FDA Approval Summary: Ruxolitinib for Treatment of Chronic Graft-Versus-Host Disease after Failure of One or Two Lines of Systemic Therapy

On September 22, 2021, the Food and Drug Administration approved ruxolitinib for the treatment of chronic graft-versus-host disease (cGVHD) after the failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older. Approval was based on Study INCB 18424-365 (REACH-3; CINC424D2301; {"type":"clinical-trial","attrs":{"text":"NCT03112603","term_id":"NCT03112603"}}NCT03112603), a randomized, open-label, multicenter trial of ruxolitinib in comparison to best available therapy (BAT) for the treatment of corticosteroid-refractory cGVHD occurring after the allogeneic hematopoietic stem cell transplantation. A total of 329 patients were randomized 1:1 to receive either ruxolitinib 10 mg twice daily (n = 165) or BAT (n = 164). BAT was selected by the investigator prior to randomization. The overall response rate through Cycle 7 Day 1 was 70% (95% CI, 63-77) in the ruxolitinib arm, and 57% (95% CI, 49-65) in the BAT arm. The median duration of response, calculated from first response to progression, death, or initiation of new systemic therapies for cGVHD, was 4.2 months (95% CI, 3.2-6.7) for the ruxolitinib arm and 2.1 months (95% CI, 1.6-3.2) for the BAT arm; and the median time from first response to death or initiation of new systemic therapies for cGVHD was 25 months (95% CI, 16.8-not estimable) for the ruxolitinib arm and 5.6 months (95% CI, 4.1-7.8) for the BAT arm. Common adverse reactions included anemia, thrombocytopenia, and infections. Given the observed response rate with durability, the clinical benefit of ruxolitinib appears to outweigh the risks of treatment for cGVHD after the failure of one or two lines of systemic therapy.     

Bolus Calculators

Matching meal insulin to carbohydrate intake, blood glucose, and activity level is recommended in type 1 diabetes management. Calculating an appropriate insulin bolus size several times per day is, however, challenging and resource demanding. Accordingly, there is a need for bolus calculators to support patients in insulin treatment decisions. Currently, bolus calculators are available integrated in insulin pumps, as stand-alone devices and in the form of software applications that can be downloaded to, for example, smartphones. Functionality and complexity of bolus calculators vary greatly, and the few handfuls of published bolus calculator studies are heterogeneous with regard to study design, intervention, duration, and outcome measures. Furthermore, many factors unrelated to the specific device affect outcomes from bolus calculator use and therefore bolus calculator study comparisons should be conducted cautiously. Despite these reservations, there seems to be increasing evidence that bolus calculators may improve glycemic control and treatment satisfaction in patients who use the devices actively and as intended.     

Psoriasis: to treat or to manage?

The recognition of psoriasis as a systemic disorder with characteristic skin symptoms and associated diseases has changed treatment concepts substantially. The complexity of psoriasis disease not only requires appropriate therapy but also weight‐loss and smoking cessation programmes as well as trigger factor elimination. The term ‘management’ may better reflect the aim for a holistic approach of disease control. Comorbidity and the presence of psoriatic arthritis are important denominators for drug selection. However, there is a lack of prospective data substantiating a benefit of associated diseases by antipsoriatic therapy. Securing success using treatment goals helps to establish an efficacious therapy and to control inflammation. A regular scoring of disease severity, patients’ quality of life and assessment of other clinically relevant conditions are mandatory to closely follow the disease course. There is debate whether an early treatment may modulate the future course of psoriasis. Concepts of minimal disease activity have not been implemented in psoriasis yet. There is a lack of evidence how long any treatment should be given and when and how to terminate. Finally, outcome tools should specifically be tailored for psoriasis to evaluate disease‐related items as well as the benefit of management from the patient's perspective.     

Managing the first period at home with a newborn: a grounded theory study of mothers’ experiences

Aim: The article aims to present a theoretical account of mothers’ first period at home with their newborn in Norway. Background: Mothers’ well‐being affects their babies and is therefore an important priority for public health. Early discharge after childbirth is common in many countries. In Norway, this has been practised for 5–10 years but without any distinct agreement between maternity hospitals and the community health services and without documented follow‐up care. Knowledge is lacking in how mothers deal with the first period at home with the baby. Method: Seven focus group discussions were conducted with 26 mothers who had babies 1.5–3 months old. The grounded theory method was used to gather and analyse data. Results: The mothers were strongly concerned about preserving their control and integrity in the new situation. This main concern was resolved by the strategy of prioritizing newborn care. The strategy encompassed a process of developing competence as a mother, changing focus in relationships, stretching to the critical level and seeking recognition. These parallel processes were inter‐related, sometimes mutually supportive and sometimes conflicting. When conflicts occurred, prioritizing newborn care guided mothers in finding solutions. Breaches of the implied conditions in health care tended to increase mothers’ level of strain and uncertainty and to influence their efficacy in breastfeeding the baby. Conclusion: Being a mother to a newborn is a dynamic and extensive process. Succeeding in breastfeeding seems especially sensitive and essential in motherhood. The idea that giving birth is a simple and normal situation may obscure the importance of seamless health care and the need for professional support and information.