A Comparison of Functional and Radiographic Outcomes following Microfracture with Extracellular Matrix Augmentation versus Osteochondral Autograft Transplantation for the Treatment of Medium-Sized Osteochondral Lesions of the Talus

Objectives: The aim of this study is to directly compare clinical outcomes following the treatment of medium-sized osteochondral lesions of the talus (OLTs) using a microfracture technique augmented with Extracellular Matrix and Bone Marrow Aspirate Concentrate (MFX) versus OAT to determine which treatment is superior for medium-sized lesions. Methods: Patients treated for an OLT between 2015 and 2018 by a single surgeon, fellowship-trained in sports medicine and foot and ankle, were screened for this study. Retrospective chart review determined treatment, lesion size, lesion location, concurrent injuries, and demographic information. Patients at a minimum of 12 months follow-up, treated with MFX or OAT, and lesions sized 80-165mm2 were eligible for inclusion. All surgical repairs were augmented with an adjuvant mixture of micronized cartilage extracellular matrix and bone marrow aspirate concentrate (ECM-BMAC). Patient-reported functional outcomes were collected through our institution’s prospective Registry database. Patients treated prior to March 2016 were administered preoperative Foot and Ankle Outcome Score questionnaires. Those treated after this date were administered preoperative Physical Function, Pain Interference, Global Physical Health, Global Mental Health, Depression, and Pain Intensity Patient-Reported Outcome Information System (PROMIS) domains. Both FAOS and PROMIS were administered postoperatively. Postoperative MRIs were assessed using a modified magnetic resonance observation of cartilage repair tissue (MOCART) score. Student’s paired and two-group t-tests were used to evaluate for statistically significant pre-to-postoperative change and differences between procedure groups (p less than 0.05). Results: Twenty-seven patients treated with MFX (age range, 14-58) and twenty-three patients treated using OAT (age range, 22-64) were identified. All OAT patients received a single-plug transplantation. The final average lesion size ± standard deviation (SD) for patients treated with MFX was 115.44mm2± 22.51 (range, 156-80mm2) and 121.78mm2± 23.98 (range, 165-80mm2) for those treated using OAT (p=0.34). On average, functional outcome scores improved pre-to-postoperatively across all scales within both groups. Statistically significant improvements were detected in PROMIS Physical Function (Δ=8.32, p=0.01), Pain Interference (Δ=-7.15, p=0.02), Global Physical Health (Δ=5.87, p=0.03), and Pain Intensity (Δ=-7.06, p=0.05) domains for the MFX cohort. For the OAT patient group, significant pre-to-postoperative change was seen in the FAOS subcategories of Pain (Δ=28.70, p=0.03), Sports Activities (Δ=43.12, p<0.01), and Quality of Life (Δ=43.75, p=0.01); overall FAOS score (Δ=29.93, p=0.01); and PROMIS Physical Function (Δ=13.66, p=0.01), Pain Interference (Δ=-14.58, p<0.01), Global Physical Health (Δ=12.2, p=0.01), Depression (Δ=-4.13, p=0.02), and Pain Intensity (Δ=-16.56, p=0.02) domains. On average, with the exception of the postoperative Sports Activities subscale, postoperative FAOS and pre-to-postoperative change in FAOS were higher and greater in the OAT patient group. Similarly, on average, the OAT group had better PROMIS t-scores indicating higher function or less pain and greater pre-to-postoperative change in each PROMIS domain. The OAT cohort’s average postoperative Pain Interference t-score (± SD) of 43.09 (± 5.81) and Depression t-score of 40.06 (± 6.84) were significantly lower than their respective counterparts in the MFX cohort: 50.08 (± 9.47) for Pain Interference and 48.09 (± 7.86) in Depression. (Table 1) Finally, the mean overall MOCART score was 55.67 (± 24.11) within the MFX cohort, average follow-up 15.29 months, and 71 (± 15.60) within the OAT cohort average follow-up 15.8 months. This difference was also statistically significant (p=0.04). Conclusion: The OAT group had a higher MOCART score indicating the use of a single osteochondral autograft plug may result in better structural repair than microfracture abrasion chondroplasty augmented with a mixture of adjuvant ECM-BMAC. In addition, higher average FAOS scores, better average PROMIS t-scores, and greater pre-to-postoperative change in the OAT patient group indicate functional results may be better in this group as well. Specifically, significantly lower Pain Interference and Depression domains and significantly higher Global Mental Health scores indicate patients treated using OAT experience less pain and better psychological benefits postoperatively compared to patients treated using MFX. These results suggest filling the lesion with transplanted autograft bone and native, hyaline cartilage may perform better than and the biomechanically inferior fibrocartilage produced following microfracture even when augmented with adjuvant therapy. OAT may result in better overall clinical outcomes, specifically in a population of patients with medium sized lesions (range, 80 mm2 -165 mm2).

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Clinical Outcomes of the Polyvinyl Alcohol (PVA) Hydrogel Implant for Hallux Rigidus

Foot & Ankle International ◽

10.1177/1071100720932526 ◽

2020 ◽

Vol 41 (9) ◽

pp. 1056-1064

Author(s):

Stephanie K. Eble ◽

Oliver B. Hansen ◽

Bopha Chrea ◽

Taylor N. Cabe ◽

Jonathan Garfinkel ◽

...

Keyword(s):

Postoperative Pain ◽

Polyvinyl Alcohol ◽

Physical Health ◽

Pain Intensity ◽

Physical Function ◽

Hallux Rigidus ◽

Pain Interference ◽

Pain Scores ◽

Patient Reported ◽

Pva Hydrogel

Background: Hallux rigidus is a common arthritic condition that has been addressed surgically with a range of techniques, from an isolated cheilectomy to first metatarsophalangeal (MTP) joint fusion. Recently, hemiarthroplasty with polyvinyl alcohol (PVA) hydrogel implant has been used as an alternative treatment to relieve pain while preserving motion of the first MTP joint. We retrospectively reviewed patient-reported outcome scores and clinical outcomes for patients treated for hallux rigidus with PVA hydrogel implant at an academic, multisurgeon center. Methods: A total of 103 patients who underwent first MTP hemiarthroplasty with PVA hydrogel implant between January 2017 and October 2018 were retrospectively reviewed (average, 26.2 months). Eight surgeons were represented. Baseline Patient-Reported Outcomes Measurement Information System (PROMIS) scores for the Physical Function, Pain Interference, Pain Intensity, Global Physical Health, Global Mental Health, and Depression domains were collected prospectively and compared with PROMIS scores collected at a minimum of 1 year postoperatively (average, 13.9 months). Seventy-three patients had both preoperative and postoperative scores. Ten of these patients had undergone a prior procedure of the first MTP, and 52 underwent concurrent Moberg osteotomy at the time of PVA hydrogel implantation. Results: For patients with baseline and postoperative PROMIS scores, significant pre- to postoperative improvement was detected for the Physical Function, Pain Interference, Pain Intensity, and Global Physical Health domains ( P < .05). Patients who had undergone a prior procedure of the first MTP had significantly higher postoperative Pain Intensity scores compared with those who did not undergo a prior procedure. Patients undergoing concurrent Moberg osteotomy had significantly lower postoperative Pain Interference and Pain Intensity scores compared with those who did not undergo a Moberg. Two patients underwent revision procedures in the first 2 years postoperatively, one with revision hemiarthroplasty and one with conversion to arthrodesis. Conclusion: On average across our entire cohort, physical function and pain scores improved significantly pre- to postoperatively; however, postoperative pain scores were significantly higher for patients who had undergone a prior procedure of the first MTP and significantly lower for patients who underwent concurrent Moberg osteotomy. The implant displayed excellent survivorship in the first 2 years postoperatively, with only 2 revision procedures. Level of Evidence: Level III, comparative series.

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Association Between Baseline PROMIS Scores, Patient-Provider Communication Factors, and Musculoskeletal Health Literacy on Patient and Surgeon Expectations in Foot and Ankle Surgery

Foot & Ankle International ◽

10.1177/1071100720959017 ◽

2020 ◽

pp. 107110072095901

Author(s):

Aoife MacMahon ◽

Elizabeth A. Cody ◽

Kristin Caolo ◽

Jensen K. Henry ◽

Mark C. Drakos ◽

...

Keyword(s):

Health Literacy ◽

Pain Intensity ◽

Physical Function ◽

Mental Status ◽

Patient Perceptions ◽

Pain Interference ◽

Foot And Ankle ◽

Musculoskeletal Health ◽

Ankle Surgery ◽

Patient Reported

Background: Various factors may affect differences between patient and surgeon expectations. This study aimed to assess associations between patient-reported physical and mental status, patient-surgeon communication, and musculoskeletal health literacy with differences in patient and surgeon expectations of foot and ankle surgery. Methods: Two hundred two patients scheduled to undergo foot or ankle surgery at an academic hospital were enrolled. Preoperatively, patients and surgeons completed the Hospital for Special Surgery Foot & Ankle Surgery Expectations Survey. Patients also completed Patient-Reported Outcomes Measurement Information System (PROMIS) scores in Physical Function, Pain Interference, Pain Intensity, Depression, and Global Health. Patient-surgeon communication and musculoskeletal health literacy were assessed via the modified Patients’ Perceived Involvement in Care Scale (PICS) and Literacy in Musculoskeletal Problems (LiMP) questionnaire, respectively. Results: Greater differences in patient and surgeon overall expectations scores were associated with worse scores in Physical Function ( P = .003), Pain Interference ( P = .001), Pain Intensity ( P = .009), Global Physical Health ( P < .001), and Depression ( P = .009). A greater difference in the number of expectations between patients and surgeons was associated with all of the above ( P ≤ .003) and with worse Global Mental Health ( P = .003). Patient perceptions of higher surgeons’ partnership building were associated with a greater number of patient than surgeon expectations ( P = .017). There were no associations found between musculoskeletal health literacy and differences in expectations. Conclusion: Worse baseline patient physical and mental status and higher patient perceptions of provider partnership building were associated with higher patient than surgeon expectations. It may be beneficial for surgeons to set more realistic expectations with patients who have greater disability and in those whom they have stronger partnerships with. Further studies are warranted to understand how modifications in patient and surgeon interactions and patient health literacy affect agreement in expectations of foot and ankle surgery. Level of Evidence: Level II, prospective comparative series.

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ODI Cannot Account for All Variation in PROMIS Scores in Patients With Thoracolumbar Disorders

Global Spine Journal ◽

10.1177/2192568219851478 ◽

2019 ◽

Vol 10 (4) ◽

pp. 399-405

Author(s):

Peter G. Passias ◽

Samantha R. Horn ◽

Frank A. Segreto ◽

Cole A. Bortz ◽

Katherine E. Pierce ◽

...

Keyword(s):

Linear Regression ◽

Pain Intensity ◽

Physical Function ◽

Social Life ◽

Thoracolumbar Spine ◽

Pain Interference ◽

Pain Intensity Score ◽

Low Back ◽

Patient Reported ◽

The Relationship

Study Design: Retrospective review of single institution. Objective: To assess the relationship between Patient-Reported Outcomes Measurement Information System (PROMIS) and Oswestry Disability Index (ODI) scores in thoracolumbar patients. Methods: Included: Patients ≥18 years with a thoracolumbar spine condition (spinal stenosis, disc herniation, low back pain, disc degeneration, spondylolysis). Bivariate correlations assessed the linear relationships between ODI and PROMIS (Physical Function, Pain Intensity, and Pain Interference). Correlation cutoffs assessed patients with high and low correlation between ODI and PROMIS. Linear regression predicted the relationship of ODI to PROMIS. Results: A total of 206 patients (age 53.7 ± 16.6 years, 49.5% female) were included. ODI correlated with PROMIS Physical Function ( r = −0.763, P < .001), Pain Interference ( r = 0.800, P < .001), and Pain Intensity ( r = 0.706, P < .001). ODI strongly predicted PROMIS for Physical Function ( R2 = 0.58, P < .001), Pain Intensity ( R2 = 0.50, P < .001), and Pain Interference ( R2 = 0.64, P < .001); however, there is variability in PROMIS that ODI cannot account for. ODI questions about sitting and sleeping were weakly correlated across the 3 PROMIS domains. Linear regression showed overall ODI score as accounting for 58.3% ( R2 = 0.583) of the variance in PROMIS Physical Function, 63.9% ( R2 = 0.639) of the variance in Pain Interference score, and 49.9% ( R2 = 0.499) of the variance in Pain Intensity score. Conclusions: There is a large amount of variability with PROMIS that cannot be accounted for with ODI. ODI questions regarding walking, social life, and lifting ability correlate strongly with PROMIS while sitting, standing, and sleeping do not. These results reinforce the utility of PROMIS as a valid assessment for low back disability, while indicating the need for further evaluation of the factors responsible for variation between PROMIS and ODI.

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Comparison Between Polyvinyl Alcohol Implant and Cheilectomy With Moberg Osteotomy for Hallux Rigidus

Foot & Ankle International ◽

10.1177/1071100720947380 ◽

2020 ◽

Vol 41 (9) ◽

pp. 1031-1040 ◽

Cited By ~ 1

Author(s):

Bopha Chrea ◽

Stephanie K. Eble ◽

Jonathan Day ◽

Scott J. Ellis ◽

Mark C. Drakos ◽

...

Keyword(s):

Postoperative Complications ◽

Polyvinyl Alcohol ◽

Pain Intensity ◽

Physical Function ◽

Patient Reported Outcomes ◽

Hallux Rigidus ◽

Measurement Information ◽

Level Of Evidence ◽

Patient Reported

Background: In 2016, the US Food and Drug Administration (FDA) approved the use of a polyvinyl alcohol (PVA) hydrogel implant for the surgical management of hallux rigidus. Though recent studies have evaluated the safety and efficacy of the implant, no study has compared outcomes following PVA implantation with those following traditional joint-preserving procedures for hallux rigidus, such as cheilectomy with Moberg osteotomy. The purpose of this study was to compare clinical and patient-reported outcomes for patients undergoing cheilectomy and Moberg osteotomy, with or without PVA implant, at a single multisurgeon academic center. Our hypothesis was that the addition of the PVA implant would result in superior clinical and patient-reported outcomes. Methods: In total, 166 patients were identified who underwent cheilectomy and Moberg osteotomy with (PVACM; n = 72) or without (CM; n = 94) a PVA implant between January 2016 and December 2018 by 1 of 8 foot and ankle fellowship-trained orthopedic surgeons at our institution. Of these patients, 60 PVACM and 73 CM patients had both baseline and minimum 1-year postoperative Patient-Reported Outcomes Measurement Information System (PROMIS) scores. The average time to survey follow-up was 14.5 months for PVACM patients and 15.6 months for CM patients. Retrospective chart review was performed to assess the incidence of postoperative complications and reoperations, with an average clinical follow-up of 27.7 (range, 16.0-46.4) months for PVACM patients and 36.6 (range, 18.6-47.8) months for CM patients. Results: Both PVACM and CM cohorts demonstrated significant improvement in the PROMIS Physical Function, Pain Interference, Pain Intensity, and Global Physical Health domains when comparing preoperative and postoperative scores within each group ( P < .01). When comparing scores between the PVACM and CM cohorts, preoperative scores were similar, while CM patients demonstrated significantly higher postoperative Physical Function (51.8 ± 8.7 vs 48.8 ± 8.0; P = .04) and significantly lower Pain Intensity (39.9 ± 8.3 vs 43.4 ± 8.7; P = .02) scores. The pre- to postoperative change in Physical Function was also significantly greater for CM patients (7.1 ± 8.5 vs 3.6 ± 6.2; P = .011). In the PVACM group, there were 3 revisions (5%), 1 reimplantation, 1 conversion to arthrodesis, and 1 revision to correct hyperdorsiflexion. In the CM group, there was 1 revision (1.4%), a conversion to arthrodesis ( P = .21). Other postoperative complications included persistent pain (7 out of 60 PVACM patients [11.7%] and 8 out of 73 CM patients [11.0%]; P = .90) and infection in 3 PVACM patients (5%) and no CM patients ( P = .05). Conclusion: Though our results generally support the safety and utility of the PVA implant as previously established by the clinical trial, at 1 to 2 years of follow-up, CM without a PVA implant may provide equivalent or better relief compared with a PVACM procedure, while avoiding potential risks associated with the implant. Level of Evidence: Level III, retrospective comparative study.

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Physical Function and Pain Interference Levels of Hallux Rigidus Patients Before and After Synthetic Cartilage Implant vs Arthrodesis Surgery

Foot & Ankle International ◽

10.1177/10711007211007843 ◽

2021 ◽

pp. 107110072110078

Author(s):

Peter Y. Joo ◽

Judith F. Baumhauer ◽

Olivia Waldman ◽

Samantha Hoffman ◽

Jeffrey Houck ◽

...

Keyword(s):

Physical Function ◽

Hallux Rigidus ◽

Pain Interference ◽

Measurement Information ◽

Complication Rates ◽

Time Points ◽

Phone Survey ◽

Patient Reported ◽

The Mean

Background: Hallux rigidus is a common and painful degenerative condition of the great toe limiting a patient’s physical function and quality of life. The purpose of this study was to investigate pre- and postoperative physical function (PF) and pain interference (PI) levels of patients undergoing synthetic cartilage implant hemiarthroplasty (SCI) vs arthrodesis (AD) for treatment of hallux rigidus using the Patient-Reported Outcomes Measurement Information System (PROMIS). Methods: PROMIS PF and PI t scores were analyzed for patients who underwent either SCI or AD. Postoperative final PROMIS t scores were obtained via phone survey. Linear mixed model analysis was used to assess differences in PF and PI at each follow-up point. Final follow-up scores were analyzed using independent sample t tests. Results: Total 181 (59 SCI, 122 AD) operatively managed patients were included for analysis of PROMIS scores. Final phone survey was performed at a minimum of 14 (mean 33, range, 14-59) months postoperatively, with 101 patients (40 SCI, 61 AD) successfully contacted. The mean final follow-up was significantly different for SCI and AD: 27 vs 38 months, respectively ( P < .01). The mean age of the SCI cohort was lower than the AD cohort (57.5 vs 61.5 years old, P = .01). Average PF t scores were higher in the SCI cohort at baseline (47.1 and 43.9, respectively, P = .01) and at final follow-up (51.4 vs 45.9, respectively, P < .01). A main effect of superior improvement in PF was noted in the SCI group (+4.3) vs the AD group (+2) across time intervals ( P < .01). PI t scores were similar between the 2 procedures across time points. Conclusion: The SCI cohort reported slightly superior PF t scores preoperatively and at most follow-up time points compared with the arthrodesis group. No differences were found for PI or complication rates between the 2 treatment groups during this study time frame. Level of Evidence: Level III.

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Is there a Difference in Outcomes between Patients who Received a Double or Triple Arthrodesis for Hindfoot Arthritis?

Foot & Ankle Orthopaedics ◽

10.1177/2473011420s00251 ◽

2020 ◽

Vol 5 (4) ◽

pp. 2473011420S0025

Author(s):

Amanda H. Holleran ◽

Judith F. Baumhauer ◽

Jeff Houck ◽

Daniel Homeier ◽

Adolph S. Flemister ◽

...

Keyword(s):

Physical Function ◽

Pain Interference ◽

Foot Deformities ◽

Triple Arthrodesis ◽

Chi Square ◽

Post Surgery ◽

Increased Risk ◽

Patient Reported ◽

Chi Square Analysis

Category: Hindfoot Introduction/Purpose: Triple arthrodesis has historically been considered the standard of treatment for arthritis of the hindfoot with or without deformity. The complications of this surgery including non-union, malunion, nerve injury, infection and wound healing problems can occur at any of the three joints. Double arthrodesis is capable of producing a similar reduction in degrees of motion and correction of foot deformity but may also cause less patient morbidity in regard to these complications due to one less joint being incorporated into the fusion procedure. What is unknown is the patient reported outcomes, specifically physical function (PF) and pain interference (PI) between these two procedures. The purpose of this study is to evaluate the clinical outcomes for hindfoot deformity using a triple compared to a double arthrodesis. Methods: A retrospective medical record review was performed (February 2015-December 2019), of 96 identified cases, 54 had complete data over 4 months post operation for either a double (Age = 58 (11); Body Mass Index (BMI) = 34.4 (6.0); n=24) or triple arthrodesis (Age= 55 (13); BMI = 33.0 (10.0); n = 30). Patient Reported Outcome Measurement Information System (PROMIS) physical function and pain interference were assessed at last available pre-operation and last follow up time points. Medical records were reviewed for complications (yes/no). ANOVA models were used to assess differences pre to post surgery (covariates included age, BMI, and length of follow up). Chi Square analysis was used to assess proportions of patients achieving a minimal clinically important difference (34.5) and complications by group. Results: There were no differences between groups in terms of age (p = 0.51), BMI (p = 0.44), or length of follow up (triple = 540 (334) days versus double = 390 (336) days; p=0.12). There were no significant differences in PROMIS PF (pre-post change 95% CI: triple= 1.2 (-4.1 to 1.6) versus double = 0.2 (-2.5 to 2.0)). The for PROMIS PI both groups experienced lower pain (average 5.1 (1.0) with the greater decrease in pain in the triple group (Figure 1; pre-post change 95% CI: triple= 7.1 (-10.2 to -4.0) versus double = 3 (-5.5 to -0.6)). Chi square analysis showed that a greater proportion of patients undergoing a triple (triple 61.9 % versus double 33.3 %) experienced MCID improvement in PROMIS PI (X2=4.4, p=0.04). There were 4 complications in the double group, and 6 in the triple group. Conclusion: Double arthrodesis can allow for similar correction of foot deformities without the increased risk of wound complication, infection or nonunion/malunion. However, we found that patients who underwent a triple arthrodesis were more likely to have an improvement in minimally important clinical difference (MCID) in the PROMIS pain interference scores than those who underwent a double arthrodesis.

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Preoperative PROMIS Physical Function Scores Predict Postoperative Outcomes Following Total Ankle Replacement

Foot & Ankle Orthopaedics ◽

10.1177/24730114211020335 ◽

2021 ◽

Vol 6 (3) ◽

pp. 247301142110203

Author(s):

Matthew S. Conti ◽

Kristin C. Caolo ◽

Agnes D. Cororaton ◽

Jonathan T. Deland ◽

Constantine A. Demetracopoulos ◽

...

Keyword(s):

Pain Intensity ◽

Physical Function ◽

Outcome Measurement ◽

Total Ankle Replacement ◽

Postoperative Outcomes ◽

Pain Interference ◽

Measurement Information ◽

Ankle Replacement ◽

Patient Reported ◽

Postoperative Improvement

Background: Despite good evidence that supports significant improvements in pain and physical function following a total ankle replacement (TAR) for end-stage ankle arthritis, there is a subset of patients who do not significantly benefit from surgery. The purpose of this study was to perform a preliminary analysis to determine if preoperative Patient-Reported Outcome Measurement Information System (PROMIS) scores could be used to predict which patients were at risk of not meaningfully improving following a TAR. Methods: Prospectively collected preoperative and ≥2-year postoperative PROMIS physical function, pain interference, pain intensity, and depression scores for 111 feet in 105 patients were included in the study. Significant postoperative improvement was defined using minimal clinically important differences (MCIDs). Logistic regression models and area under the curve (AUC) analyses were used to determine whether preoperative PROMIS scores were predictive of postoperative outcomes. Results: Receiver operating characteristic curves found statistically significant AUCs for the PROMIS physical function (AUC = 0.728, P = .004), pain intensity (AUC = 0.720, P = .018), and depression (AUC = 0.761, P < .001) domains. The preoperative PROMIS pain interference domain did not achieve a statistically significant AUC. Conclusion: Preoperative PROMIS physical function and pain intensity t scores may be used to predict postoperative improvement in patients following a fixed-bearing TAR; however, preoperative PROMIS pain interference scores were not good predictors. The results of this study may be used to guide research regarding patient-reported outcomes following TAR. Level of Evidence: Level III, retrospective comparative series.

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Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Blood ◽

10.1182/blood-2020-136193 ◽

2020 ◽

Vol 136 (Supplement 1) ◽

pp. 10-10

Author(s):

Julie Kanter ◽

John F. Tisdale ◽

Markus Y Mapara ◽

Janet L. Kwiatkowski ◽

Lakshmanan Krishnamurti ◽

...

Keyword(s):

Sleep Disturbance ◽

Pain Intensity ◽

Physical Function ◽

Board Of Directors ◽

Sickle Cell ◽

Research Funding ◽

Pain Interference ◽

Advisory Committees ◽

Patient Reported ◽

Population Norm

Background In patients with sickle cell disease (SCD), health-related quality of life (HRQoL) is worse than in the general population and comparable or worse than in patients with other chronic or painful diseases such as cystic fibrosis or cancer. Targeting SCD pathophysiology may significantly improve HRQoL in addition to clinical outcomes. In the ongoing phase 1/2 HGB-206 Study (NCT02140554), which evaluates the safety and efficacy of LentiGlobin for SCD (bb1111) gene therapy (GT), the most recently treated cohort of patients (Group C) have demonstrated improvements in laboratory assessments, including a trend toward normalization in key hemolysis markers and improvements in total hemoglobin values, and near resolution of vaso-occlusive crises and acute chest syndrome (ACS), suggesting a fundamental effect on sickle cell pathophysiology. Patient-reported HRQoL outcomes through 12 months post-treatment are presented here. Methods Patients (≥12 and ≤50 years of age) with SCD and history of stroke or severe vaso-occlusive events, including acute episodes of pain and ACS, were enrolled. CD34+ cells collected by plerixafor mobilization/apheresis were transduced with BB305 lentiviral vector. LentiGlobin was infused following myeloablative busulfan conditioning. In addition to laboratory and clinical assessments, patients were monitored for patient-reported outcomes (PROs) using the PRO Measurement Information System (PROMIS)-57. PROMIS-57 assesses HRQoL using collection of short forms containing 8 questions for each of the 7 PROMIS domains (Depression, Anxiety, Pain Interference, Fatigue, Sleep Disturbance, in which a lower score denotes improvement, and Physical Function, Satisfaction with Participation in Social Roles, in which a higher score denotes improvement) and a 0-10 Pain Intensity numeric rating scale (NRS). PROMIS-57 has been validated in patients with SCD. Data were analyzed for ten Group C patients who had at least 12 months of follow-up and had completed PROMIS-57 assessments as of March 3, 2020. For each domain, patients were stratified into 2 sub-groups based on baseline scores and population norm (i.e., baseline scores "better" than or near the population norm and baseline scores "worse" than the population norm). The stratification was built upon the premise that patients with baseline scores "better" or near the population norm would not be expected to improve. The US general population norm was 2.6 for Pain Intensity and a T-score of 50 for all other domains. The minimal clinically importance difference (2-point difference for pain intensity NRS and 5-point difference for other domains) was selected based on the PROMIS guidelines and literature. Results Patients who had baseline scores "worse" than the population norm reported improvements in all domains at Month 6, which were sustained through Month 12. These patients reported clinically meaningful improvement in 6/8 domains; mean T-scores at baseline and Month 12 were 6 and 2.4 for Pain Intensity (n=5); 63 and 48 for Pain Interference (n=7); 62 and 48 for Anxiety (n=3); 62 and 44 for Depression (n=4); 39 and 60 for Satisfaction with Social Roles (n=5); and 40 and 56 for Physical Function (n=4), respectively. Only 1 patient was included in the analysis of Fatigue and Sleep Disturbance domains, thereby limiting the conclusions in these 2 domains (Figure 1). Patients who had baseline scores that were "better" or near than the population norm reported clinically meaningful improvements in the Physical Function (n=6) and Fatigue domains (n=9); mean scores at baseline and Month 12 were 49 and 55 for Physical Function and 50 and 43 for Fatigue, respectively. Among patients in this sub-group, Pain Intensity (n=5) and Pain Interference (n=3) scores were stable from Month 6 through Month 12; there was no clinically meaningful change for the Anxiety (n=7) and Depression (n=6) domains, however, worsening was observed in the Satisfaction with Social Role (n=4) and Sleep Disturbance (n=9) domains (Figure 1). Summary LentiGlobin for SCD GT improved HRQoL in all domains of PROMIS-57 for patients whose baseline scores were "worse" than the population norm, including clinically meaningful improvements in all evaluable (6/8) domains. Larger sample sizes are required to clarify the impact of LentiGlobin for SCD for some PROMIS-57 domains. Disclosures Kanter: SCDAA Medical and Research Advisory Board: Membership on an entity's Board of Directors or advisory committees; AGIOS: Membership on an entity's Board of Directors or advisory committees; BEAM: Membership on an entity's Board of Directors or advisory committees; Jeffries: Honoraria; Cowen: Honoraria; Wells Fargo: Honoraria; NHLBI Sickle Cell Advisory Board: Membership on an entity's Board of Directors or advisory committees; bluebird bio, inc: Consultancy, Honoraria; Novartis: Consultancy; Sanofi: Consultancy; Medscape: Honoraria; Guidepoint Global: Honoraria; GLG: Honoraria. Kwiatkowski:Terumo Corp: Research Funding; Imara: Consultancy; Celgene: Consultancy; Agios: Consultancy; bluebird bio, Inc.: Consultancy, Research Funding; Novartis: Research Funding; Sangamo: Research Funding; Apopharma: Research Funding; Bristol Myers Squibb: Consultancy. Chen:bluebird bio, Inc.: Consultancy. Gallagher:bluebird bio, Inc.: Current Employment, Other: Ownership Interest and Salary. Ding:bluebird bio, Inc.: Current Employment, Other: Salary. Goyal:bluebird bio, Inc.: Current Employment, Other: Ownership Interest and Salary. Paramore:bluebird bio, Inc.: Current Employment, Other: Ownership Interest and Salary. Thompson:bluebird bio, Inc.: Consultancy, Research Funding; BMS: Consultancy, Research Funding; CRISPR/Vertex: Research Funding; Baxalta: Research Funding; Biomarin: Research Funding; Novartis: Consultancy, Honoraria, Research Funding. Walters:AllCells, Inc: Consultancy; Veevo Biomedicine: Consultancy; Editas: Consultancy.

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Comparison of Microfracture with Extracellular Matrix Augmentation and Osteochondral Autograft Transplantation for the Treatment of Medium-Size Osteochondral Lesions of the Talus

Foot & Ankle Orthopaedics ◽

10.1177/2473011420s00044 ◽

2020 ◽

Vol 5 (4) ◽

pp. 2473011420S0004

Author(s):

Oliver B. Hansen ◽

Stephanie K. Eble ◽

Taylor Cabe ◽

Karan A. Patel ◽

Jonathan T. Deland ◽

...

Keyword(s):

Extracellular Matrix ◽

Revision Surgery ◽

Hyaline Cartilage ◽

Lesion Size ◽

Medium Size ◽

Osteochondral Lesions ◽

Repair Tissue ◽

Therapeutic Injection ◽

Patient Reported

Category: Ankle; Arthroscopy; Basic Sciences/Biologics Introduction/Purpose: Historically, microfracture has been used to treat small talar osteochondral lesions (OLTs) with good results, while osteochondral autologous transplantation (OAT) has proven superior for the treatment of larger lesions. It is not clear which method is more effective for medium-sized lesions, around the critical size of 150 mm2 above which microfracture outcomes tend to be poor. While OAT carries the risk of co-morbidity at the knee and often requires a malleolar osteotomy, it is thought to result in superior repair tissue compared to microfracture by introducing native hyaline cartilage to the ankle. Microfracture, in contrast, results in the formation of structurally inferior fibrocartilage. The purpose of this study was to determine the relative benefits of OAT and microfracture in the treatment of medium-sized OLTs. Methods:: Patients treated for an OLT with OAT or microfracture by a single surgeon fellowship-trained in foot and ankle orthopedics between 2015 and 2018 were screened. Both OAT and microfracture techniques were augmented with a mixture of extracellular matrix and bone marrow aspirate concentrate (ECM-BMAC) for every case included in this study. Patients treated without ECM-BMAC were excluded. Only patients with a lesion size between 80 and 165 mm2 were included. Minimum follow-up was 12 months. Clinical outcomes were collected in the form of FAOS or PROMIS scores, depending on departmental standards at the time of treatment. MRIs were collected for radiographic analysis of cartilage repair tissue. MRIs were scored using the MOCART system by a fellowship trained radiologist and were also evaluated for the presence of cysts and edema. Patient charts were reviewed to determine rates of revision surgery and therapeutic injection for pain. Results:: 52 patients were identified who fit inclusion criteria. 27 of these patients received microfracture and 25 received OAT. The average lesion size for all patients was 117.5 mm2. Patients treated with OAT had significantly higher average total MOCART scores (69 vs. 55, p = 0.04) and significantly lower rates of cyst (14% vs. 55%, p <0.01), edema (59% vs. 90%, p = 0.04), revision surgery (0% vs. 19%, p = 0.05), and therapeutic injection for pain (4% vs. 30%, p = 0.03) compared to patients treated with microfracture. No significant differences were detected in patient reported outcome scores between groups for either FAOS or PROMIS. Age, BMI, lesion size, lesion location, and follow-up time were statistically indistinguishable between groups. Conclusion:: In treating OLTs, the native hyaline cartilage introduced by OAT appears to result in higher quality repair tissue when compared to microfracture, as evidenced by OAT patients’ higher MOCART scores and lower rates of cyst and edema. This advantage was also reflected in the fact that OAT patients required revision surgery and therapeutic injection for pain less frequently than did microfracture patients. OAT may offer benefits over MF that outweigh its greater risk of comorbidity when addressing medium-sized OLTs. [Table: see text]

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Safety and efficacy of cement augmentation with fenestrated pedicle screws for tumor-related spinal instability

Neurosurgical FOCUS ◽

10.3171/2021.2.focus201121 ◽

2021 ◽

Vol 50 (5) ◽

pp. E12

Author(s):

Elie Massaad ◽

Myron Rolle ◽

Muhamed Hadzipasic ◽

Ali Kiapour ◽

Ganesh M. Shankar ◽

...

Keyword(s):

Pain Intensity ◽

Spinal Metastases ◽

Pedicle Screws ◽

Cement Augmentation ◽

Pain Interference ◽

Safety And Efficacy ◽

Spine Stabilization ◽

Patient Reported ◽

Cement Extravasation

OBJECTIVE Achieving rigid spinal fixation can be challenging in patients with cancer-related instability, as factors such as osteopenia, radiation, and immunosuppression adversely affect bone quality. Augmenting pedicle screws with cement is a strategy to overcome construct failure. This study aimed to assess the safety and efficacy of cement augmentation with fenestrated pedicle screws in patients undergoing posterior, open thoracolumbar surgery for spinal metastases. METHODS A retrospective review was performed for patients who underwent surgery for cancer-related spine instability from 2016 to 2019 at the Massachusetts General Hospital. Patient demographics, surgical details, radiographic characteristics, patterns of cement extravasation, complications, and prospectively collected Patient-Reported Outcomes Measurement Information System Pain Interference and Pain Intensity scores were analyzed using descriptive statistics. Logistic regression was performed to determine factors associated with cement extravasation. RESULTS Sixty-nine patients underwent open posterior surgery with a total of 502 cement-augmented screws (mean 7.8 screws per construct). The median follow-up period for those who survived past 90 days was 25.3 months (IQR 10.8–34.6 months). Thirteen patients (18.8%) either died within 90 days or were lost to follow-up. Postoperative CT was performed to assess the instrumentation and patterns of cement extravasation. There was no screw loosening, pullout, or failure. The rate of cement extravasation was 28.9% (145/502), most commonly through the segmental veins (77/145, 53.1%). Screws breaching the lateral border of the pedicle but with fenestrations within the vertebral body were associated with a higher risk of leakage through the segmental veins compared with screws without any breach (OR 8.77, 95% CI 2.84–29.79; p < 0.001). Cement extravasation did not cause symptoms except in 1 patient who developed a symptomatic thoracic radiculopathy requiring decompression. There was 1 case of asymptomatic pulmonary cement embolism. Patients experienced significant pain improvement at the 3-month follow-up, with decreases in Pain Interference (mean change 15.8, 95% CI 14.5–17.1; p < 0.001) and Pain Intensity (mean change 28.5, 95% CI 26.7–30.4; p < 0.001). CONCLUSIONS Cement augmentation through fenestrated pedicle screws is a safe and effective option for spine stabilization in the cancer population. The risk of clinically significant adverse events from cement extravasation is very low.

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