Which Strategy of Immunoablative Therapy with Autologous Stem Cell Transplantation (ASCT) Is Preferable in Multiple Sclerosis (MS) Patients?.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 5093-5093
Author(s):  
Yury L. Shevchenko ◽  
Andrei A. Novik ◽  
Alexey N. Kuznetcov ◽  
Boris A. Afanasiev ◽  
Igor A. Lisukov ◽  
...  

Abstract During the last decade immunoablative therapy with ASCT (IT+ASCT) has been used with increasing frequency as a therapeutic option for MS patients. Among a number of unanswered questions is the timing of IT+ASCT. We have identified 3 strategies of IT+ASCT depending on the stage in disease process: early, conventional and salvage/late. The goal of our research was to study clinical and patient-reported outcomes in MS patients after early, conventional and salvage/late transplantation. 54 patients with MS (secondary progressive - 26 patients, primary progressive -10, progressive-relapsing - 1, and relapsing-remitting - 17) from 6 medical centers were included in this study (mean age - 32.0, range: 17–51; male/female - 21/33). 13 patients underwent early, 37 patients - conventional, and 4 patients - salvage/late transplantation. Median EDSS at base-line was 6.0 (range 1.5 – 8.0). The median follow-up duration was 18 months (range 6 – 84 months). Neurological and quality of life (QoL) evaluation was performed at baseline, at discharge, at 3, 6, 9, 12 months, and every 6 months thereafter following IT+ASCT. MRI examinations were conducted at baseline, at 6, 12 months, and at the end of follow-up. FACT-BMT and FAMS were used for QoL evaluation. Notably, no transplant-related deaths or unpredictable severe adverse events were observed. All 42 patients (6 - early transplantation; 32 - conventional transplantation; 4 - salvage/late transplantation) with the follow-up of longer than 9 months experienced clinical stabilization or improvement. More than half of them (27 patients) improved: 8 patients showed significant improvement in EDSS, 9 patients improved by 1.0 point, and 10 - by 0.5 points on EDSS. Notably, all of the patients after early transplantation (mean age - 28.0) improved at least by 0.5 points on EDSS. 15 patients achieved stabilization. 2 patients deteriorated to a worse score after 18 months of stabilization; 2 others progressed after 12 and 30 months of improvement, respectively. All of the patients with clinical stabilization and improvement had negative MRI scans. Out of 24 patients included in QoL analysis 22 exhibited improved QoL 6 months post-transplantation. In conclusion, IT+ASCT appears to be an effective treatment for MS both in terms of clinical and patient-reported outcomes. The data obtained point to feasibility of early, conventional and salvage/late transplantation in MS patients. Our data support the hypothesis that transplantation is more effective in young patients at early stages of rapidly progressing disease. Further studies should be done to establish the best timing for transplantation and to validate IT+ASCT regimens in patients receiving early, conventional and salvage transplantation.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 3072-3072
Author(s):  
Yury L. Shevchenko ◽  
Andrei A. Novik ◽  
Alexey N. Kuznetcov ◽  
Boris V. Afanasiev ◽  
Igor A. Lisukov ◽  
...  

Abstract During the last decade HDCT+ASCT is more often used as a therapeutic option for MS patients. The major treatment outcomes for MS patients are disease-progressive free period and improvement of patient’s quality of life (QoL). We aimed to study treatment outcomes in MS patients after early, conventional and salvage HDCT+ASCT. Thirty two patients with MS (secondary progressive - 16 patients, primary progressive - 8, progressive-relapsing - 1, relapsing-remitting - 7) from 6 medical centers were included in this study (mean age - 32.0, range: 19–49; male/female - 12/20). Two patients underwent early HDCT +ASCT, 27 patients - conventional HDCT+ASCT and 3 patients - salvage HDCT+ASCT. Median EDSS at base-line was 6.0 (range 2.0 – 8.0). The median follow-up duration was 18 months (range 6 – 78 months). All of the patients had previously undergone conventional treatment. Neurological and QoL evaluation was provided at baseline, at discharge, 3, 6, 9, 12 months, and then every 6 months after HDCT+ASCT. MRI was conducted at baseline, at 6, 12 months, and at the end of follow-up. FACT-BMT and FAMS were used for QoL evaluation. QoL response was evaluated using Integral QoL index. All 27 patients with the follow-up longer than 1 year, included in the analysis, experienced a clinical stabilization or improvement. More than half of them improved: 6 patients showed significant improvement in EDSS (by more than 1.0 point), 4 patients improved by 1.0 point, and 5 patients - by 0.5 points on EDSS. Twelve patients achieved stabilization. Two patients deteriorated to a worse score after 18 months of stabilization; 2 other patients progressed after 12 and 30 months of improvement, respectively. All the patients with clinical stabilization and improvement exhibited negative MRI scans. Out of 21 patients included in the analysis of QoL response 19 exhibited improved QoL 6 months post-transplantation. At one year after HDCT+ASCT 1 patient exhibited excellent QoL response; 3 patients - good QoL response; 7 patients - moderate QoL response and 8 patients - minimal QoL response. At 2.5 years post-transplantation 3 more patients had excellent QoL response. Further QoL improvement was observed at longer follow-up. One of the patients who experienced progression after stabilization had no QoL response; another patient who progressed after stabilization had stable QoL during 6 months post-transplant and significantly worsened at 9 months. In conclusion, clinical response was observed in 100% of MS patients after early, conventional and salvages HDCT+ASCT. The majority of patients with clinical response had a good or moderate QoL response. Further studies should be done to investigate the clinical and patient-reported outcomes of HDCT+ASCT in MS patients to better define treatment success.


Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 5467-5467
Author(s):  
Yury L. Shevchenko ◽  
Andrei A. Novik ◽  
Boris V. Afanasiev ◽  
Igor A. Lisukov ◽  
Tatiana I. Ionova ◽  
...  

Abstract HDCT+ASCT is a new and promising therapy for MS patients. Among a number of unclear questions is the terms of conducting HDCT+ASCT. According to our concept there are 3 strategies of HDCT+ASCT depending on the terms of disease process: early, conventional and salvage. Another important consideration is that the major treatment outcomes for MS patients are disease-progressive free period and improvement of patient’s quality of life (QoL). With this in mind, evaluation of both clinical and patient-reported outcomes in MS patients after HDCT+ASCT is worthwhile. We aimed to study the clinical and QoL response in MS patients after early, conventional and salvage HDCT +ASCT. 17 patients with MS (secondary progressive − 8 patients, primary progressive − 6, progressive relapsing − 2, relapsing remitting − 1) were included in the study (mean age − 32.3, SD − 6.6; male/female − 4/13). Fifteen patients underwent conventional HDCT +ASCT; one patient (relapsing-remitting MS) - early HDCT+ASCT and one patient (primary progressive MS) - salvage HDCT+ASCT. Median EDSS at base-line was 6.0 (range 2.0 – 7.5). The median follow-up duration was 12 months (range 6 – 72 months). All of the patients had previously undergone conventional treatment. Neurological and QoL evaluation was provided at baseline, at discharge, 3, 6, 9, 12 months, and then every 6 months. MRI was conducted at baseline, at 6, 12 months, and at the end of follow-up. FACT-BMT and FAMS were used for QoL evaluation. QoL response was evaluated using Integral QoL index, which was calculated by the method of integral profiles. 14 (82.4%) out of 17 patients including 12 patients with conventional HDCT+ ASCT, one patient with early HDCT+ ASCT and one patient with salvage HDCT+ ASCT experienced a clinical stabilization or improvement. Three patients showed significant improvement in EDSS (by more than 1.0 point), 2 patients improved by 1.0, and 3 patients - by 0.5. Six cases remained stable. All of the patients with clinical stabilization and improvement exhibited negative MRI scans. One patient continuously worsened and died 3 years after the transplantation. Two other patients worsened by 0.5 points in their EDSS. All patients with clinical response exhibited improved QoL at 6 months post-transplant and they preserved improved QoL at the end of follow-up. At one year after HDCT+ASCT the patients exhibited a good or excellent QoL response. These levels of QoL response were preserved throughout the follow-up period. In conclusion, clinical response was observed in 82.4% MS patients after HDCT+ASCT. Notably, patients undergoing early, conventional and salvage HDCT+ASCT exhibited clinical response. All patients with clinical response had good or excellent QoL response. Thus, HDCT+ASCT appears to be an effective treatment for MS both in terms of clinical and patient-reported outcomes. The data obtained point to feasibility of early, conventional and salvage HDCT+ASCT in MS patients. Further studies should be done to investigate clinical and QoL response in MS patients receiving early, conventional and salvage HDCT+ASCT to better define treatment success.


2017 ◽  
Vol 27 (6) ◽  
pp. 546-550 ◽  
Author(s):  
Ishaan Swarup ◽  
Ryan Sutherland ◽  
Jayme C. Burket ◽  
Mark P. Figgie

Background Post-traumatic arthritis of the hip is a degenerative condition that commonly affects young patients. In this study, we evaluate long-term implant survival and patient-reported outcomes after primary total hip arthroplasty (THA) in patients aged 35 or younger with post-traumatic arthritis of the hip. Methods We conducted a retrospective study with follow-up. A chart review was performed to identify young patients with post-traumatic arthritis of the hip treated with primary THA. Follow-up surveys were conducted to determine implant survival and patient-reported outcomes. Implant survival was assessed using Kaplan-Meier survival analysis, and patient outcomes were determined using the hip disability and osteoarthritis outcome score (HOOS). Results We studied 42 patient s (44 THAs) with a mean time to follow-up of 14 years. The 10-year implant survival rate was 87% and 20-year implant survival rate was 41%. Implant survival did not differ based on patient age, gender, implant type, bearing surface, or use of cement for implant fixation (p>0.05). The mean HOOS scores at follow-up were 87 for pain, 85 for symptoms, 89 for ADLs, and 76 for sports. HOOS scores were significantly worse in patients that had undergone revision THA (p<0.05). Conclusions Young patients with post-traumatic arthritis of the hip have good long-term outcomes after THA. However, revision THA is predictive of worse long-term outcomes.


2017 ◽  
Vol 27 (3) ◽  
pp. 286-292 ◽  
Author(s):  
Ishaan Swarup ◽  
Marisa Shields ◽  
Erik N. Mayer ◽  
Chelsea J. Hendow ◽  
Jayme C. Burket ◽  
...  

Background Osteonecrosis of the hip is a clinical, radiographic, and pathologic entity that commonly affects young patients. This study evaluates long-term implant survival and patient-reported outcomes after primary total hip arthroplasty (THA) in patients with osteonecrosis aged 35 or younger. Methods A retrospective study with prospective follow-up was conducted at a major academic medical center. Chart review was performed to identify young THA patients with osteonecrosis, and follow-up surveys were conducted to determine implant survival and patient-reported outcomes. Kaplan-Meier survival analysis was performed to evaluate implant survival, and the hip disability and osteoarthritis outcome score (HOOS) was used to describe patient-reported outcomes. Results The study included 135 patients (204 THAs) with a mean time to follow-up of 14 years. In this group, 10-year implant survival was 86% and 20-year implant survival was 66%. Implant survival was longer in male patients (p = 0.02) and patients that were over the age of 25 at the time of surgery (p = 0.03). The mean HOOS scores at follow-up were 87 for pain, symptoms, and ADLs, and 77 for sports. All HOOS measures were lower in patients that underwent a revision THA, and HOOS-Pain and HOOS-Sport scores were lower in patients that were over the age of 25 at the time of surgery (p<0.05). Conclusions Young patients with osteonecrosis have good implant survival and long-term outcomes after THA. Patient factors and implant characteristics should be considered when predicting implant survival and outcomes after THA in young patients with osteonecrosis.


2020 ◽  
Author(s):  
Sissel Ravn ◽  
Henriette Vind Thaysen ◽  
Lene Seibaek ◽  
Victor Jilbert Verwaal ◽  
Lene Hjerrild Iversen

BACKGROUND Cancer survivors experience unmet needs during follow-up. Besides recurrence, a follow-up includes detection of late side effects, rehabilitation, palliation and individualized care. OBJECTIVE We aimed to describe the development and evaluate the feasibility of an intervention providing individualized cancer follow-up supported by electronic patient-reported outcomes (e-PRO). METHODS The study was carried out as an interventional study at a Surgical and a Gynecological Department offering complex cancer surgery and follow-up for advanced cancer. The e-PRO screened for a priori defined clinical important symptoms and needs providing individualized follow-up. We included following questionnaires in the e-PRO; the general European Organization for Research and Treatment of Cancer (EORTC) QLQ-C30 and the EORTC validated for colorectal and ovarian cancer patients. To support individualization, we included three prioritized issues of the patient’s preference in each e-PRO. The response-algorithm was aggregated based on the severity of the patient’s response. To ensure the sensitivity of the e-PRO, we performed semi-structured interviews with five patients. All clinicians (surgeons and gynecologists) performing the consultations reviewed the e-PRO. The evaluation was divided in two, 1)The feasibility was assessed by a)Patients’ response rate of the e-PRO, b)Number of follow-up visits documenting the use of e-PRO and c)Patients’ prioritized issues prior to the consultation(‘yes’ / ‘no’), and after the follow-up 2)Patients assessment of a)The need and purpose of the follow-up visit and b)the support provided during the follow-up visit. RESULTS In total, 187 patients were included in the study, of which 73%(n=136/187) patients responded to the e-PRO and were subjected to an individualized follow-up. The e-PRO was documented as applied in 79% of the follow-up visits. In total, 23% of the prioritized issues did not include a response. Stratified by time since surgery, significantly more patients did not fill out a prioritized issue had a follow-up >6 months since surgery. In total, 72 % follow-up visits were evaluated to be necessary in order to discuss the outcome of the CT scan, symptoms, and/or prioritized issues. Contrary, 19% of the follow-up visits were evaluated to be necessary only to discuss the result of the CT scan. A range from 19.3–56.3% of patients assessed the follow-up visit to provide support with respect to physical (42% of patients), mental (56%), sexual (19%) or dietary (27%) issues. Further, a range from 34–60% of the patients reported that they did not need support regarding physical (43% of patients), mental (34%), sexual (63%) or dietary (57%) issues. CONCLUSIONS An individualized follow-up based on e-PRO is feasible, and support most patients surgically treated for advanced cancer. However, results indicate that follow-up based on e-PRO may not be beneficial for all patients and circumstances. A thorough cost-benefit analysis may be warranted before implementation in routine clinic.


2021 ◽  
pp. 036354652110154
Author(s):  
Adam M. Johannsen ◽  
Justin W. Arner ◽  
Bryant P. Elrick ◽  
Philip-C. Nolte ◽  
Dylan R. Rakowski ◽  
...  

Background: Modern rotator cuff repair techniques demonstrate favorable early and midterm outcomes, but long-term results have yet to be reported. Purpose: To determine 10-year outcomes and survivorship after arthroscopic double-row transosseous-equivalent (TOE) rotator cuff repair. Study Design: Case series; Level of evidence 4. Methods: The primary TOE rotator cuff repair procedure was performed using either a knotted suture bridge or knotless tape bridge technique on a series of patients with 1 to 3 tendon full-thickness rotator cuff tears involving the supraspinatus. Only patients who were 10 years postsurgery were included. Patient-reported outcomes were collected pre- and postoperatively, including American Shoulder and Elbow Surgeons (ASES), 12-Item Short Form Health Survey (SF-12), Single Assessment Numeric Evaluation (SANE), shortened version of the Disabilities of the Arm, Shoulder and Hand (QuickDASH), and satisfaction. Kaplan-Meier survivorship analysis was performed. Failure was defined as progression to revision surgery. Results: A total of 91 shoulders (56 men, 31 women) were included between October 2005 and December 2009. Mean follow-up was 11.5 years (range, 10.0-14.1 years). Of 91 shoulders, 5 (5.5%) failed and required revision surgery. Patient-reported outcomes for patients who survived were known for 80% (69/86). Outcomes scores at final follow-up were as follows: ASES, 93.1 ± 10.8; SANE, 87.5 ± 14.2; QuickDASH, 11.1 ± 13.5; and SF-12 physical component summary (PCS), 49.2 ± 10.1. There were statistically significant declines in ASES, SANE, and SF-12 PCS from the 5-year to 10-year follow-up, but none of these changes met the minimally clinically important difference threshold. Median satisfaction at final follow-up was 10 (range, 3-10). From this cohort, Kaplan-Meier survivorship demonstrated a 94.4% survival rate at a minimum of 10 years. Conclusion: Arthroscopic TOE rotator cuff repair demonstrates high patient satisfaction and low revision rates at a mean follow-up of 11.5 years. This information may be directly utilized in surgical decision making and preoperative patient counseling regarding the longevity of modern double-row rotator cuff repair.


2020 ◽  
Vol 6 (1) ◽  
pp. e000965
Author(s):  
Natalie A Lowenstein ◽  
Peter J Ostergaard ◽  
Daniel B Haber ◽  
Kirsten D Garvey ◽  
Elizabeth G Matzkin

ObjectivesRisk factors for anterior shoulder dislocation include young age, contact activities and male sex. The influence of sex on patient-reported outcomes of arthroscopic Bankart repair (ABR) is unclear, with few studies reporting potential differences. This study’s purpose was to compare patient-reported outcomes of males and females following ABR.MethodsProspectively collected data was analysed for 281 patients (males: 206, females: 75) after ABR with preoperative, 1-year and 2-year follow-up responses. The Wilcoxon signed-rank and χ2 tests, preoperative, 1 year and 2 year follow-up results were examined to determine differences of scores in males versus females.ResultsNo statistically significant sex differences were observed in Simple Shoulder Test (SST), American Shoulder and Elbow Surgeons (ASES), Visual Analogue Scale (VAS) or Single Assessment Numerical Evaluation (SANE) Scores at 1-year or 2-year follow-up. Females had lower Veterans RAND 12-item health survey (VR-12) mental health subscores at 2-year follow-up (females: 52.3±9.0, males: 55.8±7.6, p=0.0016). Females were more likely to report that treatment had ‘exceeded expectations’ at 2-year follow-up regarding motion, strength, function and normal sports activities.ConclusionResults of study demonstrate that ABR has similar outcomes for both males and females. There were no statistically significant sex-related differences in SST, ASES, VAS or SANE scores following ABR. VR-12 mental health subscores showed a minimal difference at 2-year follow-up, with lower scores in females.Level of evidenceRetrospective cohort study; level II.


2020 ◽  
Vol 8 (12) ◽  
pp. 232596712096518
Author(s):  
Karen Brage ◽  
Birgit Juul-Kristensen ◽  
John Hjarbaek ◽  
Eleanor Boyle ◽  
Per Kjaer ◽  
...  

Background: Shoulder pain is common, with a lifetime prevalence of up to 67%. Evidence is conflicting in relation to imaging findings and pain in the shoulder. Sonoelastography can be used to estimate tissue stiffness and may be a clinically relevant technique for diagnosing and monitoring tendon healing. Purpose: To evaluate changes in supraspinatus tendon stiffness using strain elastography (SEL) and associations with changes in patient-reported outcomes, supraspinatus tendon thickness, and grade of tendinopathy after 12 weeks of unilateral shoulder exercises in patients with supraspinatus tendinopathy. Study Design: Controlled laboratory study. Methods: A total of 23 patients with unilateral clinical supraspinatus tendinopathy performed 12 weeks of “standard care” exercises. At baseline and follow-up, supraspinatus tendon stiffness was measured bilaterally using SEL and compared with tendinopathy grading on magnetic resonance imaging scans and tendon thickness measured using conventional ultrasound. Patient-reported outcome measures included physical function and symptoms from the Disabilities of the Arm, Shoulder and Hand questionnaire and pain rating (visual analog scale). Results: No significant changes in SEL within or between groups (asymptomatic vs symptomatic tendon) were seen. All patient-reported outcomes showed significant improvement from baseline to follow-up, but with no change in tendinopathy grading and tendon thickness. No significant differences in the proportion of patients changing above the minimal detectable change in SEL and PROM were seen, except for discomfort while sleeping. Conclusion: Despite no significant within-group or between-group changes in SEL, significant improvements were found in patient-reported outcomes. An acceptable agreement between patients changing above the minimal detectable change in SEL and patient-reported outcome measure was seen. Further studies should explore the use of SEL to detect changes after tendon repair and long-term training potentially in subgroups of different tendinopathy phases. Clinical Relevance: In the short term, structural changes in supraspinatus tendons could not be visualized using SEL, indicating that a longer time span should be expected in order to observe structural changes, which should be considered before return to sports. Subgrouping based on stage of tendinopathy may also be important in order to evaluate changes over time with SEL among patients with supraspinatus tendinopathy. Registration: NCT03425357 ( ClinicalTrials.gov identifier).


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