In 12 patients, functional reconstitution of B-cells was observed. Encouraging disease outcomes were seen in all patients, along with a favorable safety profile of the treatment, with low levels of cytokine release syndrome (CRS) and only one case of grade 1 immune-effector cell associated neurotoxicity syndrome (ICANS). All patients with SLE achieved a complete remission (CR) after three months and this was maintained in the five patients with a follow-up of 14-24 months. All patients with IIM exhibited major disease improvement after three months, and three patients with SSc experienced decreased disease activity as evidenced by a 4.3 decrease in EULAR AI. As complete cessation of immunosuppressive drugs was possible in all patients and disease outcomes were highly positive, CAR-T therapy presents an exciting avenue for long-term disease control, and possibly cure, in patients with autoantibody dependent ADs.³

Evaluation of the receiver operating characteristics resulted in an AUC of 0.9, and the diagnostic classification accuracy of the test cohort was 92.3%. The diagnosis of prePMF had a final sensitivity of 66.6% and a specificity of 100%. This novel AI model has the potential to become a cost-effective and rapid diagnostic tool in clinical practice, with an ability to accurately differentiate between prePMF and ET in a matter of seconds using affordable hardware. In the future, clinicians could benefit from this model for support in clinical decision-making, as an aid in choosing an appropriate targeted treatment for patients, and in guiding clinical trial enrollment.⁴

Primary outcomes of the trial were OS, relapse-free survival (RFS) and safety, and the secondary outcome was the molecular response in patients during induction, consolidation, and maintenance. 121 patients (5 pediatric, 116 adult) were enrolled into the trial and stratified into standard-risk and high-risk groups on the basis of leukocyte count, with high-risk individuals < 65 years of age receiving daunorubicin, in addition to the AAA administered to all other patients. Consolidation and maintenance with AAA began once patients achieved first complete remission (CR1), which was achieved in all 114 patients treated with AAA/AAA+daunorubicin.

Relapse occurred in only one patient (12 months after maintenance completion), who was found to possess an arsenic trioxide resistance mutation. At the three-year follow-up, efficacy and safety data were very encouraging, with an OS of 99.1%, RFS of 97.9%, and a low rate of adverse events (AEs), including no reported cardiotoxicity. Dexamethasone was used in the 58.7% of patients who experienced APL differentiation syndrome (APL-DS). Although the early deaths which occurred prior to induction must be acknowledged (n=7), this trial found high efficacy and a good safety profile when treating newly diagnosed patients of various ages and disease risk categories with a minimal chemotherapy oral AAA regimen, indicating that this approach presents a viable option for this population.⁵

Posts which were centered around the experience of patients and caregivers were isolated from a randomized sample of 513 posts, and six common themes were identified using AI-powered theme detection in natural language processing (NLP).

Although the content of the posts analyzed was often considered to be neutral, the sentiments of the remaining posts were more often negative than positive and highlighted the prejudice and lack of empathy which patients and caregivers had experienced from HCPs. Many patients were dismissed when sharing details about their pain and the effect of the disease on their QoL, some experienced racial bias, and others found that treatment and emergency care were difficult to access and unsatisfactory. Through the use of real-world experience data, this study provides compelling evidence regarding the need for health equity in this patient population.⁷