Citations

Abstract Therapeutic options for Waldenström macroglobulinemia (WM) have expanded but improvements in overall-survival (OS) are difficult to interpret due to competing non–WM mortality. We conducted a large single-center retrospective study of 1,020 patients with WM to evaluate survival trends. Patients were stratified by era of active WM diagnosis using a two-era model (1996–2005 and post-2005) and a four-era model (pre-2001, 2001–2007, 2008–2013, and post-2013). OS did not differ significantly across eras in the overall cohort (p = 0.64 and p = 0.86 for the two- and four-era models). However, among patients aged < 65 years at the time of active WM diagnosis in the 2-era model, OS improved in the post-2006 era (median 18.8 vs 14.1 years, p = 0.018). Cause-specific-survival (CSS) improved across all eras, increasing from 15.6 years in 1996–2005 to 18.9 years post-2006 (p < 0.0001), and from 13.7 years pre-2001 to not reached in later periods in the four-era model (p < 0.0001). Competing-risk-analysis (CRA) demonstrated a reduction in WM-related mortality over time (p < 0.001). However, excess mortality persisted, with a standardized mortality ratio (SMR) of 2.79 (p < 0.001) among patients < 65 years compared with a matched U.S. population. Our findings highlight improvements in WM-specific survival, while gains in OS are observed in younger patients.

Objectives To evaluate biochemical recurrence‐free survival (BRFS) and metastasis‐free survival (MFS) as potential intermediate clinical endpoints (ICEs) for overall survival (OS) in patients undergoing salvage radical prostatectomy (sRP). Patients and Methods Evaluable patients were selected from a retrospective dataset, resulting in a cohort of 879 patients with recurrent, non‐metastatic, hormone‐sensitive prostate cancer treated with sRP at 13 centres. ICE surrogacy was evaluated using a two‐stage approach: (i) at the individual‐patient level by fitting Clayton copula models and estimating Kendall's ( >0.7 indicating a strong association) and (ii) at the centre level, by fitting weighted linear regression of 5‐year OS on 3‐year BRFS and MFS. Results The two‐step analysis included 759 patients for BRFS (366 events) and 476 for MFS (137 events), with median follow‐up of 37 months (95% confidence interval [CI] 36–42 months) and 34 months (95% CI 31–37 months), respectively. At the individual‐patient level, MFS showed a strong association with OS (Kendall's 0.85, 95% CI 0.82–0.88), which was not observed for BRFS (Kendall's 0.63, 95% CI 0.56–0.69). At the centre level, neither 3‐year BRFS ( R 2 = 0.15; slope = 0.34, P = 0.2) nor 3‐year MFS ( R 2 = 0.21; slope = 0.26, P = 0.14) predicted 5‐year OS with sufficient explanatory power. Limitations include the fact that centre‐level analysis was based on single‐arm associations rather than on treatment‐effect surrogacy across arms, and retrospective data collection. Conclusions The BRFS should not be used as a surrogate endpoint in the sRP setting. MFS requires further validation in future prospective studies to confirm its association with OS. Patient‐reported outcomes, such as quality‐of‐life and treatment‐related toxicity, should be considered in parallel with OS.

Purpose To evaluate retear rates, clinical outcomes, and complications of a consecutive series of full‐thickness rotator cuff repair cases augmented with an autograft biceps patch. Methods A retrospective review was performed of patients undergoing rotator cuff repair with autograft biceps patch augmentation. Patients underwent either magnetic resonance imaging or ultrasound to evaluate rotator cuff repair integrity at a minimum of 6 months postoperatively. Patient‐reported outcomes including the American Shoulder and Elbow Surgeons Score and Single Assessment Numeric Evaluation score were also collected and evaluated for clinical significance at a minimum of 12 months postoperatively. Analysis of retear rates was performed by comparing observed retear rates to expected retear rates using the modified Rotator Cuff Healing Index score. Results The final cohort included 25 rotator cuff repair cases. Postoperative imaging was obtained by magnetic resonance imaging in 22 patients at a mean of 9 months postoperatively (range, 6.0‐15.3 months) and ultrasound in 3 patients at a mean of 30.4 months (range, 28.8‐32.2 months). The expected retear rate using the modified Rotator Cuff Healing Index score was 21.5% for the entire cohort. There was 1 retear, resulting in an actual retear rate of 4% at an average of 23.5‐month follow‐up ( P = .0196). The average Single Assessment Numeric Evaluation score was 93 (standard deviation 9), and the average American Shoulder and Elbow Surgeons Score was 96 (standard deviation 6.3) at an average of 23.5‐month follow‐up (range 14‐32). Patient acceptable symptomatic state was achieved by 96% of the cohort for the American Shoulder and Elbow Surgeons Score and 88% of the cohort for the Single Assessment Numeric Evaluation score. There were no cases of reoperation or complications. Conclusions In this consecutive series, the biceps autograft “smash” patch for rotator cuff augmentation resulted in a low rate of retears, favorable patient‐reported outcomes, and no complications. This augmentation option has the potential to be safe and effective for improving rotator cuff repair healing rates. Level of Evidence Level IV, retrospective case series.

Primary central nervous system choriocarcinoma is a non-germinomatous germ cell tumor with a highly malignant character, comprising up to 5% of all central nervous system germ cell tumors. Locations affected can include suprasellar or pineal locations most commonly, with others including the basal ganglia and lateral ventricles. Guidelines do not specify an optimal radiation regimen but recommend proceeding with maximally safe resection if feasible and then chemotherapy before radiotherapy. Radiation is often comprised of craniospinal irradiation with subsequent tumor bed boost. We present a case of an adult male with a primary central nervous system choriocarcinoma who underwent reversed sequence radiation therapy with photon tumor bed boost before proton craniospinal irradiation due to recurrent intracranial hemorrhages with long-term control of cancer and no subsequent hemorrhages. This case highlights the excellent outcome and feasibility of reversed sequence radiotherapy on choriocarcinoma in hemorrhage and tumor control.