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Ravulizumab is a second-generation C5i engineered from eculizumab to achieve immediate, complete, and sustained inhibition of terminal complement activity in PNH. The REACTION observational cohort study describes the effectiveness and tolerability of ravulizumab in Italian patients who were previously treated with eculizumab. Eighty-one PNH patients were enrolled in this study. The primary endpoint was the percentage change in lactate dehydrogenase (LDH) from baseline to the end of observation (52 weeks follow-up). Among secondary endpoints, transfusion avoidance, breakthrough hemolysis (BTH) and patients’ quality of life (QoL) were evaluated. The median (25–75 percentiles) percentage change in LDH at 52 weeks follow-up was -2.6 (-11.5–13.4) U/L, with 92.3% of the patients presenting LDH within or < 1.5 × upper limit of normal (ULN). Overall, 20 (25.0%) patients required transfusion during the eculizumab period and 15 (18.8%) during the ravulizumab. Seven BTH events were observed, 5 during eculizumab period and 2 (triggered by other medical conditions) during ravulizumab, suggesting the reduction of pharmacokinetic BTH during ravulizumab treatment. EORTC-QLQ-C30 and FACIT-Fatigue scores were similar to the general population, and patients’ preference indicated ravulizumab as the favorite treatment. The REACTION study confirmed the effectiveness of ravulizumab in maintaining stable disease and hemolysis control in the real-world setting. Clinical trial registration. NCT05274633, 02-Mar-2022.

We report the case of a young woman who developed aplastic anemia (AA), following a serologically confirmed primary Epstein–Barr virus (EBV) infection, occurring with fever and pharyngotonsillitis, in the absence of either palpable lymph nodes or enlarged spleen. Pancytopenia persisted after EBV DNA clearance, requiring multiple red blood cell and platelet transfusions. Given the availability of a human leukocyte antigen (HLA)-matched sibling donor (MSD), hematopoietic stem cell transplantation (HSCT) from bone marrow source was performed after a non-myeloablative conditioning regimen with cyclophosphamide and thymoglobulin. Graft-versus-host disease (GVHD) prophylaxis consisted of cyclosporine A and methotrexate. EBV reactivation occurred, one month post-HSCT, peaking at 28,838 DNA copies/ml in peripheral blood, without evidence of post-transplant lymphoproliferative disorder. Two pre-emptive doses of rituximab were administered, resulting in sustained EBV DNA negativity. Subsequent bone marrow evaluation showed normal cellularity and restoration of peripheral counts. After two years of follow-up, the patient remains transfusion-independent, with stable hematologic recovery, no signs of GVHD, and persistent mixed chimerism (70–75% host cells in peripheral blood; about 60% donor CD3 + lymphocytes). To our knowledge, this is the only second reported case of EBV-related AA successfully treated with MSD HSCT. This case underscores the importance of assessing EBV serology in all patients with AA, since EBV infection may be mild or subclinical, and highlights the efficacy of early rituximab administration in high-level EBV DNA reactivation after transplantation.

ObjectivesAlthough early palliative care (EPC) is beneficial in acute myeloid leukaemia, little is known about EPC value in multiple myeloma (MM). We compared quality indicators for palliative and end-of-life (EOL) care in patients with MM receiving EPC with those of patients who received usual haematological care (UHC).MethodsThis observational, retrospective study was based on 290 consecutive patients with MM. The following indicators were abstracted: providing psychological support, assessing/managing pain, discussing goals of care, promoting advance care plan, accessing home care services; no anti-MM treatment within 14 and 30 days and hospice length of stay >7 days before death; no cardiopulmonary resuscitation, no intubation, <2 hospitalisations and emergency department visits within 30 days before death. Comparisons were performed using unadjusted and confounder-adjusted regression models.Results55 patients received EPC and 231 UHC. Compared with UHC patients, EPC patients had a significantly higher number of quality indicators of care (mean 2.62±1.25 vs 1.12±0.95; p<0.0001)); a significant reduction of pain intensity over time (p<0.01) and a trend towards reduced aggressiveness at EOL, with the same survival (5.3 vs 5.46 years; p=0.74)).ConclusionsOur data support the value of integrating EPC into MM routine practice and lay the groundwork for future prospective comparative studies.

Background/Objectives: International guidelines recommend the use of orthoses in subjects with cerebral palsy (CP), even though there is limited evidence of their effectiveness. Little is known about their effectiveness in children and adolescents with other types of neuromotor disability. Methods: The review protocol was recorded on the PROSPERO register (CRD42024509165) and conformed to the PRISMA guidelines. The inclusion criteria were any type of ankle–foot orthoses (AFOs); pediatric subjects with any non-acquired neuromotor disease; any type of outcome measure regarding gait performance; controlled studies; and those in the English language. Screening, selection, risk of bias assessment, and data extraction were performed by a group of independent researchers. Results: Fifty-seven reports were included, with most regarding CP; three involved subjects with Charcot–Marie–Tooth disease or Duchenne dystrophy. Nine were RCTs. A meta-analysis was performed for studies including subjects with CP. The meta-analysis demonstrated the effectiveness of AFOs in increasing stride length (MD −10.21 [−13.92, −6.51]), ankle dorsiflexion at IC (MD 9.66 [7.05, 12.27]), and peak ankle DF in stance (MD 5.72 [2.34, 9.09]) while reducing cadence (MD 0.13 [0.06, 0.17]) and the energy cost of walking (MD −0.02 [−0.03, −0.00]). The peak ankle power generated at push-off was significantly increased with flexible AFOs compared to rigid AFOs (MD 0.38 [0.30, 0.46]), but it decreased with both compared to walking barefoot or with shoes (MD −0.35 [−0.49, −0.22]). Evidence regarding DMD and CMT was limited but suggested opting for individualized flexible AFOs, which preserved peak ankle power generation. Conclusions: AFOs improve gait performance in CP. Flexible AFOs are preferable because they preserve the peak ankle power generated at push-off compared to rigid AFOs.