scholarly journals Stereotactic Radiosurgery for the Treatment of Primary and Metastatic Spinal Sarcomas

2016 ◽  
Vol 16 (3) ◽  
pp. 276-284 ◽  
Author(s):  
Jacob A. Miller ◽  
Ehsan H. Balagamwala ◽  
Lilyana Angelov ◽  
John H. Suh ◽  
Toufik Djemil ◽  
...  
Keyword(s):  
Pain Relief ◽  
Stereotactic Radiosurgery ◽  
Median Time ◽  
Primary Outcome ◽  
Local Treatment ◽  
Hazard Ratio ◽  
Conventional Radiotherapy ◽  
History Of ◽  
Prescription Dose ◽  
Secondary Outcomes

Purpose: Despite advancements in local and systemic therapy, metastasis remains common in the natural history of sarcomas. Unfortunately, such metastases are the most significant source of morbidity and mortality in this heterogeneous disease. As a classically radioresistant histology, stereotactic radiosurgery has emerged to control spinal sarcomas and provide palliation. However, there is a lack of data regarding pain relief and relapse following stereotactic radiosurgery. Methods: We queried a retrospective institutional database of patients who underwent spine stereotactic radiosurgery for primary and metastatic sarcomas. The primary outcome was pain relief following stereotactic radiosurgery. Secondary outcomes included progression of pain, radiographic failure, and development of toxicities following treatment. Results: Forty treatment sites were eligible for inclusion; the median prescription dose was 16 Gy in a single fraction. Median time to radiographic failure was 14 months. At 6 and 12 months, radiographic control was 63% and 51%, respectively. Among patients presenting with pain, median time to pain relief was 1 month. Actuarial pain relief at 6 months was 82%. Median time to pain progression was 10 months; at 12 months, actuarial pain progression was 51%. Following multivariate analysis, presence of neurologic deficit at consult (hazard ratio: 2.48, P < .01) and presence of extraspinal bone metastases (hazard ratio: 2.83, P < .01) were associated with pain relief. Greater pain at consult (hazard ratio: 1.92, P < .01), prior radiotherapy (hazard ratio: 4.65, P = .02), and greater number of irradiated vertebral levels were associated with pain progression. Conclusions: Local treatment of spinal sarcomas has remained a challenge for decades, with poor rates of local control and limited pain relief following conventional radiotherapy. In this series, pain relief was achieved in 82% of treatments at 6 months, with half of patients experiencing pain progression by 12 months. Given minimal toxicity and suboptimal pain control at 12 months, dose escalation beyond 16 Gy is warranted.

Is a picture-perfect thrombectomy necessary in acute ischemic stroke?

2021 ◽  
pp. neurintsurg-2020-017193
Author(s):  
Ching-Jen Chen ◽  
Reda Chalhoub ◽  
Dale Ding ◽  
Jeyan S Kumar ◽  
Natasha Ironside ◽  
...  
Keyword(s):  
Primary Outcome ◽  
Mechanical Thrombectomy ◽  
National Institutes Of Health ◽  
Stroke Patients ◽  
Anterior Circulation ◽  
Vessel Occlusion ◽  
Symptomatic Intracerebral Hemorrhage ◽  
History Of ◽  
Multivariable Models ◽  
Secondary Outcomes

BackgroundThe benefit of complete reperfusion (modified Thrombolysis in Cerebral Infarction (mTICI) 3) over near-complete reperfusion (≥90%, mTICI 2c) remains unclear. The goal of this study is to compare clinical outcomes between mechanical thrombectomy (MT)-treated stroke patients with mTICI 2c versus 3.MethodsThis is a retrospective study from the Stroke Thrombectomy and Aneurysm Registry (STAR) comprising 33 centers. Adults with anterior circulation arterial vessel occlusion who underwent MT yielding mTICI 2c or mTICI 3 reperfusion were included. Patients were categorized based on reperfusion grade achieved. Primary outcome was modified Rankin Scale (mRS) 0–2 at 90 days. Secondary outcomes were mRS scores at discharge and 90 days, National Institutes of Health Stroke Scale score at discharge, procedure-related complications, and symptomatic intracerebral hemorrhage.ResultsThe unmatched mTICI 2c and mTICI 3 cohorts comprised 519 and 1923 patients, respectively. There was no difference in primary (42.4% vs 45.1%; p=0.264) or secondary outcomes between the unmatched cohorts. Reperfusion status (mTICI 2c vs 3) was also not predictive of the primary outcome in non-imputed and imputed multivariable models. The matched cohorts each comprised 191 patients. Primary (39.8% vs 47.6%; p=0.122) and secondary outcomes were also similar between the matched cohorts, except the 90-day mRS which was lower in the matched mTICI 3 cohort (p=0.049). There were increased odds of the primary outcome with mTICI 3 in patients with baseline mRS ≥2 (36% vs 7.7%; p=0.011; pinteraction=0.014) and a history of stroke (42.3% vs 15.4%; p=0.027; pinteraction=0.041).ConclusionsComplete and near-complete reperfusion after MT appear to confer comparable outcomes in patients with acute stroke.

Implementation of a pharmacy consult service for evaluation of immune checkpoint inhibitor related adverse events at a large community hospital

2020 ◽  
pp. 107815522097026
Author(s):  
Jeff Kamta ◽  
Bren Magruder ◽  
Lisa Hymel
Keyword(s):  
Adverse Events ◽  
Immune Checkpoint ◽  
Immune Checkpoint Inhibitors ◽  
Primary Outcome ◽  
Checkpoint Inhibitors ◽  
Delayed Presentation ◽  
Consult Service ◽  
Organ Systems ◽  
History Of ◽  
Secondary Outcomes

Introduction Immune checkpoint inhibitors (ICI) are novel oncolytic therapies associated with various immune related adverse events (irAEs) affecting multiple organ systems, which may have a delayed presentation. Identification of irAEs and prompt initiation of appropriate treatment represents a challenge to clinicians. The purpose of this study was to evaluate the effectiveness of a pharmacy consult service in identification and management of irAEs. Methodology: This was a single center, retrospective study. Patients included were: ≥18 years old, admitted as inpatients, and reported a history of cancer treatment within the last year. A pharmacy consult was developed and implemented for patients who reported a history of ICI therapy within the last year. Education regarding the consult service was provided to select physicians, nurses, and all pharmacists. Primary outcome: percent of admitted patients reporting ICI therapy within the last year, who required pharmacist intervention for an irAE. Secondary outcomes: types of interventions performed, percentage of recommendation acceptance, pharmacist time spent. Results Fifty-one patients received a pharmacy immunotherapy consult. Seventeen patients (33%) met the primary outcome. Thirty-three separate recommendations were made by pharmacists for these 17 patients. The secondary outcomes of interventions made and percentage accepted (n; % accepted): Initiation/adjustment of steroid therapy (20; 40%), placement of a consult for oncology or other specialist (10; 70%), other therapeutic interventions (3; 67%). Average time spent by pharmacist on initial consultations (SD): 29 minutes (15). Conclusion A pharmacy consult service may help to increase identification of patients receiving immune checkpoint inhibitors and initiate timely interventions.

scholarly journals Conventional Radiotherapy and Stereotactic Radiosurgery in the Management of Metastatic Spine Disease

2020 ◽  
Vol 19 ◽  
pp. 153303382094579
Author(s):  
Hao-ran Zhang ◽  
Ji-kai Li ◽  
Xiong-gang Yang ◽  
Rui-qi Qiao ◽  
Yong-Cheng Hu
Keyword(s):  
Spinal Cord ◽  
Pain Relief ◽  
Life Expectancy ◽  
Stereotactic Radiosurgery ◽  
Spinal Cord Compression ◽  
Spinal Metastases ◽  
Scoring Systems ◽  
Cord Compression ◽  
Conventional Radiotherapy ◽  
Radiation Induced

Spinal metastases are a common manifestation of malignant tumors that can cause severe pain, spinal cord compression, pathological fractures, and hypercalcemia, and these clinical manifestations will ultimately reduce the health-related quality of life and even shorten life expectancy in patient with cancer. Effective management of spinal bone metastases requires multidisciplinary collaboration, including radiologists, surgeons, radiation oncologists, medical oncologists, and pain specialists. In the past few decades, conventional radiotherapy has been the most common form of radiotherapy, which can achieve favorable local control and pain relief; however, it lacks precise methods of delivering radiation and thus cannot provide sufficient tumoricidal dose. The advent of stereotactic radiosurgery has changed this situation by using highly focused radiation beams guided by 3-dimensional imaging to deliver a high biologic equivalent dose to the target region, and the spinal cord can be identified and excluded from the target volume to reduce the risk of radiation-induced myelopathy. Separation surgery can provide a 2- to 3-mm safe separation of tumor and spinal cord to avoid radiation-induced damage to the spinal cord. Targets for separation surgery include decompression of metastatic epidural spinal cord compression and spinal stabilization without partial or en bloc tumor resection. Combined with conventional radiotherapy, stereotactic radiosurgery can provide better local tumor control and pain relief. Several scoring systems have been developed to estimate the life expectancy of patients with spinal metastases treated with radiotherapy. Thorough understanding of radiotherapy-related knowledge including the dose-fractionation schedule, separation surgery, efficacy and safety, scoring systems, and feasibility of combination with other treatment methods is critical to providing optimal patient care.

FC 021EFFICACY OF INTRAVENOUS FERRIC CARBOXYMALTOSE IN PATIENTS WITH IRON DEFICIENCY FOLLOWING ACUTE HEART FAILURE, ACCORDING TO BASELINE EGFR: A SUBGROUP ANALYSIS OF THE AFFIRM-AHF TRIAL

2021 ◽  
Vol 36 (Supplement_1) ◽  
Author(s):  
Iain Macdougall ◽  
Ewa A Jankowska ◽  
Marco Metra ◽  
Gerasimos Filippatos ◽  
Bridget-Anne Kirwan ◽  
...  
Keyword(s):  
Heart Failure ◽  
Placebo Group ◽  
Iron Deficiency ◽  
Medical History ◽  
Rate Ratio ◽  
Primary Outcome ◽  
Ferric Carboxymaltose ◽  
History Of ◽  
Secondary Outcomes ◽  
Post Randomisation

Abstract Background and Aims In the AFFIRM-AHF trial, treatment with intravenous (IV) ferric carboxymaltose (FCM) reduced the risk of heart failure (HF) hospitalisations vs placebo in patients with iron deficiency after an episode of acute HF. Of these patients, 41% had a medical history of chronic kidney disease (CKD). This prespecified subanalysis of AFFIRM-AHF data was performed to investigate the effect of renal function on FCM efficacy. Methods In AFFIRM-AHF, patients stabilised following hospitalisation for acute HF with concomitant iron deficiency (defined as ferritin &lt;100 μg/L, or 100–299 μg/L with transferrin saturation &lt;20%) were randomised to receive either IV FCM or placebo before discharge for the index hospitalisation. In this analysis, patients who had received at least one dose of the study drug, and who had at least one post-randomisation data point and a baseline value for estimated glomerular filtration rate (eGFR; calculated using the CKD-EPI formula and baseline creatinine value), were stratified into tertiles according to baseline eGFR. The primary outcome was a composite of total HF hospitalisations and CV death. Secondary outcomes included total HF hospitalisations, CV death, time to first HF hospitalisation or CV death, composite of total CV hospitalisations and CV death, and days lost due to HF hospitalisations or CV death. All outcomes were evaluated up to 52 weeks post-randomisation. Results Of the 1,108 patients included in primary AFFIRM-AHF analyses, 967 (FCM: 487; placebo: 480) had a baseline eGFR value and were included in this analysis. In both groups, 60% of patients had an eGFR &lt;60 mL/min/1.73 m2 following the index acute HF episode. Patients were divided into eGFR tertiles 1, 2 and 3, with corresponding respective baseline eGFR values of &lt;42.96, 42.96 to &lt;64.32, and ≥64.32 mL/min/1.73 m2. At baseline, the mean age, proportion of females, and proportions of patients with ischaemic HF aetiology, a documented history of HF, and a medical history of percutaneous coronary intervention, coronary artery bypass graft and/or cardiac resynchronisation therapy, were highest in eGFR tertile 1 and lowest in eGFR tertile 3. In eGFR tertiles 1, 2 and 3, the number of total HF hospitalisations and CV deaths in the FCM group vs placebo group were, respectively, 115 vs 152, 76 vs 83, and 56 vs 79, with respective annualised rate ratios (95% confidence interval [CI]) of 0.76 (0.50, 1.16), 0.76 (0.48, 1.22) and 0.69 (0.42, 1.12) (Figure). In eGFR tertile 3, the total number of CV hospitalisations and CV deaths was significantly lower in the FCM group vs the placebo group (69 vs 107; rate ratio [95% CI] 0.60 [0.39, 0.93]), with a nominally lower number of total HF hospitalisations with FCM vs placebo (44 vs 66; rate ratio [95% CI] 0.62 [0.38, 1.01]). In the time to first event analysis, FCM significantly reduced HF hospitalisation or CV death vs placebo in eGFR tertile 3 (hazard ratio [95% CI] 0.64 [0.42, 0.98]). In eGFR tertiles 1 and 2, differences between FCM and placebo arms for secondary endpoints did not reach statistical significance. The p-trend for treatment by baseline eGFR subgroup was non-significant for the primary outcome (0.941) and also for the secondary outcomes specified here. Conclusion In patients with iron deficiency who were stabilised after an episode of acute HF, numerically fewer primary and secondary events, endpoints or outcomes were consistently observed with FCM vs placebo across the eGFR tertiles. In addition, no significant interaction between kidney function and FCM efficacy was noted. Given that this analysis was limited by small patient numbers following subgroup stratification, further studies in larger cohorts with CKD may help to clarify the effect of IV FCM in this patient population.

scholarly journals Spine stereotactic radiosurgery for the treatment of multiple myeloma

2017 ◽  
Vol 26 (3) ◽  
pp. 282-290 ◽  
Author(s):  
Jacob A. Miller ◽  
Ehsan H. Balagamwala ◽  
Samuel T. Chao ◽  
Todd Emch ◽  
John H. Suh ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Radiation Therapy ◽  
Vertebral Fracture ◽  
Pain Relief ◽  
Stereotactic Radiosurgery ◽  
Median Time ◽  
Cumulative Incidence ◽  
Single Fraction ◽  
Pain Scores

OBJECTIVE The objective of this study was to define symptomatic and radiographic outcomes following spine stereotactic radiosurgery (SRS) for the treatment of multiple myeloma. METHODS All patients with pathological diagnoses of myeloma undergoing spine SRS at a single institution were included. Patients with less than 1 month of follow-up were excluded. The primary outcome measure was the cumulative incidence of pain relief after spine SRS, while secondary outcomes included the cumulative incidences of radiographic failure and vertebral fracture. Pain scores before and after treatment were prospectively collected using the Brief Pain Inventory (BPI), a validated questionnaire used to assess severity and impact of pain upon daily functions. RESULTS Fifty-six treatments (in 38 patients) were eligible for inclusion. Epidural disease was present in nearly all treatment sites (77%). Moreover, preexisting vertebral fracture (63%), thecal sac compression (55%), and neural foraminal involvement (48%) were common. Many treatment sites had undergone prior local therapy, including external beam radiation therapy (EBRT; 30%), surgery (23%), and kyphoplasty (21%). At the time of consultation for SRS, the worst, current, and average BPI pain scores at these treatment sites were 6, 4, and 4, respectively. The median prescription dose was 16 Gy in a single fraction. The median clinical follow-up duration after SRS was 26 months. The 6- and 12-month cumulative incidences of radiographic failure were 6% and 9%, respectively. Among painful treatment sites, 41% achieved pain relief adjusted for narcotic usage, with a median time to relief of 1.6 months. The 6- and 12-month cumulative incidences of adjusted pain progression were 13% and 15%, respectively. After SRS, 1-month and 3-month worst, current, and average BPI scores all significantly decreased (p < 0.01). Vertebral fracture occurred following 12 treatments (21%), with an 18% cumulative incidence of fracture at 6 and 12 months. Two patients (4%) developed pain flare following spine SRS. CONCLUSIONS This study reports the largest series of myeloma lesions treated with spine SRS. A rapid and durable symptomatic response was observed, with a median time to pain relief of 1.6 months. This response was durable among 85% of patients at 12 months following treatment, with 91% local control. The efficacy and minimal toxicity of spine SRS is likely related to the delivery of ablative and conformal radiation doses to the target. SRS should be considered with doses of 14–16 Gy in a single fraction for patients with multiple myeloma and limited spinal disease, myelosuppression requiring “marrow-sparing” radiation therapy, or recurrent disease after EBRT.

scholarly journals Pre-operative fasting times for clear liquids at a tertiary children’s hospital; what can be improved?

2021 ◽  
Author(s):  
Alexander R. Schmidt ◽  
James Fehr ◽  
Janice Man ◽  
Genevieve D’Souza ◽  
Ellen Wang ◽  
...  
Keyword(s):  
Median Time ◽  
Primary Outcome ◽  
Arrival Time ◽  
Task Force ◽  
Pulmonary Aspiration ◽  
Clear Liquid ◽  
Preoperative Fasting ◽  
Parental Compliance ◽  
Secondary Outcomes ◽  
American Society

Background: The goal of preoperative fasting is to prevent pulmonary aspiration during general anesthesia. Fasting times are often prolonged leading to patient discomfort and risk for adverse events. This retrospective quality improvement survey evaluated effective nil-per-os (NPO) times and causes for prolonged NPO times with the aim to suggest improvement strategies by a newly founded fasting task force.Methods: Data from all electronic anesthesia records from 2019 at our institution were reviewed for fasting times. Our NPO instructions follow American Society of Anesthesiology guidelines and are calculated based on the patient’s arrival time (90 min before OR time). Primary outcome was the effective NPO time for clear liquids, secondary outcomes were incidence of delays and the parental compliance with the NPO instructions. Data are presented as median (interquartile range).Results: 9,625 cases were included in the analysis. NPO time was documented in 72.1% with a median effective NPO time of 7:13 h (7:36). OR in room times were documented in 72.8%, 2,075 (29.5%; median time 0:10 h [0:21]) were earlier and 4,939 (70.5%; median time 0:29 h [0:54]) were later than scheduled. Parental NPO compliance showed a median deviation for clear liquid intake of 0:55 h (8:30).Conclusions: This study revealed that effective NPO times were longer than current ASA guidelines. Contributing causes include case delays and parental non-compliance to NPO instructions. Thus, task force recommendations include change NPO instruction calculations to scheduled OR time versus arrival time, and encourage parents to give their child clear liquids at the instructed time.

Nebulised liposomal-amphotericin-B as maintenance therapy in ABPA: a randomised, multicentre, trial

2021 ◽  
pp. 2102218
Author(s):  
Cendrine Godet ◽  
Francis Couturaud ◽  
Sylvain Marchand-Adam ◽  
Christophe Pison ◽  
Frédéric Gagnadoux ◽  
...  
Keyword(s):  
Maintenance Therapy ◽  
Amphotericin B ◽  
Median Time ◽  
Liposomal Amphotericin ◽  
Primary Outcome ◽  
Immunoglobulin E ◽  
Liposomal Amphotericin B ◽  
Absolute Difference ◽  
Clinical Equipoise ◽  
Secondary Outcomes

BackgroundIn allergic bronchopulmonary aspergillosis (ABPA), prolonged nebulised antifungal treatment may be a strategy for maintaining remission.MethodsWe performed a randomised, single-blind, clinical trial in 30 centres. Patients with controlled ABPA after a 4-month attack treatment (corticosteroids and itraconazole) were randomly assigned to nebulised liposomal-amphotericin-B or placebo for 6 months. The primary outcome was occurrence of a first severe clinical exacerbation within 24 months following randomisation. Secondary outcomes included the median time-to-first severe clinical exacerbation, number of severe clinical exacerbations per patient, ABPA-related biological parameters.ResultsAmong 174 enrolled patients with ABPA from March 2015 through July 2017, 139 were controlled after 4-month attack treatment and were randomised. The primary outcome occurred in 33 (50.8%) of 65 patients in nebulised liposomal-amphotericin-B group and 38 (51.3%) of 74 in placebo group (absolute difference −0.6%, 95% CI −16.8% to +15.6%, odds ratio 0.98, 95% CI 0.50 to 1.90; p=0.95). The median time-to-first severe clinical exacerbation was longer in liposomal-amphotericin-B group, 337 days (IQR, 168 to 476) versus 177 (64 to 288). At the end of maintenance therapy, total immunoglobulin-E and Aspergillus precipitins were significantly decreased in nebulised liposomal-amphotericin-B group.ConclusionsIn ABPA, maintenance therapy using nebulised liposomal-amphotericin-B did not reduce the risk of severe clinical exacerbation. The presence of some positive secondary outcomes creates clinical equipoise for further research.

Multimodal Pain Management for Cesarean Delivery: A Double-Blinded, Placebo-Controlled, Randomized Clinical Trial

2019 ◽  
Vol 36 (11) ◽  
pp. 1097-1105 ◽  
Author(s):  
Emily E. Hadley ◽  
Luis Monsivais ◽  
Lucia Pacheco ◽  
Rovnat Babazade ◽  
Giuseppe Chiossi ◽  
...  
Keyword(s):  
Pain Management ◽  
Drug Use ◽  
Cesarean Delivery ◽  
Primary Outcome ◽  
Opioid Use ◽  
Multimodal Pain Management ◽  
Significant Difference ◽  
History Of ◽  
Secondary Outcomes ◽  
Double Blinded

Objective Our objective was to evaluate the efficacy of perioperative multimodal pain management in reducing opioid use after elective cesarean delivery (CD). Study Design A single-center, double-blinded, placebo-controlled randomized trial of women undergoing elective CD. Participants were allocated 1:1 to receive the multimodal protocol or matching placebos. The multimodal protocol consisted of a preoperative dose of intravenous acetaminophen, preincision injection of subcutaneous bupivacaine, and intraoperative injection of intramuscular ketorolac. Primary outcome was total opioid intake at 48 hours postoperatively. Secondary outcomes were pain scores, time to first opioid intake, neonatal outcomes, and total outpatient opioid intake on postoperative day (POD) 7. Data were analyzed using parametric and nonparametric tests and quantile regression as appropriate. Results A total of 242 women were screened with 120 randomized, 60 to the multimodal group and 60 to control group. There was no significant difference in the primary outcome of opioid use nor in the secondary outcomes. Smokers and patients with a history of drug use had higher median postoperative opiate use and earlier administration. On POD 7, only 40% of prescribed opioids had been used, and there was no difference between the groups. Conclusion This perioperative multimodal pain regimen did not reduce opioid use in 48 hours after CD. Patients who smoke or with a history of drug use required more opioids in the postoperative period. Providers significantly overprescribed opioids after CD.

scholarly journals Efficacy and Safety of Azathioprine during Remission of Immune-Mediated Thrombotic Thrombocytopenic Purpura

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 773-773
Author(s):  
Christian Bichard ◽  
Ilaria Mancini ◽  
Pasquale Agosti ◽  
Pasqualina De Leo ◽  
Andrea Artoni ◽  
...  
Keyword(s):  
Adverse Events ◽  
Median Time ◽  
Clinical Remission ◽  
Primary Outcome ◽  
Clinical Relapse ◽  
Adamts13 Activity ◽  
Efficacy And Safety ◽  
Immune Mediated ◽  
Azathioprine Treatment ◽  
Secondary Outcomes

Abstract Introduction Acquired immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare and life-threatening thrombotic microangiopathy caused by the development of anti-ADAMTS13 autoantibodies. Up to 50% of patients surviving an acute iTTP event experience one or more relapses. Recent guidelines suggest treatment with rituximab in clinical remission in iTTP patients with low ADAMTS13 activity to prevent acute iTTP relapses. A 10% of cases who do not respond to rituximab or develop allergic reactions requiring therapy discontinuation have been reported. In these patients, azathioprine might be an alternative treatment to prevent acute iTTP relapses. The aim of this study was to assess the efficacy and safety of azathioprine treatment in iTTP patients in clinical remission. Methods We designed a retrospective cohort study including all iTTP patients treated with azathioprine during clinical remission, enrolled in the Milan TTP Registry between May 2003 and October 2020 and followed for at least six months. The efficacy of azathioprine was assessed in patients with at least four weeks of treatment. The primary outcome was clinical relapse during azathioprine treatment. The secondary outcomes were the partial and complete responses of ADAMTS13 (an ADAMTS13 activity increase above 20% and 45%, respectively) in patients with ADAMTS13 activity below 20% before azathioprine start. ADAMTS13 relapse (an ADAMTS13 activity decrease below 20%) in patients with a prior ADAMTS13 response was also assessed. As for the safety analysis, adverse events (AEs), associated or not with treatment discontinuation, were retrieved from clinical charts of all patients. Lastly, we assessed the association between variables such as sex, age, and concomitant autoimmune diseases, and ADAMTS13 response, using logistic regression models. Results We enrolled 43 patients with iTTP treated with azathioprine during clinical remission (Table 1). Forty-two percent of the patients had at least one concomitant autoimmune disease. The median exposure to azathioprine was 16 months (interquartile range [IQR] 8-49 months), the median dosage 1.3 mg/kg/day (IQR 0.9-1.6 mg/kg/day). Thirty-five cases were available for the primary outcome analysis (Table 2). Seven patients relapsed after a median time of 14 months after azathioprine start (IQR 8-58 months). The cumulative incidence of clinical relapse during azathioprine treatment was 10% at 1 year (95% Confidence Interval [CI] 0-21%), 22% at 2 years (95% CI 6-38%). Twenty-one cases were available for the secondary outcomes analysis. An ADAMTS13 partial response occurred in 10 patients (48%), after a median time of 3 months (IQR 3-9 months) after azathioprine start and lasted for a median time of 40 months (IQR 16-56 months). An ADAMTS13 complete response was achieved in 33% of patients, after a median time of 8 months (IQR 3-16 months) after azathioprine start and lasted for a median time of 16 months (IQR 0-53 months). Of the 10 responders, 3 (30%) had a subsequent ADAMTS13 relapse at 22, 24 and 37 months after azathioprine start. Adverse events were observed in 30% of patients, after a median time of 50 days after azathioprine start (IQR 26-331 days). Liver and pancreas alterations with increased levels of ALT, AST, GGT, ALP and lipase were the most frequent, followed by nausea and leukopenia (Table 3). In one patient, acute myeloid leukemia was diagnosed 6 months after azathioprine start. Fifteen percent of the patients had to stop the treatment due to AEs. No demographic or anamnestic variables were associated with ADAMTS13 response to azathioprine. Conclusions Forty-eight percent of patients attained an ADAMTS13 activity above 20%, with a durable response lasting more than 3 years, while adverse events were generally mild in nature. Azathioprine could be a valid and safe alternative in case of ineligibility or failure to respond to rituximab. Figure 1 Figure 1. Disclosures Mancini: Instrumentation Laboratory, Sanofi: Honoraria. Peyvandi: Roche: Consultancy, Honoraria; Takeda: Consultancy, Honoraria; Sanofi: Consultancy, Honoraria; Sobi: Consultancy, Honoraria. OffLabel Disclosure: Azathioprine. It is an immunosuppressive drug used for the prevention of rejection in renal homotransplantation and for treatment of active rheumatoid arthritis.

scholarly journals Proton Beam Stereotactic Radiosurgery for Pediatric Cerebral Arteriovenous Malformations

Neurosurgery ◽  
2014 ◽  
Vol 74 (4) ◽  
pp. 367-374 ◽  
Author(s):  
Brian P. Walcott ◽  
Jona A. Hattangadi-Gluth ◽  
Christopher J. Stapleton ◽  
Christopher S. Ogilvy ◽  
Paul H. Chapman ◽  
...  
Keyword(s):  
High Risk ◽  
Stereotactic Radiosurgery ◽  
Median Time ◽  
Arteriovenous Malformations ◽  
Pediatric Patients ◽  
Pediatric Population ◽  
Univariate Analysis ◽  
Cerebral Arteriovenous Malformations ◽  
Maximal Diameter ◽  
Prescription Dose

ABSTRACT BACKGROUND: For cerebral arteriovenous malformations (AVMs) determined to be high risk for surgery or endovascular embolization, stereotactic radiosurgery (SRS) is considered the mainstay of treatment. OBJECTIVE: To determine the outcomes of pediatric patients with AVMs treated with proton SRS. METHODS: We reviewed the records of 44 consecutively treated pediatric patients (younger than 18 years of age) who underwent proton SRS at our institution from 1998 to 2010. The median target volume was 4.5 ± 5.9 mL (range, 0.3-29.0 mL) and the median maximal diameter was 3.6 ± 1.5 cm (range, 1-6 cm). Radiation was administered with a median prescription dose of 15.50 ± 1.87 CGE to the 90% isodose. RESULTS: At a median follow-up of 52 ± 25 months, 2 patients (4.5%) had no response, 24 patients (59.1%) had a partial response, and 18 patients (40.9%) experienced obliteration of their AVM. The median time to obliteration was 49 ± 26 months, including 17 patients who underwent repeat proton radiosurgery. Four patients (9%) experienced hemorrhage after treatment at a median time of 45 ± 15 months. Univariate analysis identified modified AVM scale score (P = .045), single fraction treatment (0.04), larger prescription dose (0.01), larger maximum dose (&lt;0.001), and larger minimum dose (0.01) to be associated with AVM obliteration. CONCLUSION: High-risk AVMs can be safely treated with proton radiosurgery in the pediatric population. Because protons deposit energy more selectively than photons, there is the potential benefit of protons to lower the probability of damage to healthy tissue in the developing brain.

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