Citations

ABSTRACT This study assessed the level of agreement of perceived improvements among pediatric patients with chronic pain and their parents following an interdisciplinary pain management program. This is a retrospective chart review of a heterogeneous group of pediatric patients with chronic pain who completed an interdisciplinary pain management program (IPMP) and their parents ( N = 172 pairs). The IPMP took place in an outpatient pain management center affiliated with an academic rehabilitation hospital. The outcome measures included the Multidimensional Patient Impression of Change (MPIC) and the MPIC parent version were administered post‐treatment. The levels of agreement on the MPIC were analyzed between child and parent using the free‐marginal kappa statistic. The majority of both the parents and the children evaluated the patients to be much or very much improved in six of the eight domains assessed. The levels of agreement in the ratings were fair to moderate among the patient–parent dyads. Parents endorsed significantly higher levels of improved overall pain and mood than their child did, whereas the patients reported greater levels of improved ability to cope with pain than their parents did. Both the patients and their parents reported high levels of perceived improvement following the IPMP. However, there were some differences in their ratings of improvements in overall pain, mood, and ability to cope with pain. Given that some of the perceptions were discrepant, it is recommended that feedback be obtained from the patient and their parent.

Abstract Background Breast cancer chemoprevention is underutilized among high-risk women. We examined whether decision support could increase informed decision making. Methods We conducted a cluster randomized controlled trial to evaluate the effect of a patient-facing decision aid (RealRisks) and provider tool (BNAV) compared with standard education on the number of women with high-risk breast lesions making informed choices. Questionnaires were administered at baseline and 6 and 12 months. The primary outcome was chemoprevention informed choice (defined as adequate knowledge and attitudes congruent with decision) at 6 months. Secondary endpoints included breast cancer risk perceptions, worry, chemoprevention knowledge, decision conflict, and chemoprevention decision. Results Across 31 randomized sites, we enrolled 210 providers, who were primarily breast health specialists. Of these, 24 sites (14 intervention, 10 control) enrolled 412 patients. Among 287 patients evaluable for the primary outcome, there were no statistically significant differences in informed choice between the intervention and control arms at 6 months (35% vs 27%, respectively; P = .20) and 12 months (37% vs 25%, respectively; P = .05). At 6 months, women in the intervention compared with control arm were more likely to have accurate breast cancer risk perceptions (31% vs 21%, respectively; P = .03) and adequate chemoprevention knowledge (33% vs 23%, respectively; P = .04). At 12 months, 52% of women in the control and 50% in the intervention arm self-reported initiating chemoprevention. Conclusion Decision support led to modest improvements in accurate risk perceptions and chemoprevention knowledge but not informed choice. Relatively high chemoprevention uptake was achieved among women with high-risk breast lesions managed mainly by breast health specialists. Trial Registration NCT04496739, https://clinicaltrials.gov/study/NCT04496739?cond=NCT04496739&viewType=Card&rank=1

Background: Patients with spina bifida are living longer and more productive lives; however, many continue to experience functional limitations in adulthood. This study evaluated long-term functional progression and ambulatory status using the combined Myelomeningocele Functional Classification (MMFC) and Functional Mobility Scale (FMS), and examined factors associated with long-term changes. Methods: A retrospective review at a tertiary care rehabilitation center identified 168 patients with spina bifida. MMFC and FMS classifications were recorded at initial and final visits. A subgroup of ambulatory patients who underwent 3D-gait analysis was analyzed to assess the relationship between MMFC, body mass index (BMI), and functional speed (FS) using multiple linear regression. Results: The average follow-up was 15±10.31 years. The mean age at final follow-up was 20 years (range, 5 to 52), with 52% of patients being male. Fifty-two percent of patients were ≥18 years of age at final follow-up. Within the MMFC1 cohort, 48% used wheelchair mobility (FMS 1,1,1) at initial presentation; this increased to 80% at final follow-up. Eighty percent of the MMFC2 patients were able to complete the 50-yard walk test at initial classification, whereas only 60% were able to complete it at final follow-up. Within the MMFC3 cohort, there was a decline in patients requiring no external support (FMS 6,6,6) from 90% to 78%. All MMFC4 patients maintained an FMS of 6,6,6. A statistically significant relationship was found between functional speed (FS) and MMFC (with MMFC4 faster than MMFC3 and MMFC2) ( P =0.01414). Conclusions: Long-term follow-up demonstrates that functional classification may decline over time in MMFC1 to 3 cohorts, whereas MMFC4 remains stable. In addition, within individual cohorts (MMFC1 to 3), there was also a decline in FMS and functional walk testing. A positive and statistically significant relationship was observed between gait analysis FS and MMFC, and a negative and statistically significant relationship was observed between BMI and MMFC. The combined use of MMFC and FMS provides a structured framework for longitudinal functional assessment in patients with spina bifida. Level of evidence: Level III.

e13615 Background: Bacillus Calmette-Guérin (BCG) remains the standard intravesical therapy for high-risk non-muscle-invasive bladder cancer (NMIBC), but weekly clinic-based instillations create substantial travel, time, and indirect costs that may threaten adherence. Therefore, we proposed a pilot trial of delivering BCG at Home as a prospective, single-center pilot testing a pharmacy-to-home pathway with digital patient-reported outcome (PRO) monitoring. Methods: BCG at Home is a prospective, single-center pilot evaluating feasibility and safety of home delivery of induction BCG for treatment-naïve, high-grade T1 and/or carcinoma in situ NMIBC. Dose 1 is administered in clinic to confirm tolerability and catheterization feasibility; doses 2-6 are administered at home by Penn Medicine at Home nurses after a 3 month training and proctorship program in intravesical therapy and exposure precautions. To enable transport beyond conventional short post-preparation windows, the pharmacy prepares BCG using a closed system transfer device (CSTD); bench testing demonstrated stability and sterility for 24 hours after connection. A medical courier delivers the prepared medication to the patient’s home, where the nurse receives the delivery and performs the instillation. The primary endpoint is induction adherence (completion of all recommended doses). Secondary endpoints include PROs (EORTC QLQ-C30/QLQ-NMIBC24), satisfaction, and acute care utilization. PROs are captured before and after treatment using a conversational chatbot; domain summaries are assessed at baseline, 6 weeks, and 12 weeks. Results: The pilot began enrollment in June 2025 (planned sample size: 20). To date, eight participants have enrolled and completed induction. Median age was 76 years (range 41-88); 50% were female and 25% were Black/African American. Participants lived a mean of 30 miles from the treating facility. Forty home instillations were delivered, and all participants completed all recommended induction doses (100% adherence). Median nurse time in the home was 40 minutes. Five episodes of hematuria were self-limited; one urinary tract infection resolved with antibiotics. Self-efficacy improved from a median of 3 pre-treatment to 7 post-treatment. The median COST-FACIT score was 30 (scale 1-44). Mean EORTC QLQ-C30 global health status was 71 (0-100). EORTC QLQ-NMIBC24 averages showed urinary symptoms rising during induction (23 at baseline to 30 at 6 weeks) with improvement by 12 weeks (18) and future worries decreasing (33 to 25); intravesical treatment issues remained low (5 to 2). Conclusions: BCG at Home is, to our knowledge, the first prospective trial to administer intravesical BCG in the home setting using a standardized pharmacy-to-home pathway. Early results support feasibility, complete adherence, and an acceptable short-term safety profile. Clinical trial information: 07048496 .

7094 Background: Waldenström Macroglobulinemia (WM) remains incurable with current available therapeutic options. Treatment options for relapsed/refractory pts are limited and often continuous, underscoring the need for new agents with novel mechanisms of action and pt-centered, fixed duration therapy. Iopofosine I 131 is a novel radiopharmaceutical compound composed of a lipid raft-targeting phospholipid ether covalently bound to 131 I, a beta emitting radioisotope. Here, we report preliminary sub-group results of the CLOVER-WaM study [NCT02952508] in WM pts treated with a Bruton’s Tyrosine Kinase inhibitor (BTKi) immediately prior to iopofosine. Methods: Eligible WM pts had received >2 prior therapies and had symptomatic disease requiring therapy according to consensus recommendations for WM. Treatment consisted of 2 cycles of iopofosine I 131 administered at 15 mCi/m 2 on days 1 and 15 of each 57-day cycle. The primary efficacy endpoint was major response rate (MRR; partial response or better, as determined by the modified IWWM-VI). Secondary and exploratory endpoints included overall response rate (ORR), treatment-free survival (TFS), progression free survival (PFS), duration of response (DOR), and clinical benefit rate (CBR; stable disease or better). Herein, efficacy endpoints were assessed in the modified intent-to-treat (mITT) population (all pts who received ≥60 mCi total administered dose of iopofosine I 131), and whose treatment regimen prior to iopofosine included a BTKi. Safety was evaluated using CTCAE v4.03 in pts who received ≥1 dose of iopofosine. Data cut off for this analysis was Jan. 16, 2026. Results: As previously reported, 65 pts were dosed and 55 are included in the mITT population. Of those, 22 (40%) had received a BTKi containing regimen immediately prior to iopofosine; and 11 who previously received at least 2 regimens utilizing BTKis and 12 who were refractory to rituximab. In this subgroup, median age was 70.5 (range 55-88), 17 (77%) were male, and 5 (23%) were female. The median number of prior therapies was 3 (range 2-7), 15/22 (68%) were MYD88 mutated and 18/22 (82%) were TP53 wildtype. The MRR was 15/22 (68.2%), ORR was 19/22 (86.4%) and CBR was 100%, median PFS was 12 months (range 1.6 to 28.0) and median TFS was 19.1 months (range 2.5-34.4). Grade ≥ 3, related treatment emergent adverse events (TEAE) occurring in > 10% of pts included anemia (41%), leukopenia (41%), neutropenia (58%), and thrombocytopenia (86%). Conclusions: Iopofosine I 131 demonstrated significant efficacy; 100% CBR, 86.4% ORR and 68.2% MRR with a median PFS of 12 months (range: 1.6 – 28.0 months) in a pt population with no current standard of care therapy. The treatment was well-tolerated with a manageable toxicity profile with cytopenias as the only Grade 3 or greater TEAE. Based on the results of this pivotal trial, iopofosine I 131 could be a promising novel treatment for WM pts after BTKi therapy. Clinical trial information: NCT02952508 .

Background: Spasticity after upper motor neuron injury (stroke, spinal cord injury, etc.) alters muscle activity and induces long-term structural remodeling of skeletal muscle [1]. Elbow flexors, including the biceps brachii, are frequently affected, leading to abnormal tone, involuntary co-contraction, and impaired elbow function. Hyper-selective neurectomy (HSN) is a well-established surgical technique that has gained renewed clinical adoption with advances in anatomic understanding and intraoperative nerve mapping [2], yet its effects on muscle properties are poorly defined. Objective: The purpose of this study was to define pre- vs. post-HSN differences in muscle gene expression, sarcomere length, and clinical outcomes of the spastic human biceps. Methods: Individuals with elbow flexor spasticity undergoing a planned two-stage HSN were prospectively enrolled (n=10). Stage 1 released the elbow flexors and provided a pre-biopsy; Stage 2, performed ~3 months later to treat the forearm flexors, provided a second exposure to the biceps and a second biopsy. In both stages, biceps biopsies were obtained with customized clamps [3] at predefined elbow angles (50°, 90°, 135°), fixed, and analyzed for sarcomere length [4]. Total RNA was extracted from pre- and post-HSN biceps samples to characterize transcriptional changes. Clinical spasticity was assessed before Stage 1 and again before Stage 2 using the Modified Ashworth Scale (MAS). Data were compared using repeated-measures models, and single-cell transcriptional changes were analyzed using established analytical pipelines. Results: Sarcomere length (SL) increased significantly after HSN at all joint angles. At 50°, SL increased from 2.45 ± 0.18 µm pre-HSN to 3.05 ± 0.28 µm post-HSN; at 90°, from 2.70 ± 0.29 µm to 2.91 ± 0.16 µm; and at 135°, from 2.72 ± 0.36 µm to 3.24 ± 0.32 µm (all p < 0.05). Clinical spasticity assessment improved significantly as well (p < 0.05), with MAS scores decreasing from 1.89± 0.93 to 0.35 ± 0.53. RNA sequencing revealed changes in key pathways and overall gene expression patterns such as enhanced inflammatory response through increased cytokine production and fibroblast proliferation, leading to elevated collagen synthesis and subsequent fibrosis. We also observed upregulated immune modulation and chemotaxis, while pathways involved in mitochondrial respiration, muscle development, and cellular homeostasis were downregulated. At the transcript level, HSN led to decreased inflammation and adipogenesis, while promoting increased extracellular matrix assembly, and vascular formation. Conclusion: HSN produced coordinated improvements across structural, clinical, and transcriptional levels in the spastic human biceps muscle. Sarcomere lengths increased toward longer resting lengths at all joint angles, clinical spasticity decreased significantly, and RNA sequencing revealed distinct post-operative transcriptional remodeling. The longer sarcomere length and decreased spasticity should contribute to improved patient function after HSN. References: 1. Lieber RL et al. Muscle Nerve. 2002;26:98–105. 2. Leclercq C et al. Hand Clin. 2018;34:537–545. 3. Lieber RL et al. J Neurophysiol. 1994;71:874–881. 4. Ward SR et al. J Biomech. 2009;42:193–196. This abstract was presented at the American Physiology Summit 2026 and is only available in HTML format. There is no downloadable file or PDF version. The Physiology editorial board was not involved in the peer review process.