Abstract
The Joint United Nations Program on human immunodeficiency virus/acquired immunodeficiency syndrome (HIV/AIDS) (Joint United Nations Program on HIV/AIDS, UNAIDS) has recommended 90–90–90 ...goals to increase the number of patients who are aware of their status, on antiretroviral therapy, and have undetectable viral loads. Mexico City has made several achievements to aid in prevention, early diagnosis, and treatment; however, the incidence of HIV has not decreased over the past decade. This article reviews global initiatives that were successful in achieving some or all these metrics and provide a road map for Mexico to reach the desired goals.
•Treatment of SMA within the first days of life may result in essentially normal outcome.•Early diagnosis of SMA is mandatory for the outcome of treatment.
New molecular therapies are available for ...the treatment of spinal muscular atrophy (SMA) but early intervention is required. We report two cases that were diagnosed prenatally, where treatment with nusinersen was initiated within 7 h and three days respectively. The children were followed up for 13 months and almost six years respectively. Both children have developed within entirely normal centiles, indicating that initiating treatment immediately after birth, as in these cases, is essential for a good outcome.
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GEOZS, IJS, IMTLJ, KILJ, KISLJ, NLZOH, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UILJ, UL, UM, UPCLJ, UPUK, ZAGLJ, ZRSKP
Abstract Background In the EDITA trial, patients with systemic sclerosis (SSc) and mild pulmonary vascular disease (PVD) treated with ambrisentan had a significant decline of pulmonary vascular ...resistance (PVR) but not of mean pulmonary arterial pressure (mPAP) vs. placebo after six months. The EDITA-ON study aimed to assess long-term effects of open label therapy with ambrisentan vs. no pulmonary arterial hypertension (PAH) therapy. Methods Patients who participated in the EDITA study and received regular follow-up were included in EDITA-ON. Clinical, echocardiographic, laboratory, exercise and hemodynamic parameters during follow-up were analysed. The primary endpoint was to assess whether continued treatment with ambrisentan vs. no treatment prevented the development of PAH according to the new definition. Results Of 38 SSc patients included in the EDITA study four were lost to follow-up. Of the 34 remaining patients (age 55 ± 11 years, 82.1% female subjects), 19 received ambrisentan after termination of the blinded phase, 15 received no PAH medication. The mean follow-up time was 2.59 ± 1.47 years, during which 29 patients underwent right heart catheterization. There was a significant improvement of mPAP in catheterised patients receiving ambrisentan vs. no PAH treatment (-1.53 ± 2.53 vs. 1.91 ± 2.98 mmHg, p = 0.003). In patients without PAH treatment 6/12 patients had PAH vs. 1/17 of patients receiving ambrisentan ( p < 0.0001). Conclusion In SSc patients with early PVD, the development of PAH and/or deterioration was less frequent among patients receiving ambrisentan, indicating that early treatment and close follow-up could be beneficial in this high-risk group. Future trials in this field are needed to confirm these results.
Abstract
Background
The impact of very early infant treatment on human immunodeficiency virus (HIV) reservoir, and markers for treatment success, require study.
Methods
The Early Infant Treatment ...Study (EIT) enrolled 40 children living with HIV started on antiretroviral treatment (ART) at <7 days of age, with 23 who had started treatment between 30–365 days to serve as controls. Quantitative HIV DNA was evaluated every 1–3 months in peripheral blood mononuclear cells. 84-week repeat qualitative whole blood DNA polymerase chain reaction and dual enzyme immunosorbent assay were performed.
Results
Median quantitative cell-associated DNA after at least 84 weeks was significantly lower among the first 27 EIT children tested than among 10 controls (40.8 vs 981.4 copies/million cells; P < .001) and correlated with pre-ART DNA. Median DNA after 84 weeks did not differ significantly by negative or positive serostatus at 84 weeks (P = .94), and appeared unaffected by periods of unsuppressed plasma RNA from 24–84 weeks (P = .70). However, negative 84-week serostatus was 67% predictive for sustained RNA suppression, and positive serostatus was 100% predictive for viremia. Loss of qualitative DNA positivity at 84 weeks was 73% predictive for sustained suppression, and persistent positivity was 77% predictive for viremia.
Conclusions
Lower viral reservoir was associated with starting ART at <1 week. Negative serostatus and qualitative DNA were useful markers of sustained viral suppression from 24–84 weeks.
Infants starting antiretroviral therapy at <1 week had lower 84-week proviral reservoir than controls. Serostatus and qualitative DNA were useful markers for rebound, but transient viremia between 24–84 weeks had no apparent impact on the viral reservoir in peripheral blood mononuclear cells.
This study aimed to establish an optional newborn screening program for spinal muscular atrophy (SMA-NBS) in Osaka.
A multiplex TaqMan real-time quantitative polymerase chain reaction assay was used ...to screen for SMA. Dried blood spot samples obtained for the optional NBS program for severe combined immunodeficiency, which covers about 50% of the newborns in Osaka, were used. To obtain informed consent, participating obstetricians provided information about the optional NBS program to all parents by giving leaflets to prospective parents and uploading the information onto the internet. We prepared a workflow so that babies that were diagnosed with SMA through the NBS could be treated immediately.
From 1 February 2021 to 30 September 2021, 22,951 newborns were screened for SMA. All of them tested negative for survival motor neuron (SMN)1 deletion, and there were no false-positives. Based on these results, an SMA-NBS program was established in Osaka and included in the optional NBS programs run in Osaka from 1 October 2021. A positive baby was found by screening, diagnosed with SMA (the baby possessed 3 copies of the SMN2 gene and was pre-symptomatic), and treated immediately.
The workflow of the Osaka SMA-NBS program was confirmed to be useful for babies with SMA.
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GEOZS, IJS, IMTLJ, KILJ, KISLJ, NLZOH, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UILJ, UL, UM, UPCLJ, UPUK, ZAGLJ, ZRSKP
Abstract
There have been major advances in the management of axial spondyloarthritis (axSpA) with the introduction of effective biologic agents targeting TNF and IL-17A. Clinicians now have more ...choice but, despite treatment recommendations, are still faced with significant uncertainty when deciding on the optimal treatment strategy for an individual patient in clinical practice. Management of axSpA typically requires both non-pharmacological and pharmacological interventions. NSAIDs remain the first line drug therapies for axSpA with proven efficacy for symptomatic management but uncertainty remains regarding their optimal long-term use relating to radiographic progression and safety in axSpA. To-date there are no head-to-head trials of biologics in axSpA. Clinicians need to consider other factors, including extra-articular manifestations, comorbidities, safety and radiographic progression when deciding on which biologic to recommend for an individual patient. This article will explore the evidence relating to these factors and highlight areas of unmet need.
ABSTRACT
Objective
Determine whether early weight gain predicts full remission at end‐of‐treatment (EOT) and follow‐up in two different treatments for adolescent anorexia nervosa (AN), and to track ...the rate of weight gain throughout treatment and follow‐up.
Method
Participants were 121 adolescents with AN (mean age = 14.4 years, SD = 1.6), from a two‐site (Chicago and Stanford) randomized controlled trial. Adolescents were randomly assigned to family‐based treatment (FBT) (n = 61) or individual adolescent focused therapy (AFT) (n = 60). Treatment response was assessed using percent of expected body weight (EBW) and the global score on the Eating Disorder Examination (EDE). Full remission was defined as having achieved ≥95% EBW and within one standard deviation of the community norms of the EDE. Full remission was assessed at EOT as well as 12‐month follow‐up.
Results
Receiver operating characteristic analyses showed that the earliest predictor of remission at EOT was a gain of 5.8 pounds (2.65 kg) by session 3 in FBT (area under the curve (AUC) = 0.670; p = .043), and a gain of 7.1 pounds (3.20 kg) by session 4 in AFT (AUC = 0.754, p = .014). Early weight gain did not predict remission at follow‐up for either treatment. A survival analysis showed that weight was marginally superior in FBT as opposed to AFT (Wald chi‐square = 3.692, df = 1, p = .055).
Discussion
Adolescents with AN who receive either FBT or AFT, and show early weight gain, are likely to remit at EOT. However, FBT is superior to AFT in terms of weight gain throughout treatment and follow‐up. (Int J Eat Disord 2014; 47:124–129)
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BFBNIB, DOBA, FZAB, GIS, IJS, IZUM, KILJ, NLZOH, NUK, OILJ, PILJ, PNG, SAZU, SBCE, SBMB, UILJ, UKNU, UL, UM, UPUK
Summary
The only way to prevent immune thrombocytopenia (ITP) from becoming refractory would be to restore tolerance to platelets at an early phase of the disease. Numerous immune alterations ...probably accumulate in chronic ITP; thus, the chances of cure decrease significantly with time. Currently, sustained remission off treatment (SROT) is a clinical definition describing patients who can discontinue their ITP treatment without risk and maintain a state of remission. Different treatment strategies are presently being evaluated with the goal of attaining SROT, mostly combining drugs targeting the innate and/or the adaptive immune system, the inflammation state, so as increasing the platelet load. In this sense, thrombopoietin receptor agonists (TPO‐RAs) have shown promising results if used as upfront treatment. TPO‐RAs seem to exhibit immunomodulation and immune tolerance properties, increasing not only the platelet antigen mass but also increasing the transforming growth factor‐β concentration, and stimulating regulatory T and B lymphocytes. However, more immunological studies are needed to establish accurate molecular alterations in ITP that are potentially reversed with treatments.
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BFBNIB, FZAB, GIS, IJS, KILJ, NLZOH, NUK, OILJ, SAZU, SBCE, SBMB, UL, UM, UPUK
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