To evaluate the real-world effectiveness, overall survival (OS), durability of response, and tolerability of daratumumab-based therapy for systemic amyloid light-chain (AL) amyloidosis in Japan, where bortezomib and daratumumab were only recently introduced, using 3-year follow-up data. We retrospectively reviewed 43 patients diagnosed with AL amyloidosis. Fifteen patients received daratumumab-containing regimens (Dara group) and 28 received non-daratumumab therapy (non-Dara group). Clinical characteristics, hematologic response (HR), organ response (OR), OS, and treatment tolerability were compared. Four patients with Mayo 2012 stage IV disease underwent stepwise dose modification of daratumumab-based therapy. The Dara group showed significantly higher HR (93
We report a case of Epstein–Barr virus-associated smooth muscle tumor (EBV-SMT) of the liver that developed two years after umbilical cord blood transplantation (UCBT) for acute myeloid leukemia. A woman in her 50 s was found to have a hepatic mass in segment 3 during imaging follow-up for a gallbladder polyp. Histopathological examination of the resected specimen revealed a fascicular proliferation of spindle-shaped tumor cells that were immunohistochemically positive for α-smooth muscle actin (α-SMA) and h-caldesmon, and positive for Epstein–Barr virus–encoded RNA (EBER) by in situ hybridization. The lesion was therefore diagnosed as EBV-SMT. EBV-SMT is a rare neoplasm that typically arises in immunocompromised individuals. Because immune reconstitution after UCBT requires a prolonged period, delayed recovery of T cell-mediated immunity and post-transplant immunosuppression were considered potential contributing factors in this case. When spindle cell tumors develop in immunosuppressed patients, EBV-SMT should be included in the differential diagnosis.
BackgroundThe Time to Walking Independently after Stroke (TWIST) prediction tool is designed to predict the number of post-stroke weeks at which patients are expected to achieve walking independence. The external validation of the TWIST tool's clinical applicability and generalizability has been desired.ObjectiveWe performed a geographic external validation of TWIST prediction in a multicenter prospective cohort.Patients and MethodsAdult post-stroke patients with lower-limb weakness and inability to walk independently were enrolled. Each patient's TWIST score (max. score: 4 points) was calculated using age, knee-extension strength, and the Berg Balance Scale score at 1 week post-stroke. The TWIST score predicts independent walking by 4, 6, 9, 16, or 26 weeks post-stroke, and we calculated the overall fit, calibration, and discrimination at each of these timepoints to assess the model's performance.ResultsThe validation cohort consisted of 145 patients (median age 73 years, 44% women, 36% moderate-severe stroke). At 9 and 26 post-stroke weeks, 60.7% and 72.4% of the patients achieved walking independence. The overall fit explained a moderate proportion of the outcome's variance (Nagelkerke R2 = 0.28-0.49), and the discrimination performance was good (c-statistic >0.75). Calibration performance showed over-prediction at all timepoints (calibration-in-the-large = -1.62 to -0.34). Higher TWIST scores (3 or 4 points) over-predicted early post-stroke; lower TWIST scores (1 or 2 points) became increasingly over-predictive over time.ConclusionsThe TWIST prediction tool optimistically predicted walking independence in Japanese patients with stroke. Further validation studies are necessary to assess this tool's precise clinical impact and generalizability.Clinical Trial Registration URL:https://www.umin.ac.jp/UMIN000050588.
[Purpose] To clarify the relationship between mothers' health-related quality of life and their perceptions of healthcare services, including physical therapy, for their children with disability, along with factors affecting health-related quality of life. [Participants and Methods] Survey items were the Short Form Health Survey, Measure of Processes of Care 56, as an indicator of the perception of healthcare services, type of disability and activities of daily living, and the mother's sleep patterns and employment status. The correlation between health-related quality of life and service perceptions was investigated. Further, multiple regression analysis was conducted by combining the child's and mother's statuses as an interaction term. [Results] The mental component summary score of the Short Form Health Survey showed a significant weak positive correlation with Measure of Processes of Care 56. The number of awakenings from sleep at night was found to be a significant moderator of the physical component summary score of the Short-Term Health Survey and showed a limited but significant effect on the 'providing general information' domain of Measure of Processes of Care 56. [Conclusion] Mothers with a low awareness of 'providing general information' and a high number of times waking from sleep at night tended to show low health-related quality of life.
