ObjectiveTo investigate the pharmacokinetics, effectiveness, and safety of subcutaneous (SC) abatacept treatment over 24 months in patients with polyarticular‐course juvenile idiopathic arthritis (JIA).MethodsIn this phase III, open‐label, international, multicenter, single‐arm study, patients with polyarticular JIA (cohort 1, ages 6–17 years and cohort 2, ages 2–5 years) in whom treatment with ≥1 disease‐modifying antirheumatic drug was unsuccessful received weight‐tiered SC abatacept weekly: 10 to <25 kg (50 mg), 25 to <50 kg (87.5 mg), ≥50 kg (125 mg). Patients who had met the JIA–American College of Rheumatology 30% improvement criteria (achieved a JIA‐ACR 30 response) at month 4 were given the option to continue SC abatacept to month 24. The primary end point was the abatacept steady‐state serum trough concentration (Cminss) in cohort 1 at month 4. Other outcome measures included JIA‐ACR 30, 50, 70, 90, 100, and inactive disease status, the median Juvenile Arthritis Disease Activity Score in 71 joints using the C‐reactive protein level (JADAS‐71–CRP) over time, safety, and immunogenicity.ResultsThe median abatacept Cminss at month 4 (primary end point) and at month 24 was above the target therapeutic exposure (10 μg/ml) in both cohorts. The percentage of patients who had achieved JIA‐ACR 30, 50, 70, 90, or 100 responses or had inactive disease responses at month 4 (intent‐to‐treat population) was 83.2%, 72.8%, 52.6%, 28.3%, 14.5%, and 30.1%, respectively, in cohort 1 (n = 173) and 89.1%, 84.8%, 73.9%, 58.7%, 41.3%, and 50.0%, respectively, in cohort 2 (n = 46); the responses were maintained to month 24. The median (interquartile range) JADAS‐71–CRP improved from baseline to month 4: cohort 1, from 21.0 (13.5, 30.3) to 4.6 (2.1, 9.4); cohort 2, from 18.1 (14.0, 23.1) to 2.1 (0.3, 4.4). Improvements were sustained to month 24, at which time 27 of 173 patients (cohort 1) and 11 of 22 patients (cohort 2) had achieved JADAS‐71–CRP remission. No unexpected adverse events were reported; 4 of 172 patients (2.3%) in cohort 1 and 4 of 46 (8.7%) in cohort 2 developed anti‐abatacept antibodies, with no clinical effects.ConclusionWeight‐stratified SC abatacept yielded target therapeutic exposures across age and weight groups, was well tolerated, and improved polyarticular JIA symptoms over 24 months.

Background Birth polymerase chain reaction (PCR) testing improves early detection of HIV and allows for early treatment initiation. National guidelines exist, but it is unknown whether these are being implemented correctly. Objectives To determine whether HIV-exposed infants at the Mangaung University Community Partnership Programme Community Health Centre (MUCPP CHC) received PCR tests at birth, if HIV-positive infants were initiated on treatment, if follow-up dates were scheduled and the percentage of mothers or caregivers who returned to collect the results. Methods The study was a retrospective descriptive file audit (1304 files) of births from 01 January to 31 December 2016 at MUCPP CHC. The study sample was 428 infants born to HIV-positive mothers. The birth register was used to collect the infants’ HIV PCR test barcodes. The birth and 10-week PCR results were retrieved from an electronic database at the Virology Department, University of the Free State. Results In total, 375 infants received a birth PCR test (87.6%) of which 4 (1.1%) tested HIV positive and 327 (87.2%) negative. Follow-up tests were not scheduled. However, 145 (44.3%) HIV-negative infants returned for a 10-week test. Irrespective of the PCR birth result, 157 (36.7%) infants were brought for a 10-week follow-up test at which time 3 (1.9%) tested positive and 151 (96.2%) negative. Conclusion The majority of HIV-exposed infants received a PCR test at birth; however, the clinic is below the national target (90%) for HIV testing. A record-keeping system of infants’ visits does not exist at MUCPP CHC, making it impossible to determine whether HIV-positive infants were started on antiretroviral treatment.

While the progress towards reaching the UNAIDS 95-95-95 targets in South African adults seems promising, the progress in the paediatric population is lagging far behind; only 79% percent of children living with HIV know their status. Of these, only 47% are on treatment, and a mere 34% of those are virally suppressed. Thus, virological suppression has been attained in only 13% of children living with HIV in South Africa. Multiple factors contribute to the high treatment failure rate, one of them being a lack of paediatric-friendly antiretroviral treatment (ART) formulations. For example, the Lopinavir/ritonavir syrup, which is the current mainstay of ART for young children, has an extremely unpleasant taste, contributing to the poor tolerability and lack of adherence by children using the formulation. Furthermore, the lack of appropriate formulations limits the optimisation of regimens, especially for young children and those who cannot swallow tablets. Switching from syrups to dispersible tablets will improve ease of administration and adherence and result in cost-saving. Despite the approval of simplified paediatric-friendly formulations internationally, including other sub-Saharan African countries, unnecessary delays are experienced in South Africa. Clinician groups and community organisations must speak up and demand that approvals be expedited to ensure the delivery of life-changing and life-saving formulations to our patients as a matter of urgency.

Background: Therapeutic patient education (TPE) is now recommended in the health care pathway of patients with chronic inflammatory rheumatism (CIR) but few pediatric programs are currently available. MIRAJE is a program for children and adolescents with CIR. It has been developed within a French health network (RESRIP (Réseau Rhumatismes Inflammatoires Pédiatriques)) to improve patient care for paediatric patients with CIR living in the Ile de France region. The originality of the program was to design cross-cutting TPE using common issues to different pathologies: the experience of chronic illness, the management of daily life, inflammatory attacks, long-term treatments, physical activity. Objectives: Assessment of acquisition of coping, self-care and safety skills. Methods: After signing a written consent, an educational assessment is conducted in an individual interview with the patients and/or parents (for children under 6 years old), by a member of the multidisciplinary team.Five group sessions and one self-injection session are offered to patients. During the collective sessions, patients and parents are reappointed according to 4 groups: the group of parents, that of adolescents (12-18 years), that of children (6-11 years) and that of toddlers (<6 years). To investigate the cognitive and behavioral impact of TPE on our patients and/or parents, we selected patients aged over 6 years who completed TPE program. A pre-post comparison was performed using a skill/knowledge questionnaire, based on three educational objectives: adaptation (8 skills), self-care (5 skills) and safety (1 skill), before and after the intervention. Results: We present the results of the first year of implementation of our TPE program (2017). Nineteen children and 20 parents attended at least one group session. Ten participants (6 children aged 6 to 17 and 4 parents) completed the TPE program. The diseases covered were juvenile idiopathic arthritis (7), systemic lupus (2) and mevalonate kinase deficiency (1). All participants improved their knowledge and skills following TPE with greatest improvement in management of inflammatory attacks (self-care) (10 patients after TPE versus 1 at initial assessment), chronic fatigue (6 versus 1) and in development of physical activity (8 versus 2) (adaptation). Otherwise, all 10 participants acquired self-care skills (4.1 versus 1.4 average skills per patient at initial assessment), 9 acquired coping skills (6.8 versus 2.6 skills) and 8 security skills. Regarding selfinjection workshop, most patients (4/5) have acquired skills in subcutaneous self-injection. Conclusion: MIRAJE is the first French cross-cutting TPE program for children suffering from CIR and their parents. Although the number of participants having benefited from a full TPE program is relatively low, the first results are very encouraging. Indeed, TPE has allowed improving knowledge and skills for most children and parents. TPE allows a better acceptance of the disease and fosters patient's empowerment in daily management. We plan t...

ObjectiveDescribe the efficacy and safety data of children with polyarticular-course juvenile idiopathic arthritis (pcJIA) treated with abatacept + methotrexate (MTX) or abatacept monotherapy when prior MTX use was either ineffective or not tolerated.MethodsPost hoc analysis of 2 phase III trials of subcutaneous (SC) and intravenous (IV) abatacept over 2 years in patients with pcJIA (aged 2–17 years). Patients were stratified by treatment for abatacept + MTX or abatacept monotherapy and further by prior biologic use. Efficacy outcomes included JIA-American College of Rheumatology (JIA-ACR) responses, Juvenile Arthritis Disease Activity Score in 27 joints (JADAS27) using C-reactive protein, and safety. Descriptive pharmacokinetic analyses were also performed.ResultsEfficacy responses (JIA-ACR and JADAS27) were similar between patients receiving abatacept + MTX (n = 310) or abatacept monotherapy (n = 99) and persisted over 2 years. Clinical response rates were similar in biologic-naive patients and prior biologic users; this was independent of MTX use. Across both studies, abatacept + MTX and abatacept monotherapy displayed similar safety profiles. Pharmacokinetic results revealed similar minimum steady-state trough abatacept concentrations between studies. Further, baseline MTX did not influence abatacept clearance and was not a significant predictor of JIA-ACR responses.ConclusionAbatacept monotherapy (SC and IV) was effective and well tolerated in children with pcJIA when prior MTX use was ineffective or not tolerated. Treatment effects of abatacept appear to be independent of MTX co-administration. Consequently, abatacept monotherapy can be considered for those with prior biologic therapy if MTX use is inappropriate.