Gene and cell therapy in South Africa: current status and future prospects

South Africa has a high disease burden resulting from communicable and non-communicable diseases. Current therapeutic interventions rarely result in a cure and the associated lifelong treatment places a considerable strain on an overburdened health sector. Gene and cell therapies present novel alternatives to disease management, offering the promise of a single treatment and a lifelong cure. Although challenges remain, investment in the field has started to bear fruit, with a number of gene and cell therapeutics reaching the market in the past decade. To take full advantage of these developments, it is important that a proactive approach to nurturing appropriate human and material resources is adopted in the country

Home therapy for inherited bleeding disorders in south Africa: results of a modified delphi consensus process

Background. Optimal care of patients with inherited bleeding disorders requires that bleeding episodes are treated early, or still better prevented, through extension of patient care beyond hospital-based treatment to home-based therapy. In South Africa (SA), adoption of home therapy is variable, in part owing to lack of consensus among healthcare providers on what constitutes home therapy, which patients should be candidates for it, how it should be monitored, and what the barriers to home therapy are.Objectives. To conduct a modified Delphi process in order to establish consensus on home therapy among haemophilia healthcare providers in SA.Methods. Treaters experienced in haemophilia care were invited to participate in a consensus-seeking process conducted in three rounds. In round 1, provisional statements around home therapy were formulated as questions and collated in a structured list. In rounds 2 and 3, evolving versions of the questionnaire were administered to participants. Consensus was defined as ≥70% agreement among the participants.Results. The panel composition included an equal number of physicians and non-physicians. The participation rate was 100% through all three consensus rounds. The group reached consensus for 92% of the statements. Consensus of 100% was reached on starting home therapy in paediatric patients, requiring all patients on home therapy to sign informed consent and indemnity, and providing round-the-clock support for patients on home therapy.Conclusions. The home therapy consensus statements in this report have the potential to translate to policy on home therapy and to guide the initiation, practice and evaluation of home therapy programmes in SA

Deep-vein thrombosis in the era of high HIV and tuberculosis prevalence: A prospective review of its diagnosis and treatment in a quaternary centre

Background. Venous thromboembolic disease (VTE) is a leading cause of morbidity and mortality worldwide. HIV and tuberculosis (TB) infections have an aetiological association with VTE. Implementation of national HIV and TB programmes in South Africa (SA) has changed the burden of these two conditions, with resultant effects on VTE prevalence. Furthermore, with the increased use of direct oral anticoagulants (DOACs), baseline thrombosis data are needed to evaluate the effect of these new agents.Objectives. To determine real-life baseline VTE characteristics in a pre-DOAC era, and to document the association of HIV and TB infections with VTE.Methods. This was a single-centre prospective cohort study performed in a quaternary care centre at Charlotte Maxeke Johannesburg Academic Hospital, SA. Key inclusion criteria included signed informed consent by adults (≥18 years) with a new episode of thrombosis. Procedures included physical examination, thrombosis risk factor assessment, duplex Doppler examination, thrombophilia screening, inpatient treatment and outpatient follow-up.Results. Ninety-nine participants with confirmed thrombosis met the inclusion criteria. Participants were predominantly black (79.8%) and female (64.6%), with a median age of 46 (interquartile range (IQR) 38 - 57) years. The prevalences of HIV and TB were 53.0% and 21.2%, respectively. The most common risk factors for thrombosis were TB (17.2%) and malignancies (14.1%). Thrombophilia screening had a low diagnostic yield. The median time to target international normalised ratio during hospitalisation was 5.5 (IQR 4.0 - 7.0) days and the median duration of hospitalisation was 9 (IQR 7 - 11) days. The overall mortality rate in the cohort at 3 months post hospitalisation was 12.1%.Conclusions. This prospective study provides real-life data on thrombosis diagnosis and management at a quaternary public healthcare facility, providing a valuable baseline against which the effect of new DOAC anticoagulants can be assessed. Further research is required to clarify the aetiological association between thrombosis and HIV and TB