Systemic therapy with bevacizumab in patients with hereditary hemorrhagic telangiectasia (HHT)

VASA ◽  
2013 ◽  
Vol 42 (2) ◽  
pp. 106-110 ◽  
Author(s):  
Ajay Chavan ◽  
Silke Schumann-Binarsch ◽  
Lars Luthe ◽  
Britta Nickau ◽  
Albrecht Elsässer ◽  
...  

Background: Frequent epistaxis, the hallmark of hereditary hemorrhagic telangiectasia (HHT) significantly affects quality of life. Hepatic involvement may be associated with capsular pain, abdominal angina, high-output cardiac failure and portal hypertension with ascites and variceal bleeding. Liver transplantation as well as hepatic artery embolization as invasive treatment options for hepatic involvement are both associated with a certain morbidity and mortality. Patients and methods: Three patients with HHT and symptomatic hepatic involvement prospectively underwent off-label systemic treatment with the vascular endothelial growth factor (VEGF)-inhibitor Bevacizumab in 6 cycles. Clinical symptoms and cardiac output were assessed before as well as 3 months after therapy. Results: At 3 months follow-up, capsular pain and abdominal angina were significantly reduced in two of the three patients (Numerical Rating Scale (NRS) grade 4 and 5 changed to grade 2). Cardiac output, which was initially raised in both patients, normalized and was associated with an improvement in the signs and symptoms of cardiac insufficiency by one stage according to the NYHA classification in both patients. Symptoms however returned back gradually to pre-therapeutic levels after 9 months in one of these two patients. In the third patient with a normal cardiac status prior to therapy, a marked subjective improvement in the performance status, fatigability and quality of life was noted. Grade 2 nasal bleeding reduced to grade 0 - 1 in all three patients; this clinical improvement was persistent at 3, 7 and 9 months follow-up. No significant lasting complications were observed. Conclusions: Sytemic Bevacizumab therapy could become an important therapy option in the non-invasive medical treatment of patients with HHT. Further studies to document long-term results, to determine the appropriate drug dosage as well as to evaluate the necessity of a maintenance drug regime are warranted.

Neurology ◽  
2019 ◽  
Vol 93 (24) ◽  
pp. e2284-e2293 ◽  
Author(s):  
Casey H. Halpern ◽  
Veronica Santini ◽  
Nir Lipsman ◽  
Andres M. Lozano ◽  
Michael L. Schwartz ◽  
...  

ObjectiveTo test the hypothesis that transcranial magnetic resonance–guided focused ultrasound (tcMRgFUS) thalamotomy is effective, durable, and safe for patients with medication-refractory essential tremor (ET), we assessed clinical outcomes at 3-year follow-up of a controlled multicenter prospective trial.MethodsOutcomes were based on the Clinical Rating Scale for Tremor, including hand combined tremor–motor (scale of 0–32), functional disability (scale of 0–32), and postural tremor (scale of 0–4) scores, and total scores from the Quality of Life in Essential Tremor Questionnaire (scale of 0–100). Scores at 36 months were compared with baseline and at 6 months after treatment to assess for efficacy and durability. Adverse events were also reported.ResultsMeasured scores remained improved from baseline to 36 months (all p < 0.0001). Range of improvement from baseline was 38%–50% in hand tremor, 43%–56% in disability, 50%–75% in postural tremor, and 27%–42% in quality of life. When compared to scores at 6 months, median scores increased for hand tremor (95% confidence interval [CI] 0–2, p = 0.0098) and disability (95% CI 1–4, p = 0.0001). During the third follow-up year, all previously noted adverse events remained mild or moderate, none worsened, 2 resolved, and no new adverse events occurred.ConclusionsResults at 3 years after unilateral tcMRgFUS thalamotomy for ET show continued benefit, and no progressive or delayed complications. Patients may experience mild degradation in some treatment metrics by 3 years, though improvement from baseline remains significant.Clinicaltrials.gov identifierNCT01827904.Classification of evidenceThis study provides Class IV evidence that for patients with severe ET, unilateral tcMRgFUS thalamotomy provides durable benefit after 3 years.


2016 ◽  
Vol 7 (01) ◽  
pp. 87-90 ◽  
Author(s):  
Serdal Albayrak ◽  
Sait Ozturk ◽  
Emre Durdag ◽  
Ömer Ayden

ABSTRACT Background: Aim of this paper is to recall the surgical technique used in the recurrent lumbar disc herniations (LDHs) and to share our experiences. Materials and Methods: Out of series of 1115 patients who underwent operations for LDH between 2006 and 2013, 70 patients underwent re-operations, which were included in this study. During surgery, lateral decompression performed over the medial facet joint to the superior facet joint border was seen after widening the laminectomy defect, and microdiscectomy was performed. The demographic findings of the patients, their complaints in admission to hospital, the level of operation, the condition of dural injury, the first admission in the prospective analysis, and their quality of life were evaluated through the Oswestry scoring during their postoperative 1st, 3rd, 6th-month and 1st, 3rd, 5th and 7th-year follow-up. In the statical analysis, Friedman test was performed for the comparison of the Oswestry scores and Siegel Castellan test was used for the paired nonparametrical data. A P < 0.05 was considered statistically significant. Results: Considering the Oswestry Index during the follow-ups, the values in the postoperative early period and follow-ups were seen to be significantly lower than those at the time of admission to hospital (P < 0.05). None of the patients, who re-operated by microdiscectomy, presented with iatrogenic instability in 7 years follow-up period. Conclusion: Microdiscectomy performed through a proper technique in the re-operation of recurrent disc herniations eases complaints and improves the quality of life. Long-term follow-ups are required for more accurate results.


2019 ◽  
Vol 39 (2) ◽  
pp. 112-118 ◽  
Author(s):  
Osasuyi Iyasere ◽  
Edwina Brown ◽  
Fabiana Gordon ◽  
Helen Collinson ◽  
Richard Fielding ◽  
...  

Background In-center hemodialysis (HD) has been the standard treatment for older dialysis patients, but reports suggest an associated decline in physical and cognitive function. Cross-sectional data suggest that assisted peritoneal dialysis (aPD), an alternative treatment, is associated with quality of life (QoL) outcomes that are comparable to in-center HD. We compared longitudinal changes in QoL between modalities. Methods We enrolled 106 aPD patients, matched with 100 HD patients from 20 renal centers in England and Northern Ireland. Patients were assessed quarterly for 2 years using the Hospital Anxiety and Depression Scale (HADS), SF-12 physical and mental scores, symptom score, Illness Intrusiveness Rating Scale (IIRS), Barthel's score, and the Renal Treatment Satisfaction Questionnaire (RTSQ). Mixed model analysis was used to assess the impact of dialysis modality on these outcomes during follow-up. P values were adjusted for multiple significance testing. Results Multivariate analysis showed no difference in any of the outcome measures between aPD and HD. Longitudinal trends in outcomes were also not significantly different. Higher age at baseline was associated with lower IIRS and RTSQ scores during follow-up. One-hundred and twenty-five (60.6%) patients dropped out of the study: 59 (28.6%) died, 61 (29.6%) withdrew during follow-up, and 5 (2.5%) were transplanted. Conclusions Quality of life outcomes in frail older aPD patients were equivalent to those receiving in-center HD. Assisted PD is thus a valid alternative to HD for older people with end-stage kidney disease (ESKD) wishing to dialyze at home.


2008 ◽  
Vol 28 (2) ◽  
pp. 81-88 ◽  
Author(s):  
Linda M. Martin ◽  
Michelle Bliven ◽  
Rosemary Boisvert

A non-randomized pretest—posttest design was used to evaluate changes in occupational performance, self-esteem, and quality of life among clients completing a substance abuse recovery program that included occupational therapy services addressing life skills. Seventy-five clients participated in intake and discharge assessment (4- to 6-month interval) that used the Occupational Performance History Interview (Version 2.0), the Rosenberg Self-Esteem Scale, and the Quality of Life Rating scale. A limited number of follow-up interviews at 3 and 6 months were also conducted. Marked improvement with significant differences and large effect sizes were found between intake and discharge scores for all measures. Most clients scored in the range of moderate occupational dysfunction at discharge, when their highest performance level was seen. Follow-up data revealed that occupational performance declined at 3 months and improved again at 6 months. Change in occupational performance is clearly reflected in the recovery process, but recovery is challenged when clients leave the supportive halfway house environment.


Neurosurgery ◽  
2014 ◽  
Vol 75 (5) ◽  
pp. 509-514 ◽  
Author(s):  
Roberto Tarantino ◽  
Pasquale Donnarumma ◽  
Loenzo Nigro ◽  
Marika Rullo ◽  
Antonio Santoro ◽  
...  

Abstract BACKGROUND: Intradural extramedullary tumors (IDEMTs) are uncommon lesions that cause pain and neurological deficits. OBJECTIVE: To evaluate the effects of surgery for IDEMTs. METHODS: This cohort study recruited all patients operated on for IDEMTs at the Department of Neurology and Psychiatry of Sapienza University of Rome from January 2003 to January 2013. The analysis was conducted on clinical records evaluation over a 1-year follow-up. The Graphic Rating Scale was used to assess pain. Neurological deficits were detected through neurological examination. Quality of life was evaluated with the EuroQol (EQ-5D). Statistical interpretation of the data was performed with SPSS version 19 software. RESULTS: One hundred seven patients were recruited. Three were lost to follow-up. Patients reported lower level of pain 1 year after surgery (before surgery, 6.05; after surgery, 3.65). Mean comparison showed a significant decrease of −2.400 (P &lt; .001). Ninety-two patients (88.5%) were neurologically asymptomatic 1 year after surgery. Only 12 patients (11.5%) presented with a deficit, with a global decrease of 39% (χ2 = 27.6; P &lt; .005). The quality of life in patients was middle to high (mean rating of EQ-5D visual analog score, 61.78%). The lowest levels of quality of life were found in patients with sphincter dysfunctions (mean, 33.4). CONCLUSION: Surgery for IDEMTs has a good outcome. Patients reported lower levels of pain and a drastic reduction in neurological symptoms 1 year after surgery. The quality of life is middle to high. It is influenced mainly by the neurological outcome.


2015 ◽  
Vol 11 (2) ◽  
pp. 321-326 ◽  
Author(s):  
Matthieu Bruzzi ◽  
Cédric Rau ◽  
Thibault Voron ◽  
Martino Guenzi ◽  
Anne Berger ◽  
...  

2019 ◽  
Vol 25 (3) ◽  
pp. 37-46 ◽  
Author(s):  
A. N. Kovalenko ◽  
A. A. Dzhavadov ◽  
I. I. Shubnyakov ◽  
S. S. Bilyk ◽  
A. O. Denisov ◽  
...  

Introduction. The matter of the replacement of acetabular defects is becoming increasingly relevant today. In their clinic the authors used custom-made implants with trabecular coating for replacement of defects in the acetabulum where and when it was necessary to ensure extended fixation of the component. Purpose — to evaluate short-term clinical and functional outcomes, osteointegration of porous coated custom-made implants, the overall survival of structural components and to identify the causes of failures and complications. Materials and Methods. During the study the authors interviewed and evaluated radiographs of 48 patients operated from October 2015 to June 2018. The patients were interviewed before and after the surgery using the Oxford hip joint scale, quality of life EQ-5D and visual analogue pain scale. Radiographs were evaluated for a period of at least 12 months for the signs of osteointegration. Results. The average OHS scores increased from 14.9 (±7) to 37.6 (±7) (p<0.01). Quality of life according to the EQ-5D index increased from 0.2 (±0.2) to 0.7 (±0.2) (p<0.01). The average value by general health scales: before operations — 49 (±17) and 73 (±18) scores after surgeries (p<0.01). The average VAS pain score decreased from 73 (±10) to 19 (±19) points (p<0.01). Osteointegration was observed in 98 percent of cases. Migration of the implant with fracture of a flange was observed in one case. Conclusions. In the short-term follow up the use of custom-made implants significantly improves hip joint function and the patients’ life quality. Custom-made implants enable primary reliable fixation in case of complex acetabular defects. High rate of osteointegration is observed for custom-made implants with porous coating within at least 12 months. Longer follow-up is needed to evaluate long-term results.


2017 ◽  
Vol 98 (5) ◽  
pp. 709-713
Author(s):  
S V Tarasenko ◽  
A A Natal’skiy ◽  
O V Zaytsev ◽  
S N Sokolova ◽  
A Yu Bogomolov ◽  
...  

Aim. Comparative analysis of the long-term results of surgical treatment of patients with chronic pancreatitis. Methods. The study included patients with complicated clinical forms of chronic pancreatitis including: ductal pancreatic hypertension, main pancreatic duct concrements, marked fibrous restructuring of the pancreatic parenchyma and presence of calcifications, presence of biliary hypertension, portal hypertension syndrome, duodenostasis syndrome, chronic abdominal pain syndrome, retention and post-necrotic cysts. Patients were divided into 3 groups: group 1 included patients with chronic pancreatitis receiving conservative treatment (n=32), group 2 - patients who underwent the Frey pancreatic resection (n=24) and group 3 - Beger pancreatic resection (n=9). The assessment was performed according to the most spread specific questionnaire in gastroenterology GSRS (Gastrointestinal Symptom Rating Scale). Results. The quality of life of patients who underwent both Frey and Beger pancreatic resection was significantly higher than the quality of life of patients in whom chronic pancreatitis was treated conservatively. Quality of life of patients who underwent Frey or Beger pancreatic resection did not differ significantly. Malabsorption syndrome has a significant effect on the quality of life of patients who underwent surgical treatment of chronic pancreatitis. The GSRS questionnaire demonstrated low sensitivity in detecting malabsorption syndrome. Conclusion. Quality of life of patients who underwent surgical treatment of chronic pancreatitis is statistically significantly higher than in patients receiving conservative treatment; correction of existing quality of life questionnaires is required in order to identify malabsorption syndrome.


2013 ◽  
Vol 94 (3) ◽  
pp. 406-408 ◽  
Author(s):  
I V Barkhatov

Aim. To assess the Gastrointestinal Symptom Rating Scale (GSRS) questionnaire as an ambulatory screening test for early diagnosis of abdominal angina. Methods. An assessment of abdominal symptoms severity and quality of life was performed using the GSRS questionnaire in 110 patients with abdominal angina. No sings of abdominal angina and no vascular abnormalities were found at Doppler sonography in 39 (35.5%) of patients (main group). In 71 (64.5%) patients distributed to the second group signs of insufficient blood flow (including vascular wall calcification and thickening, medium and small arteries compression and narrowing up to 40-70%, and inadequate blood flow after exercise. Results. There were no statistically significant differences found in prevalence of abdominal pain, reflux and diarrhea between two groups. Dyspepsia was noted in 29 (74.4%) patients of first group and in 71 (100%) patients of the second group. Average GSRS scores were 3.67±0.51 and 5.07±0.32 accordingly in those patients. Constipation was present in 16 (41%) patients of first group and in 54 (76.1%) patients of the second group, with the GSRS score 1.33±0.31 and 3.04±0.32 accordingly in those patients. Average total GSRS score was significantly higher in patients with abdominal angina - 13.51±0.84 points versus 9.94±1.13 in patients without signs of abdominal angina on Doppler sonography. Conclusion. The use of GSRS questionnaire allows to detect syndromes characteristic for abdominal angina.


2021 ◽  
Vol 12 ◽  
Author(s):  
Henrik Giese ◽  
Jennifer Antritter ◽  
Andreas Unterberg ◽  
Christopher Beynon

Objective: An increased interest in the surgical procedures of decompressive craniectomy (DC) and subsequent cranioplasty (CP) has emerged during the last decades with specific focus on mortality and complication rates. The aim of the present study was to evaluate long-term neurological and cosmetic outcomes as well as Quality of Life (QoL) after CP surgery.Methods: We retrospectively reviewed the medical records of CP patients treated at our institution between 2004 and 2014 and performed a follow-up examination, with evaluation of neurological outcome using the modified Rankin Scale (mRS) and the Glasgow outcome scale (GOS), QoL (SF-36 and EQ-5D-3L). Furthermore, the cosmetic results after CP were analyzed.Results: A total of 202 CP-patients were included in the present study. The main indications for DC and subsequent CP were space-occupying cerebral ischemia (32%), traumatic brain injury (TBI, 26%), intracerebral or subarachnoid hemorrhage (32%) and infection (10%). During a mean follow-up period of 91.9 months 46/42.6% of patients had a favorable neurological outcome (mRS ≤ 3/GOS ≥ 4). Patients with ischemia had a significant worse outcome (mRS 4.3 ± 1.5) compared with patients after TBI (3.1 ± 2.3) and infectious diseases requiring CP (2.4 ± 2.3). The QoL analysis showed that &lt;1/3rd of patients (31.2%) had a good QoL (SF-36) with a mean EQ-5D-VAS of 59 ± 26. Statistical analysis confirmed a significant worse QoL of ischemia patients compared to other groups whereas multivariate regression analysis showed no other factors which may had an impact on the QoL. The majority (86.5%) of patients were satisfied with the cosmetic result after CP and regression analysis showed no significant factors associated with unfavorable outcomes.Conclusion: Long-term outcome and QoL after CP were significantly influenced by the medical condition requiring DC. Early detection and evaluation of QoL after CP may improve the patient's outcome due to an immediate initiation of targeted therapies (e.g., occupational- or physiotherapy).


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