistically significant; p = 0.04), probably because of less advanced disease. CONCLUSION: Poor outcome is probably a result of late diagnosis. It is important to implement a strategy that will ensure early diagnosis and optimal management of RB in SA.

The abdominal cocoon. A case report.

The abdominal cocoon, or idiopathic sclerosing encapsulating peritonitis, is a rare cause of intestinal obstruction. This rare condition, in which the small intestine is encased in a thick fibrous membrane, has been reported predominantly in females. Despite some reports of a preoperative diagnosis, in the majority of cases diagnosis is only made at laparotomy for acute or subacute intestinal obstruction. Treatment consists of resection of the membrane with lysis of adhesions. We present a case of abdominal cocoon in a man where early preoperative diagnosis by radiological evaluation was overlooked. A better awareness of this condition may result in early diagnosis and proper management, thus avoiding unnecessary bowel resections and bad outcomes.

Hepatitis B virus (HBV) and hepatitis C virus (HCV) infections in health care workers (HCWs): guidelines for prevention of transmission of HBV and HCV from HCW to patients.

The transmission of viral hepatitis from health care workers (HCW) to patients is of worldwide concern. Since the introduction of serologic testing in the 1970s there have been over 45 reports of hepatitis B virus (HBV) transmission from HCW to patients, which have resulted in more than 400 infected patients. In addition there are six published reports of transmissions of hepatitis C virus (HCV) from HCW to patients resulting in the infection of 14 patients. Additional HCV cases are known of in the US and UK, but unpublished. At present the guidelines for preventing HCW to patient transmission of viral hepatitis vary greatly between countries. It was our aim to reach a Europe-wide consensus on this issue. In order to do this, experts in blood-borne infection, from 16 countries, were questioned on their national protocols. The replies given by participating countries formed the basis of a discussion document. This paper was then discussed at a meeting with each of the participating countries in order to reach a Europe-wide consensus on the identification of infected HCWs, protection of susceptible HCWs, management and treatment options for the infected HCW. The results of that process are discussed and recommendations formed. The guidelines produced aim to reduce the risk of transmission from infected HCWs to patients. The document is designed to complement existing guidelines or form the basis for the development of new guidelines. This guidance is applicable to all HCWs who perform EPP, whether newly appointed or already in post.

[Community health workers are capable of determining reliably the target population for health programs]. / Les agents de santé communautaires sont capables de déterminer avec précision la population-cible des programmes de santé.

The target population is a key concept for the planning and evaluation of health services. However, in developing countries, health professionals in the field are often uneasy with the determination of the population denominator. In Zaire, where reliable demographic data are hardly available, we make use of two different methods aimed to the determination of the size of the target population: (1) an exhaustive population based survey performed by trained interviewers, and (2) a rapid census of the target population performed by community health workers. This paper presents both the results of a demographic survey organized in 1984, and two consecutive census performed by community health workers in 1986 an 1988, in the same area (Northern-Kivu, Zaire). Our results suggest that community health workers under close supervision are able to perform rapidly and at low cost a reliable collection of the demographic data needed for the implementation and monitoring of health programmes at the local level.