More II It than Meets the Eye: Outcomes After Single-Fraction Stereotactic Radiosurgery in a Case Series of Low-Grade Arteriovenous Malformations

Abstract BACKGROUND Surgical resection is typically cited as the optimal treatment of patients with Spetzler-Martin Grade I-II arteriovenous malformation (AVM). OBJECTIVE To report our experience with single-fraction stereotactic radiosurgery (SRS) for Spetzler-Martin Grade I-II AVM. METHODS A prospectively maintained registry was reviewed for patients with nonsyndromic Spetzler-Martin Grade I-II AVM having SRS from 1990 to 2011. Patients with <24 mo of follow-up or prior radiotherapy/SRS were excluded, resulting in a study population of 173 patients. Actuarial analysis was performed using the Kaplan-Meier method, and Cox proportional hazards modeling was performed with excellent outcomes (obliteration without new deficits) as the dependent variable. RESULTS Median post-SRS follow-up was 68 mo (range, 24-275). AVM obliteration was achieved in 132 (76%) after initial SRS. Eleven additional patients achieved obliteration after repeat SRS for an overall obliteration rate of 83%. The rate of obliteration was 60% at 4 yr and 78% at 8 yr. Post-SRS hemorrhage occurred in 7 patients (4%), resulting in 3 minor deficits (2%) and 1 death (<1%). Radiation-induced complications occurred in 5 patients (3%), resulting in minor deficits only. One hundred and thirty-seven patients (79%) had excellent outcomes at last follow-up. CONCLUSION SRS is a safe and effective treatment for patients with Spetzler-Martin Grade I-II AVM. Selection bias is likely a contributing factor to explain the superior outcomes generally noted in reported series of microsurgery for patients with low grade AVM.

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Radiosurgery for low-grade intracranial arteriovenous malformations

Journal of Neurosurgery ◽

10.3171/2014.1.jns131713 ◽

2014 ◽

Vol 121 (2) ◽

pp. 457-467 ◽

Cited By ~ 66

Author(s):

Dale Ding ◽

Chun-Po Yen ◽

Zhiyuan Xu ◽

Robert M. Starke ◽

Jason P. Sheehan

Keyword(s):

Arteriovenous Malformations ◽

Proportional Hazards ◽

Cox Proportional Hazards ◽

Low Grade ◽

Surgical Morbidity ◽

Radiation Induced ◽

Prescription Dose ◽

Martin Grade ◽

Induced Changes

Object Low-grade, or Spetzler-Martin (SM) Grades I and II, arteriovenous malformations (AVMs) are associated with lower surgical morbidity rates than higher-grade lesions. While radiosurgery is now widely accepted as an effective treatment approach for AVMs, the risks and benefits of the procedure for low-grade AVMs, as compared with microsurgery, remain poorly understood. The authors of this study present the outcomes for a large cohort of low-grade AVMs treated with radiosurgery. Methods From an institutional radiosurgery database comprising approximately 1450 AVM cases, all patients with SM Grade I and II lesions were identified. Patients with less than 2 years of radiological follow-up, except those with complete AVM obliteration, were excluded from analysis. Univariate and multivariate Cox proportional-hazards and logistic regression analyses were used to determine factors associated with obliteration, radiation-induced changes (RICs), and hemorrhage following radiosurgery. Results Five hundred two patients harboring low-grade AVMs were eligible for analysis. The median age was 35 years, 50% of patients were male, and the most common presentation was hemorrhage (47%). The median AVM volume and prescription dose were 2.4 cm3 and 23 Gy, respectively. The median radiological and clinical follow-up intervals were 48 and 62 months, respectively. The cumulative obliteration rate was 76%. The median time to obliteration was 40 months, and the actuarial obliteration rates were 66% and 80% at 5 and 10 years, respectively. Independent predictors of obliteration were no preradiosurgery embolization (p < 0.001), decreased AVM volume (p = 0.005), single draining vein (p = 0.013), lower radiosurgery-based AVM scale score (p = 0.016), and lower Virginia Radiosurgery AVM Scale (Virginia RAS) score (p = 0.001). The annual postradiosurgery hemorrhage rate was 1.4% with increased AVM volume (p = 0.034) and lower prescription dose (p = 0.006) as independent predictors. Symptomatic and permanent RICs were observed in 8.2% and 1.4% of patients, respectively. No preradiosurgery hemorrhage (p = 0.011), a decreased prescription dose (p = 0.038), and a higher Virginia RAS score (p = 0.001) were independently associated with postradiosurgery RICs. Conclusions Spetzler-Martin Grade I and II AVMs are very amenable to successful treatment with stereotactic radiosurgery. While patient, physician, and institutional preferences frequently dictate the final course of treatment, radiosurgery offers a favorable risk-to-benefit profile for the management of low-grade AVMs.

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P109 Impact of ethnicity on colorectal cancer incidence in a cohort of colitis-associated dysplasia patients

Journal of Crohn s and Colitis ◽

10.1093/ecco-jcc/jjab076.236 ◽

2021 ◽

Vol 15 (Supplement_1) ◽

pp. S201-S202

Author(s):

M Kabir ◽

K Curtius ◽

P Kalia ◽

I Al Bakir ◽

C H R Choi ◽

...

Keyword(s):

Colorectal Cancer ◽

Racial Differences ◽

Proportional Hazards ◽

Hazard Analysis ◽

Cox Proportional Hazards ◽

Time Interval ◽

Low Grade ◽

Kaplan Meier ◽

The Impact

Abstract Background Racial disparities in inflammatory bowel disease (IBD) phenotypic presentations and outcomes are recognised. However, there are conflicting data from Western population-based cohort studies as to whether racial differences in colitis-associated colorectal cancer (CRC) incidence exists. To our knowledge this is the first study to investigate the impact of ethnicity on the natural history of dysplasia in ulcerative colitis (UC). Methods We performed a retrospective multi-centre cohort study of adult patients with UC whose first low-grade dysplasia (LGD) diagnosis within the extent of colitis was made between 1 January 2001 and 30 December 2018. Only patients with at least one follow-up colonoscopy or colectomy by 30 August 2019 were included. The study end point was time to CRC or end of follow-up. Statistical differences between groups were evaluated using Mann-Whitney U tests and Chi-squared tests. Survival analyses were performed using Kaplan-Meier estimation and multivariate Cox proportional hazards models. Results 408 patients met the inclusion criteria (see Figure 1 for patient and clinical demographics). More patients from a Black or Asian (BAME) background progressed to CRC [13.4% vs. 6.4%; p=0.036] compared to their White Caucasian counterparts, despite having surveillance follow-up. Figure 2 displays Kaplan-Meier curves demonstrating the probability of remaining CRC-free after LGD diagnosis and categorised by ethnicity. BAME patients were more likely to have moderate-severe inflammatory activity on colonic biopsy within the 5 preceding years [42.0% vs. 28.9%; p=0.023], but no significant differences in medication use and a longer median time interval from LGD diagnosis to colectomy date [32 months vs. 11 months; p=0.021]. After adjusting for sex, age and UC duration at time of LGD diagnosis and presence of moderate-severe histological inflammation, being Black or Asian was a predictive factor for CRC progression on multivariate Cox proportional hazard analysis [HR 2.97 (95% CI 1.22 – 7.20); p = 0.016]. However, ethnicity was no longer predictive of CRC progression on sub-analysis of the 317 patients who did not have a colectomy during the follow-up period. Conclusion In this UK multi-centre cohort of UC surveillance patients diagnosed with LGD, delays in receiving cancer preventative colectomy may contribute to an increased CRC incidence in certain ethnic groups. Further work is required to elucidate whether these delays are related to institutional factors (e.g. inequity in the content of decision-making support given or access to healthcare) or cultural factors.

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The Efficacy of Etoposide-Based Therapy in Adult Secondary Hemophagocytic Lymphohistiocytosis

Acta Haematologica ◽

10.1159/000514920 ◽

2021 ◽

pp. 1-9

Author(s):

Leonard Naymagon ◽

Douglas Tremblay ◽

John Mascarenhas

Keyword(s):

Hemophagocytic Lymphohistiocytosis ◽

Proportional Hazards ◽

Multivariable Analysis ◽

Cox Proportional Hazards ◽

Rank Test ◽

Kaplan Meier ◽

Hazard Ratios ◽

Significant Difference ◽

The Impact

Data supporting the use of etoposide-based therapy in hemophagocytic lymphohistiocytosis (HLH) arise largely from pediatric studies. There is a lack of comparable data among adult patients with secondary HLH. We conducted a retrospective study to assess the impact of etoposide-based therapy on outcomes in adult secondary HLH. The primary outcome was overall survival. The log-rank test was used to compare Kaplan-Meier distributions of time-to-event outcomes. Multivariable Cox proportional hazards modeling was used to estimate adjusted hazard ratios (HRs) with 95% confidence intervals (CIs). Ninety adults with secondary HLH seen between January 1, 2009, and January 6, 2020, were included. Forty-two patients (47%) received etoposide-based therapy, while 48 (53%) received treatment only for their inciting proinflammatory condition. Thirty-three patients in the etoposide group (72%) and 32 in the no-etoposide group (67%) died during follow-up. Median survival in the etoposide and no-etoposide groups was 1.04 and 1.39 months, respectively. There was no significant difference in survival between the etoposide and no-etoposide groups (log-rank <i>p</i> = 0.4146). On multivariable analysis, there was no association between treatment with etoposide and survival (HR for death with etoposide = 1.067, 95% CI: 0.633–1.799, <i>p</i> = 0.8084). Use of etoposide-based therapy was not associated with improvement in outcomes in this large cohort of adult secondary HLH patients.

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Impact of Pathological Stratification on the Clinical Outcomes of Advanced Well-Differentiated/Dedifferentiated Liposarcoma Treated with Trabectedin

Cancers ◽

10.3390/cancers13061453 ◽

2021 ◽

Vol 13 (6) ◽

pp. 1453

Author(s):

Chiara Fabbroni ◽

Giovanni Fucà ◽

Francesca Ligorio ◽

Elena Fumagalli ◽

Marta Barisella ◽

...

Keyword(s):

Clinical Outcomes ◽

Proportional Hazards ◽

Case Series ◽

Progression Free Survival ◽

Cox Proportional Hazards ◽

Low Grade ◽

High Grade ◽

Retrospective Case ◽

Well Differentiated ◽

The Impact

Background. We previously showed that grading can prognosticate the outcome of retroperitoneal liposarcoma (LPS). In the present study, we aimed to explore the impact of pathological stratification using grading on the clinical outcomes of patients with advanced well-differentiated LPS (WDLPS) and dedifferentiated LPS (DDLPS) treated with trabectedin. Patients: We included patients with advanced WDLPS and DDLPS treated with trabectedin at the Fondazione IRCCS Istituto Nazionale dei Tumori between April 2003 and November 2019. Tumors were categorized in WDLPS, low-grade DDLPS, and high-grade DDLPS according to the 2020 WHO classification. Patients were divided in two cohorts: Low-grade (WDLPS/low-grade DDLPS) and high-grade (high-grade DDLPS). Results: A total of 49 patients were included: 17 (35%) in the low-grade cohort and 32 (65%) in the high-grade cohort. Response rate was 47% in the low-grade cohort versus 9.4% in the high-grade cohort (logistic regression p = 0.006). Median progression-free survival (PFS) was 13.7 months in the low-grade cohort and 3.2 months in the high-grade cohort. Grading was confirmed as an independent predictor of PFS in the Cox proportional-hazards regression multivariable model (adjusted hazard ratio low-grade vs. high-grade: 0.45, 95% confidence interval: 0.22–0.94; adjusted p = 0.035). Conclusions: In this retrospective case series, sensitivity to trabectedin was higher in WDLPS/low-grade DDLPS than in high-grade DDLPS. If confirmed in larger series, grading could represent an effective tool to personalize the treatment with trabectedin in patients with advanced LPS.

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Abstract 14293: Time to Wound Healing and Major Adverse Limb Events in Patients With Critical Limb Ischemia Treated With Endovascular Therapy

Circulation ◽

10.1161/circ.132.suppl_3.14293 ◽

2015 ◽

Vol 132 (suppl_3) ◽

Author(s):

Grant W Reed ◽

Negar Salehi ◽

Pejman Raeisi-Giglou ◽

Umair Malik ◽

Rami Kafa ◽

...

Keyword(s):

Wound Healing ◽

Endovascular Therapy ◽

Critical Limb Ischemia ◽

Proportional Hazards ◽

Limb Ischemia ◽

Major Amputation ◽

Cox Proportional Hazards ◽

Time Dependent ◽

Kaplan Meier

Introduction: There have been few studies evaluating the influence of time to wound healing on outcomes in patients with critical limb ischemia (CLI) after endovascular therapy. Methods: In this prospective study, patients with CLI treated with endovascular therapy were assessed for comorbidities, presence of wounds, wound healing, and major adverse limb events (MALE; major amputation, surgical endartectomy, or bypass) over time. The incidence of MALE was compared across patient and wound characteristics by Kaplan-Meier analysis. Associations between these variables and MALE were determined by Cox proportional hazards analysis. Results: A total of 252 consecutive patients with CLI were treated between November 1, 2011 and April 1, 2015; 179 (71%) had wounds, of which 97 (54%) healed. During median follow-up of 12.7 months (interquartile range 3.9 - 23.9 months), 46 (18%) had MALE. Wounds were associated with a greater risk of MALE (Hazard Ratio [HR] 3.5; 95% Confidence Interval [CI] 1.4-8.9; p=0.008). As a time-dependent covariate, wound healing was associated with less MALE (HR 0.23; 95% CI 0.10-0.53; p<0.001), and MALE was more frequent in patients with unhealed wounds (23% vs 11%; p<0.0001) (Figure - A). There was significantly less MALE in patients whose wounds healed within 4 months (24% vs 10%; p=0.032) (Figure - B), and less major amputation in those with healed wounds within 3 months (16% vs 5%; p=0.033). After multivariate adjustment for age, presence of diabetes, renal function, wound size, and procedural failure, independent predictors of MALE were wound healing as a time-dependent covariate (HR 0.18; 95% CI 0.08 - 0.40; p<0.0001), and creatinine ≥ 2 (HR 2.3; 95% CI 1.3-4.2; p=0.005). Conclusions: A shorter time to wound healing is associated with less MALE in patients with CLI after endovascular therapy. Efforts should be made to achieve wound healing as quickly as possible in this population, especially in those with renal dysfunction.

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A Proposal of a Personalized Surveillance Strategy for Gastric Cancer: A Retrospective Analysis of 9191 Patients

Gastroenterology Research and Practice ◽

10.1155/2019/3248727 ◽

2019 ◽

Vol 2019 ◽

pp. 1-9 ◽

Cited By ~ 3

Author(s):

Si-wei Pan ◽

Peng-liang Wang ◽

Han-wei Huang ◽

Lei Luo ◽

Xin Wang ◽

...

Keyword(s):

Gastric Cancer ◽

Proportional Hazards ◽

Cox Proportional Hazards ◽

Rank Test ◽

Surveillance Strategy ◽

Risk Of Death ◽

Kaplan Meier ◽

Advanced Stages ◽

Personalized Cancer

Background. In gastric cancer, various surveillance strategies are suggested in international guidelines. The current study is intended to evaluate the current strategies and provide more personalized proposals for personalized cancer medicine. Materials and Methods. In the aggregate, 9191 patients with gastric cancer after gastrectomy from 1998 to 2009 were selected from the Surveillance, Epidemiology, and End Results database. Disease-specific survival was analyzed by Kaplan-Meier method and the log-rank test. Cox proportional hazards regression analyses were used to confirm the independent prognostic factors. As well, hazard ratio (HR) curves were used to compare the risk of death over time. Conditional survival (CS) was applied to dynamically assess the prognosis after each follow-up. Results. Comparisons from HR curves on different stages showed that earlier stages had distinctly lower HR than advanced stages. The curve of stage IIA was flat and more likely the same as that of stage I while that of stage IIB is like that of stage III with an obvious peak. After estimating CS at intervals of three months, six months, and 12 months in different periods, stages I and IIA had high levels of CS all along, while there were visible differences among CS levels of stages IIB and III. Conclusions. The frequency of follow-up for early stages, like stages I and IIA, could be every six months or longer in the first three years and annually thereafter. And those with unfavorable conditions, such as stages IIB and III, could be followed up much more frequently and sufficiently than usual.

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Efficacy and safety of selective internal radiation therapy with yttrium-90 for the treatment of unresectable hepatocellular carcinoma

BMC Gastroenterology ◽

10.1186/s12876-021-01805-6 ◽

2021 ◽

Vol 21 (1) ◽

Author(s):

Nguyen Van Thai ◽

Nguyen Tien Thinh ◽

Thai Doan Ky ◽

Mai Hong Bang ◽

Dinh Truong Giang ◽

...

Keyword(s):

Prognostic Factors ◽

Proportional Hazards ◽

Tumor Response ◽

Complete Response ◽

Tumor Burden ◽

Cox Proportional Hazards ◽

Factors Affecting ◽

Kaplan Meier ◽

Modified Recist

Abstract Background This retrospective analysis was undertaken to evaluate the efficiency of SIRT with Y-90 microspheres and determined prognostic factors affecting patients with unresectable HCC. Methods A total of 97 patients diagnosed with unresectable HCC who underwent SIRT with Y-90 microspheres. Patient survival was assessed using the Kaplan–Meier method, and prognostic factors affecting survival were assessed using log-rank tests and Cox proportional hazards regression. Results Among the 97 patients (90 males, mean age 60.4 ± 12.3 years) who underwent SIRT, the median clinical follow-up was 16.4 (1.8–62) months. The median overall survival (OS) was 23.9 ± 2.4 months. Tumor response according to the Modified RECIST in patients followed up beyond 6 months included a complete response (CR) to treatment in 12 patients (18.8%), partial response (PR) in 23 (35.8%), stable disease (SD) in 8 (12.5%), and progressive disease (PD) in 21 (32.8%). Factors associated with longer OS included age > 65 years, BCLC stage B, tumor size < 5 cm, tumor burden < 25%, and tumor response (CR/PR). In multivariate analysis, unilobar disease and objective tumor response (CR/PR) were predictors of longer OS. Conclusion SIRT was an effective treatment for unresectable HCC. Unilobar disease before SIRT and tumor response (CR/PR) were positive prognostic factors.

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Pretreatment albumin may help select patients for intrahepatic Y-90 microsphere transarterial radioembolization (TARE) for malignancies of the liver.

Journal of Clinical Oncology ◽

10.1200/jco.2015.33.3_suppl.453 ◽

2015 ◽

Vol 33 (3_suppl) ◽

pp. 453-453

Author(s):

Kelly Elizabeth Orwat ◽

Samuel Lewis Cooper ◽

Michael Ashenafi ◽

M. Bret Anderson ◽

Marcelo Guimaraes ◽

...

Keyword(s):

South Carolina ◽

Proportional Hazards ◽

Cox Proportional Hazards ◽

University Of South Carolina ◽

Liver Malignancies ◽

Kaplan Meier ◽

Cox Proportional Hazards Models ◽

Synthetic Function ◽

Radiation Induced ◽

Grade 3

453 Background: Systemic therapies for unresectable liver malignancies may provide a survival benefit, but eventually prove intolerable or ineffective. TARE provides an additional liver-directed treatment option to improve local control for these patients, but there is limited data on patient factors associated with survival. Methods: All patients that received TARE at the Medical University of South Carolina from March 2006 through May of 2014 were included in this analysis of overall survival (OS) and toxicity. Kaplan-Meier estimates of OS from date of first procedure are reported. Potential prognostic factors for OS were evaluated using log rank tests and Cox proportional hazards models. Results: In 114 patients that received TARE at our institution, median follow-up was 6.4 months [range 0-86], with the following tumor histology: colorectal (CR) n=55, hepatocellular (HC) n=20, cholangiocarcinoma (CC) n=16, neuroendocrine (NE) n=12, breast (BR) n=6, other n=5. At least 1 line of systemic therapy prior to TARE was noted in 79% of patients. Median OS was 6.6 months and 1-year OS was 30.7%. The percentage of patients who died within 3 months of TARE were 46.2% for patients with albumin < 3 but only 20.3% for patients with albumin ≥ 3. Grade ≥ 2 toxicity was observed in 22 patients (19.3%) including 9 (7.9%) with Grade 3 and 1 (0.9%) with Grade 4 toxicity. A single patient with a pre-existing pulmonary arteriovenous malformation experienced Grade 3 pneumonitis that resolved with steroids. No deaths were attributed to radiation-induced liver disease. Conclusions: TARE is a relatively safe and effective treatment for unresectable intrahepatic malignancies. Patients with NE or BR histology as well as those with better hepatic synthetic function were associated with significantly better survival. Our data suggest that patients with albumin below 3 may not benefit from TARE. [Table: see text]

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Population-based cohort study examining the association between splenectomy and empyema in adults in Taiwan

BMJ Open ◽

10.1136/bmjopen-2016-015101 ◽

2017 ◽

Vol 7 (9) ◽

pp. e015101 ◽

Cited By ~ 12

Author(s):

Hsien-Feng Lin ◽

Kuan-Fu Liao ◽

Ching-Mei Chang ◽

Cheng-Li Lin ◽

Shih-Wei Lai

Keyword(s):

Cohort Study ◽

Incidence Rate ◽

Proportional Hazards ◽

Population Based ◽

Cox Proportional Hazards ◽

Proportional Hazards Regression ◽

Kaplan Meier ◽

Confounding Variables ◽

Taiwan National Health Insurance

ObjectiveThis study aimed to investigate the association between splenectomy and empyema in Taiwan.MethodsA population-based cohort study was conducted using the hospitalisation dataset of the Taiwan National Health Insurance Program. A total of 13 193 subjects aged 20–84 years who were newly diagnosed with splenectomy from 2000 to 2010 were enrolled in the splenectomy group and 52 464 randomly selected subjects without splenectomy were enrolled in the non-splenectomy group. Both groups were matched by sex, age, comorbidities and the index year of undergoing splenectomy. The incidence of empyema at the end of 2011 was calculated. A multivariable Cox proportional hazards regression model was used to estimate the HR with 95% CI of empyema associated with splenectomy and other comorbidities.ResultsThe overall incidence rate of empyema was 2.56-fold higher in the splenectomy group than in the non-splenectomy group (8.85 vs 3.46 per 1000 person-years). The Kaplan-Meier analysis revealed a higher cumulative incidence of empyema in the splenectomy group than in the non-splenectomy group (6.99% vs 3.37% at the end of follow-up). After adjusting for confounding variables, the adjusted HR of empyema was 2.89 for the splenectomy group compared with that for the non-splenectomy group. Further analysis revealed that HR of empyema was 4.52 for subjects with splenectomy alone.ConclusionThe incidence rate ratio between the splenectomy and non-splenectomy groups reduced from 2.87 in the first 5 years of follow-up to 1.73 in the period following the 5 years. Future studies are required to confirm whether a longer follow-up period would further reduce this average ratio. For the splenectomy group, the overall HR of developing empyema was 2.89 after adjusting for age, sex and comorbidities, which was identified from previous literature. The risk of empyema following splenectomy remains high despite the absence of these comorbidities.

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A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment

Journal of Neuromuscular Diseases ◽

10.3233/jnd-210665 ◽

2021 ◽

pp. 1-14 ◽

Cited By ~ 1

Author(s):

Olga Mitelman ◽

Hoda Z. Abdel-Hamid ◽

Barry J. Byrne ◽

Anne M. Connolly ◽

Peter Heydemann ◽

...

Keyword(s):

Natural History ◽

Repeated Measures ◽

Proportional Hazards ◽

Clinical Care ◽

Standard Of Care ◽

Cox Proportional Hazards ◽

Kaplan Meier ◽

Cox Proportional Hazards Models

Background: Studies 4658-201/202 (201/202) evaluated treatment effects of eteplirsen over 4 years in patients with Duchenne muscular dystrophy and confirmed exon-51 amenable genetic mutations. Chart review Study 4658-405 (405) further followed these patients while receiving eteplirsen during usual clinical care. Objective: To compare long-term clinical outcomes of eteplirsen-treated patients from Studies 201/202/405 with those of external controls. Methods: Median total follow-up time was approximately 6 years of eteplirsen treatment. Outcomes included loss of ambulation (LOA) and percent-predicted forced vital capacity (FVC%p). Time to LOA was compared between eteplirsen-treated patients and standard of care (SOC) external controls and was measured from eteplirsen initiation in 201/202 or, in the SOC group, from the first study visit. Comparisons were conducted using univariate Kaplan-Meier analyses and log-rank tests, and multivariate Cox proportional hazards models with regression adjustment for baseline characteristics. Annual change in FVC%p was compared between eteplirsen-treated patients and natural history study patients using linear mixed models with repeated measures. Results: Data were included from all 12 patients in Studies 201/202 and the 10 patients with available data from 405. Median age at LOA was 15.16 years. Eteplirsen-treated patients experienced a statistically significant longer median time to LOA by 2.09 years (5.09 vs. 3.00 years, p < 0.01) and significantly attenuated rates of pulmonary decline vs. natural history patients (FVC%p change: –3.3 vs. –6.0 percentage points annually, p < 0.0001). Conclusions: Study 405 highlights the functional benefits of eteplirsen on ambulatory and pulmonary function outcomes up to 7 years of follow-up in comparison to external controls.

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