scholarly journals POS0853 DECREASE IN ANTI-TOPOISOMERASE-1 ANTIBODY TITER IN PATIENTS WITH SYSTEMIC SCLEROSIS DURING LONG-TERM RITUXIMAB THERAPY

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 681.1-681
Author(s):  
L. Garzanova ◽  
L. P. Ananyeva ◽  
O. Koneva ◽  
O. Desinova ◽  
O. Ovsyannikova ◽  
...  

Background:Rituximab (RTX) is a new option in the treatment of systemic sclerosis (SSc) [1]. There is not enough data on changes in the level of autoantibodies and their clinical significance during RTM therapy. There are only a few reports on the higher efficiency of RTX in patients (pts) with SSc positive for anti-topoisomerase-1 antibodies (a-Topo-1), therefore the study of this issue might be interested.Objectives:To compare clinical parameters and B-lymphocytes (B-lymph) level in SSc pts depending on the presence or absence of a-Topo-1 during RTX therapy with prospective long-term follow-up.Methods:This study included 88 pts with SSc. The mean follow-up period was 26,3±10,7 months. The mean age was 47years (17-71), female-73 pts (83%), the diffuse cutaneous subset of the disease had 50 pts (57%). Symptoms of the interstitial lung disease (ILD) were observed in 70 pts (80%). The mean disease duration was 5,9±4,8 years. The cumulative mean dose of RTX was 2,9±1,1 grams. All patients received prednisone at a dose of 11,7±4,4 mg, immunosuppressants received 42% of them. There were 63 pts positive for a-Topo-1 and 25 pts - negative. The pts of the compared groups did not differ in the main demographic and clinical parameters, excepting lung involvement. In a-Topo-1 positive group 55 (87%) pts had ILD and only 15 (60%) – in a-Topo-1-negative group (p=0,02). The results at baseline and at the end of the follow up are presented in the form of mean values and changes in parameters (delta).Results:Considering the entire cohort, an improvement of almost all outcome parameters was found. When a-Topo-1 positive and a-Topo-1-negative pts were analyzed separately, we observed a significantly higher decrease in the activity score, depletion of B-lymph, an increase in forced vital capacity (FVC) and diffusion capacity for carbon monoxide (DLCO) in a-Topo-1 positive group of pts (table 1).Table 1.Changes of the main outcome parameters depending on the presence of a-Topo-1 on RTX therapy.Parametersa-Topo-1positive ptsa-Topo-1negative ptsPDelta Activity score (EScSG-AI)1,790,90,001Delta Rodnan skin score (mRSS)4,95,2NSDelta B-lymphocytes (absolute count)0,2120,1930,001Delta FVC*, %8,646,460,001Delta DLCO**, %2,860,0320,001*FVC - forced vital capacity % predicted, **DLCO - diffusion capacity for carbon monoxide % predictedThe a-Тopo-1 level decreased from 174,2±50,1 to 148,1±66,1 units/ml (p=0,0009). In this group, a-Тopo-1 became negative in 5 pts (7,9%). The disappearance of a-Topo-1 positivity was accompanied by a more pronounced decrease in mRSS (delta mRSS=7,4) and a higher depletion of B-lymph. There was a higher cumulative dose of RTX (4±1,4grams) in this 5 pts compared with the pts who sustained a-Topo-1 positivity. There was a moderate negative statistically significant correlation between the a-Topo-1 and the total dose of RTX (r=-0,298, p=0,017). A moderate negative statistically significant correlation was found between the a-Topo-1 and FVC (r=-0,322, p=0,009).Conclusion:In our study, the a-Topo-1 level significantly decreased during RTX therapy in Russian pts. The decrease in a-Topo-1 titers correlated with the total dose of RTX and was accompanied by a decrease in mRSS, disease activity index and an increase in FVC and DLCO. A higher efficacy of RTX in the a-Topo-1 positive group with prevalence of ILD was revealed, therefore a-Topo-1 positivity could be considered as a predictor of a better response to RTX therapy.References:[1]Jordan S, et al. Effects and safety of rituximab in systemic sclerosis: an analysis from the European Scleroderma Trial and Research (EUSTAR) group. Ann Rheum Dis.2015;74:1188–94.Doi:10.1136/annrheumdis-2013-204522.[2]Ebata S, Yoshizaki A, et.al. Rituximab therapy is more effective than cyclophosphamide therapy for Japanese patients with anti-topoisomerase I-positive systemic sclerosis-associated interstitial lung disease. J Dermatol.2019.Nov;46(11):1006-1013.doi:10.1111/1346-8138.15079.Disclosure of Interests:None declared

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 404.2-404
Author(s):  
L. Garzanova ◽  
L. P. Ananyeva ◽  
O. Koneva ◽  
O. Desinova ◽  
O. Ovsyannikova ◽  
...  

Background:Anti-B-cell therapy is seen as a promising therapeutic option for systemic sclerosis (SSc). The study of antinuclear antibody levels during treatment with rituximab (RTX) in patients (pts) with SSc could have theoretical and practical interest.Objectives:To assess the changes in ANA, anti-topoisomerase-1 (Scl-70) levels and B-lymphocytes (B-lymph) count during RTX therapy during prospective observation.Methods:This prospective study included 88 pts with SSc, 83% of them had interstitial lung disease and 75% had positive Scl-70 autoantibody. The mean age was 47 yrs (17-71), female-73 pts (83%), the diffuse cutaneous subset of the disease had 50 pts (57%). The mean disease duration was 5,9±4,8 yrs. The mean follow-up period was 27 months (12-42). The cumulative mean dose of RTX was 2,9±1,1grams. All patients received prednisolone at a dose of 11,7±4,4 mg, immunosuppressants received 42% of them. Patients were divided into groups depending on the duration of the disease: group 1 (n=33) - up to 3 yrs, group 2 (n=25) - from 3 to 6 yrs, group 3 (n=30) - more than 6 years (6-18yrs). The results are presented in the form of mean values, median, upper and lower quartiles.Results:Parallel to clinical improvement in most patients (96%) we found positive changes in many parameters at the end of the study compared to the baseline. The Rodnan skin score decreased from 11,21±9,33 to 6,19±4,74 (p<0,001). The disease activity index (EScSG-AI) decreased from 2,9±1,74 to 1,36±1,15 (p<0,001). Forced vital capacity, % predicted, increased from 76,35±19,65 to 84,37±21,04 (p<0,001). Diffusing capacity for carbon monoxide, % predicted, increased from 45,56±17,72 to 47,62±16,96 (p<0,019). The dose of prednisolone decreased from 11,7±4,4 to 9,2±3,2 mg (p<0,001). The absolute number of B-lymph decreased from 0,224±0,19 to 0,0175±0,058 (p<0,001). The pts of the group 1 showed the highest values of B-lymph at baseline and level of B-lymph decreased from 0,326±0,22 to 0,008±0,01 (Δ 0,318) at the end of the study. In group 2 depletion was less pronounced (from 0,197±0,14 to 0,026±0,07 (Δ 0,171) and the lowest depletion was observed in group 3 (from 0,151±0,16 to 0,019±0,07 (Δ 0,131), p<0,001 for all groups. An initially positive ANA was found in 92% of pts (range 1/320-1/1280). During observation, the number of pts with high (1/640-1/1280) ANA titers decreased from 70 to 41 (p<0,001), and the average level of ANA decreased by 30-40% in all groups. At baseline 63 pts (75%), had positive Scl-70 with equal levels in all groups. At the end of the study level of Scl-70 decreased from 125,02±89,12 to 108,6±86,89 units/ml (p<0,007). A negative correlation was found between the duration of the disease and ANA (r = -0,54; p<0,003) and Scl-70 (r = -0,44; p<0,017).Conclusion:In our study a clinical improvement was shown in most pts at the long-term complex therapy, including RTM. We found a significant decrease in the absolute number of B-lymph, as well as decrease of ANA and Scl-70 levels. Initially pts with a short duration of the disease had a higher level of B-lymph and in these pts depletion was more pronounced, compared to those with a longer duration of the disease. However, the level of Scl-70 and ANA decreased both to those who started RTX therapy at an early stage of the disease (<3yrs) and to those who had a long disease duration.Disclosure of Interests:None declared


2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 164-164
Author(s):  
N. Cristina ◽  
L. Groseanu ◽  
F. Berghea ◽  
A. Balanescu ◽  
V. Bojinca ◽  
...  

Background:Two pivotal studies, RAPIDS-1 and RAPIDS-2 revealed that bosentan reduces the development of new digital ulcers (DUs) in patients with systemic sclerosis (SSc). However data regarding the long-term use of this dual endothelin antagonist receptor in the treatment of DUs is scarce.Objectives:The aim of the present study was to evaluate long term efficacy and safety profile of bosentan in patients with DUs related to SSc.Methods:A prospective observational case-control study, conducted between 2014 and 2020 enrolled 65 SSc patients with ≥1 active DUs at baseline, who received bosentan therapy. Demographic and clinical features, including DUs incidence and patients subjective perception of DU pain and/or Raynaud’s Phenomenon, were collected. Nailfold videocapillaroscopy was performed in all patients.Results:The study included 51 females and 14 males, with a mean age of 52.6 years, 30 with diffuse subset, most of them with late scleroderma pattern (46/65). Number of DUs at baseline was 4.55 (±2.8), median duration of treatment was 25.95 (±19.4) months. Microangiopathy evolution score (MES) was 5.1 (2.19), visual analog scale (VAS) for DU was 77.9, VAS for Raynaud was 73.4.Patients receiving bosentan had clinically significant reduction in the mean number of DU (p<0.001). The effect was most powerful for the first 6 months of treatment, but the improvement was sustained until 24 months’ follow-up, when the mean DU number reached a plateau that was kept until end of study. 6 month and 24 month evaluations also revealed significant decrease in the VAS for DU (p<0.05) and in the VAS for Raynaud (p<0.01). Statistically significant difference was noted between bosentan-treated and the control group with respect to the decrease in the mean number of digital ulcers. (p=0.005).There was a clear trend towards an improvement in MES score, between baseline and the next follow-up assessments (p=0.003). The difference was statistically significant when compared to control group, but only for the first 18 months of treatment (p<0.001).14 patients (28.75%) discontinued bosentan therapy for administrative reasons. The median time among patients who interrupted the treatment was 6.9 months. An accelerated development of new DU was described 6 months after (p=0.02). Following recommencement of bosentan, the mean number of DU has rapidly decreased (p=0.008). There was no significant difference between patients who temporarily discontinued bosentan for 6 or 12 months.Bosentan was stopped due to lack of efficacy in 2 cases and due to side effects in 7 cases: 4 elevated liver enzymes, 1 severe trombocytopenia, 1 dyspneea agravation and low blood pressure.Conclusion:The present data suggest that treatment with endothelin receptor antagonist bosentan was associated with a significant reduction in the mean number of DU in patients with SSc. The beneficial effect of bosentan persisted throughout the study but was most evident in the first 6 months of treatment. Statistical analysis showed a significant improvement of the microangiopathy evolution score from baseline to end of therapy. 14 patients had a high relapse rate due to potential rebound effect, 6 months after bosentan withdrawal.The drug was reintroduced succesfully for 10 (70%) patients with a significant decrease in the number of DU.References:[1]UK Scleroderma Study Group: digital vasculopathy in systemic sclerosis, Rheumatology, Volume 54, Issue 11, November 2015, Pages 2015[2]Groseanu L, Berghea F, Bojinca V, et al AB0779 Long term follow-up of a systemic sclerosis group treated with bosentan, Annals of the Rheumatic Diseases 2018;77:1523-1524.Disclosure of Interests:None declared


2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 1239.3-1240
Author(s):  
L. Garzanova ◽  
L. P. Ananyeva ◽  
O. Koneva ◽  
O. Desinova ◽  
O. Ovsyannikova ◽  
...  

Background:Rituximab (RTX) has been used for the treatment of systemic sclerosis (SSc) for a long time, but data on tolerance and long-term adverse events (AE) are insufficient.Objectives:To assess the tolerability and safety of RTX in the patients (pts) with SSc in long-term prospective follow-up.Methods:Data on the safety and tolerability of RTX were evaluated in 149 SSc pts who received at least one RTX infusion in a long-term open-label prospective observational study. The mean age was 48±13,5 years (17-74), women - 122 (82%), diffuse cutaneous subset of the disease had 52%, limited-37% and overlap-11%. The mean disease duration was 6,4±5,8 years (0,5-30). The observation period was 12 years. All pts received prednisolone at a dose of 11,7±4,8mg/day, and 73 patients (49%) received immunosuppressants at inclusion. The indications for the appointment of RTX were ineffectiveness or impossibility of standard therapy and a severe course of the disease with high activity and unfavorable prognosis factors. AE were assessed and recorded by a physician at a hospital immediately after the infusion of RTX, then by patient reported outcome during the observation period. Severe AE were defined as those that required hospitalization for more than 24 hours, exacerbation of the disease requiring therapy, malignancies, life-threatening situations. All causes of death were considered, regardless of treatment.Results:The mean follow-up period after the first infusion of RTX was 5,6±2,6 years [834,4 patient-years (PY)]. Pts received a mean of 3,4 courses of RTX (1–10). The cumulative mean dose of RTX was 3,2±2,4 gr (0,5-11). AE were reported in 77 patients (52%), the overall frequency of AE was 9,3/100 PY (95% Confidence Interval (CI) 8-11). The highest frequency of all AE was observed in the first 2-6 months after the first infusion of RTX, however these were mainly mild AE (71%). There was a decrease of AE in the follow-up period (3,4/100 PY, 95% CI 2,4-4,9 – at the period from 3 to 10 course of RTX). The overall incidence of serious AE was 2,22/100 PY (95% CI 1,4–3,5). The specter of serious AE included: pneumonia in 7 pts, infusion reactions in 5, as well as in one case: cerebral ischemia, acute pancreatitis, allergic pneumonitis, lymphoma of pharynx, purulent arthritis, lower limb vein thrombosis, pulmonary embolism of small arteries. The most frequent AE were infections (n=53), with no serious opportunistic infections reported. The overall incidence of all infections was 6,4/100 PY (95% CI 4,9-8,3), serious infections – 1,32/100 PY (95% CI 0,7-2,4). The level of immunoglobulin G during follow-up period decreased from 12,9±4,9 to 10,1±3,4g/l (p=0,0001), but remained within normal limits. Infusion reactions occurred in 15 pts (1,8/100 PY, 95% CI 1-3). Other AE were observed in 9 pts (6%) (1,1/100 PY, 95% CI 0,53-2,12). Sixteen deaths were recorded – 10,7% or 1,91/100 PY, 95% CI 1,2-3,1. In most cases, pts died from the progression of the major organ failure. The causes of death were: progression of the interstitial lung disease (ILD) in 4 pts, heart failure associated with SSc cardiomyopathy (2), renal crisis (4), pulmonary arterial hypertension and ILD (2), pneumonia (2), sepsis after tooth extraction (1), acute pulmonary embolism (1).Conclusion:In our study, we considered the overall safety profile of RTX in SSc as favorable. It was similar to the AE profile in other autoimmune diseases treated with RTX. With an increase of the cumulative dose of RTX, there was no increase in AE. RTX could be considered as a relatively safe drug for the complex therapy of SSc when standard therapy is ineffective or impossible.Disclosure of Interests:None declared


2019 ◽  
Vol 24 (5) ◽  
pp. 549-557
Author(s):  
Malia McAvoy ◽  
Heather J. McCrea ◽  
Vamsidhar Chavakula ◽  
Hoon Choi ◽  
Wenya Linda Bi ◽  
...  

OBJECTIVEFew studies describe long-term functional outcomes of pediatric patients who have undergone lumbar microdiscectomy (LMD) because of the rarity of pediatric disc herniation and the short follow-up periods. The authors analyzed risk factors, clinical presentation, complications, and functional outcomes of a single-institution series of LMD patients over a 19-year period.METHODSA retrospective case series was conducted of pediatric LMD patients at a large pediatric academic hospital from 1998 to 2017. The authors examined premorbid risk factors, clinical presentation, physical examination findings, type and duration of conservative management, indications for surgical intervention, complications, and postoperative outcomes.RESULTSOver the 19-year study period, 199 patients underwent LMD at the authors’ institution. The mean age at presentation was 16.0 years (range 12–18 years), and 55.8% were female. Of these patients, 70.9% participated in competitive sports, and among those who did not play sports, 65.0% had a body mass index greater than 25 kg/m2. Prior to surgery, conservative management had failed in 98.0% of the patients. Only 3 patients (1.5%) presented with cauda equina syndrome requiring emergent microdiscectomy. Complications included 4 cases of postoperative CSF leak (2.0%), 1 case of a noted intraoperative CSF leak, and 3 cases of wound infection (1.5%). At the first postoperative follow-up appointment, minimal or no pain was reported by 93.3% of patients. The mean time to return to sports was 9.8 weeks. During a mean follow-up duration of 8.2 years, 72.9% of patients did not present again after routine postoperative appointments. The total risk of reoperation was a rate of 7.5% (3.5% of patients underwent reoperation for the same level; 4.5% underwent adjacent-level decompression, and one patient [0.5%] ultimately underwent a fusion).CONCLUSIONSMicrodiscectomy is a safe and effective treatment for long-term relief of pain and return to daily activities among pediatric patients with symptomatic lumbar disc disease in whom conservative management has failed.


2020 ◽  
Vol 133 (3) ◽  
pp. 830-838 ◽  
Author(s):  
Andrea Franzini ◽  
Giuseppe Messina ◽  
Vincenzo Levi ◽  
Antonio D’Ammando ◽  
Roberto Cordella ◽  
...  

OBJECTIVECentral poststroke neuropathic pain is a debilitating syndrome that is often resistant to medical therapies. Surgical measures include motor cortex stimulation and deep brain stimulation (DBS), which have been used to relieve pain. The aim of this study was to retrospectively assess the safety and long-term efficacy of DBS of the posterior limb of the internal capsule for relieving central poststroke neuropathic pain and associated spasticity affecting the lower limb.METHODSClinical and surgical data were retrospectively collected and analyzed in all patients who had undergone DBS of the posterior limb of the internal capsule to address central poststroke neuropathic pain refractory to conservative measures. In addition, long-term pain intensity and level of satisfaction gained from stimulation were assessed. Pain was evaluated using the visual analog scale (VAS). Information on gait improvement was obtained from medical records, neurological examination, and interview.RESULTSFour patients have undergone the procedure since 2001. No mortality or morbidity related to the surgery was recorded. In three patients, stimulation of the posterior limb of the internal capsule resulted in long-term pain relief; in a fourth patient, the procedure failed to produce any long-lasting positive effect. Two patients obtained a reduction in spasticity and improved motor capability. Before surgery, the mean VAS score was 9 (range 8–10). In the immediate postoperative period and within 1 week after the DBS system had been turned on, the mean VAS score was significantly lower at a mean of 3 (range 0–6). After a mean follow-up of 5.88 years, the mean VAS score was still reduced at 5.5 (range 3–8). The mean percentage of long-term pain reduction was 38.13%.CONCLUSIONSThis series suggests that stimulation of the posterior limb of the internal capsule is safe and effective in treating patients with chronic neuropathic pain affecting the lower limb. The procedure may be a more targeted treatment method than motor cortex stimulation or other neuromodulation techniques in the subset of patients whose pain and spasticity are referred to the lower limbs.


1996 ◽  
Vol 3 (4) ◽  
pp. 369-379 ◽  
Author(s):  
Michel Henry ◽  
Max Amor ◽  
Rafael Beyar ◽  
Isabelle Henry ◽  
Jean-Marc Porte ◽  
...  

Purpose: To evaluate a new self-expanding nitinol coil stent in stenotic or occluded peripheral arteries. Methods: Seventy-three symptomatic patients (58 men; mean age 67 years) were treated with nitinol stents for lesions in the iliac artery (9 stenoses); superficial femoral artery (SFA) (39 stenoses, 6 occlusions); popliteal artery and tibioperoneal trunk (9 stenoses, 7 occlusions); and 3 bypass grafts. Mean diameter stenosis was 84.4% ± 9.9% (range 75% to 100%), and mean lesion length was 45 ± 23 mm (range 20 to 120 mm). Results: Eighty-eight 40-mm-long stents with diameters between 5 and 8 mm were implanted percutaneously for suboptimal dilation (n = 45); dissection (n = 21); and restenosis (n = 7). All stents but one were implanted successfully; the malpositioned stent was removed, and another stent was successfully deployed. There were 3 (4.1%) failures due to thrombosis at 24 hours. During the mean 16-month follow-up (range to 44 months), 4 restenoses (3 femoral, 1 popliteal) have occurred; 2 were treated with repeat dilation and 2 underwent bypass. Primary and secondary patency rates at 18 months were 87% and 90%, respectively, for all lesions (iliac: 100% for both; femoral: 85% and 88%; popliteal: 87% and 100%). Conclusions: This new nitinol stent seems to be safe and effective with favorable long-term results, even in distal SFA lesions and popliteal arteries. Its flexibility and resistance to external compression allow its placement in tortuous arteries and near joints.


2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Michał Waszczykowski ◽  
Bożena Dziankowska-Bartkowiak ◽  
Michał Podgórski ◽  
Jarosław Fabiś ◽  
Arleta Waszczykowska

AbstractThe aim of this study was to estimate the long-term results of complex and supervised rehabilitation of the hands in systemic sclerosis (SSc) patients. Fifty-one patients were enrolled in this study: 27 patients (study group) were treated with a 4-week complex, supervised rehabilitation protocol. The control group of 24 patients was prescribed a home exercise program alone. Both groups were evaluated at baseline and after 1-, 3-, 6-, and 12-months of follow-up with the Disability of the Arm, Shoulder and Hand Questionnaire (DAHS) as the primary outcome, pain (VAS—visual analog scale), Cochin Hand Function Scale (CHFS), Health Assessment Questionnaire Disability Index (HAQ-DI), Scleroderma-HAQ (SHAQ), range of motion (d-FTP—delta finger to palm, Kapandji finger opposition test) and hand grip and pinch as the secondary outcomes. Only the study group showed significant improvements in the DASH, VAS, CHFS and SHAQ after 1, 3 and 6 months of follow-up (P = 0.0001). Additionally, moderate correlations between the DASH, CHFS and SHAQ (R = 0.7203; R = 0.6788; P = 0.0001) were found. Complex, supervised rehabilitation improves hand and overall function in SSc patients up to 6 months after the treatment but not in the long term. The regular repetition of this rehabilitation program should be recommended every 3–6 months to maintain better hand and overall function.


Author(s):  
Quinten G. H. Rikken ◽  
Jari Dahmen ◽  
Sjoerd A. S. Stufkens ◽  
Gino M. M. J. Kerkhoffs

Abstract Purpose The purpose of the present study was to evaluate the clinical and radiological outcomes of arthroscopic bone marrow stimulation (BMS) for the treatment of osteochondral lesions of the talus (OLTs) at long-term follow-up. Methods A literature search was conducted from the earliest record until March 2021 to identify studies published using the PubMed, EMBASE (Ovid), and Cochrane Library databases. Clinical studies reporting on arthroscopic BMS for OLTs at a minimum of 8-year follow-up were included. The review was performed according to the PRISMA guidelines. Two authors independently conducted the article selection and conducted the quality assessment using the Methodological index for Non-randomized Studies (MINORS). The primary outcome was defined as clinical outcomes consisting of pain scores and patient-reported outcome measures. Secondary outcomes concerned the return to sport rate, reoperation rate, complication rate, and the rate of progression of degenerative changes within the tibiotalar joint as a measure of ankle osteoarthritis. Associated 95% confidence intervals (95% CI) were calculated based on the primary and secondary outcome measures. Results Six studies with a total of 323 ankles (310 patients) were included at a mean pooled follow-up of 13.0 (9.5–13.9) years. The mean MINORS score of the included studies was 7.7 out of 16 points (range 6–9), indicating a low to moderate quality. The mean postoperative pooled American Orthopaedic Foot and Ankle Society (AOFAS) score was 83.8 (95% CI 83.6–84.1). 78% (95% CI 69.5–86.8) participated in sports (at any level) at final follow-up. Return to preinjury level of sports was not reported. Reoperations were performed in 6.9% (95% CI 4.1–9.7) of ankles and complications related to the BMS procedure were observed in 2% (95% CI 0.4–3.0) of ankles. Progression of degenerative changes was observed in 28% (95% CI 22.3–33.2) of ankles. Conclusion Long-term clinical outcomes following arthroscopic BMS can be considered satisfactory even though one in three patients show progression of degenerative changes from a radiological perspective. These findings indicate that OLTs treated with BMS may be at risk of progressing towards end-stage ankle osteoarthritis over time in light of the incremental cartilage damage cascade. The findings of this study can aid clinicians and patients with the shared decision-making process when considering the long-term outcomes of BMS. Level of evidence Level IV.


2021 ◽  
pp. 1-11
Author(s):  
Hamid Reza Niknejad ◽  
Melissa Frederickx ◽  
Emiel Salaets ◽  
Jurgen Lemiere ◽  
Lieven Lagae ◽  
...  

<b><i>Introduction:</i></b> Tectal plate gliomas (TPG) constitute a distinct entity of benign tumors of the brain stem which show an indolent clinical course. Adequate treatment of secondary hydrocephalus is undoubtedly a major factor in the outcome. However, little is known about to what degree the tumor itself determines the long-term outcome of these patients. <b><i>Methods:</i></b> We retrospectively analyzed and compared the clinical and radiological data of 16 pediatric TPG patients with data of 12 pediatric idiopathic aqueductal stenosis (IAS) patients treated in our center from 1988 to 2018. For both groups, we assessed the long-term outcome in terms of hydrocephalus management, and for the TPG group, we assessed tumor growth during follow-up. In a separate prospective part of the study, we performed a neuropsychological evaluation in a subgroup of patients using a standardized testing battery, covering intelligence, learning, memory, executive functions, and an inventory on depression. <b><i>Results:</i></b> In the TPG group, the mean clinical and radiological follow-up was 84 and 70 months, respectively. On average, the maximum diameter of the tumor increased by 11% (<i>p</i> = 0.031) and the estimated tumor volume with 35% (<i>p</i> = 0.026) on radiological follow-up. The fronto-occipital horn ratio (FOHR) decreased by 23% on average after treatment. In the IAS group, the mean clinical and radiological follow-up was 117 and 85 months, respectively. In this group, the FOHR decreased by 21% on average. Neurocognitive testing revealed significant higher scores in the TPG group on global intelligence (TPG = 109, IAS = 85.5, <i>U</i> = 3, <i>p</i> &#x3c; 0.01, <i>z</i> = −2.71), performance (TPG= 100, IAS = 85, <i>U</i> = 7, <i>p</i> = 0.03, <i>z</i> = −2.2), and verbal intelligence (TPG = 122, IAS = 91.5, <i>U</i> = 2, <i>p</i> &#x3c; 0.00, <i>z</i> = −2.87) as well as working memory (TPG = 109.5, IAS = 77, <i>U</i> = 0.5, <i>p</i> = 0.01, <i>z</i> = −2.46). <b><i>Conclusion:</i></b> Our results suggest that the long-term outcome in TPG patients is acceptable and that cognition is substantially better preserved than in patients with IAS. This puts the idea of a significant contribution of the tumoral mass to disease outcome on the long term in question. Adequate and prompt management of hydrocephalus is the most important factor in long-term cognitive outcome.


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