Research Spotlight

Ogura, Y., J. L. Gum, R. A. Hostin, C. Robinson, C. P. Ames, S. D. Glassman, D. C. Burton, R. S. Bess, C. I. Shaffrey, J. S. Smith, S. Yeramaneni, V. F. Lafage, T. Protopsaltis, P. G. Passias, F. J. Schwab and L. Y. Carreon (2020). “Cost-effectiveness of surgical treatment of adult spinal deformity: comparison of posterior-only versus anteroposterior approach.” Spine J Apr 12. pii: S1529-9430(20)30136-4. [Epub ahead of print].

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BACKGROUND CONTEXT: Considerable debate exists regarding the optimal surgical approach for adult spinal deformity (ASD). It remains unclear which approach, posterior-only or combined anterior-posterior (AP), is more cost-effective. Our goal is to determine the 2-year cost per quality-adjusted life year (QALY) for each approach. PURPOSE: To compare the 2-year cost-effectiveness of surgical treatment for ASD between the posterior-only approach and combined AP approach. STUDY DESIGN: Retrospective economic analysis of a prospective, multicenter database PATIENT SAMPLE: From a prospective, multicenter surgical database of ASD, patients undergoing five or more level fusions through a posterior-only or AP approach were identified and compared. METHODS: QALYs gained were determined using baseline, 1-year, and 2-year postoperative Short Form 6D. Cost was calculated from actual, direct hospital costs including any subsequent readmission or revision. Cost-effectiveness was determined using cost/QALY gained. RESULTS: The AP approach showed significantly higher index cost than the posterior-only approach ($84,329 vs. $64,281). This margin decreased at 2-year follow-up with total costs of $89,824 and $73,904, respectively. QALYs gained at 2 years were similar with 0.21 and 0.17 in the posterior-only and the AP approaches, respectively. The cost/QALY at 2 years after surgery was significantly higher in the AP approach ($525,080) than in the posterior-only approach ($351,086). CONCLUSIONS: We assessed 2-year cost-effectiveness for the surgical treatment through posterior-only and AP approaches. The posterior-only approach is less expensive both for the index surgery and at 2-year follow-up. The QALY gained at 2-years was similar between the two approaches. Thus, posterior-only approach was more cost-effective than the AP approach under our study parameters. However, both approaches were not cost-effective at 2-year follow-up.

Nakagawa, T., J. Cho and D. Yeung (2020). “Successful Aging in East Asia: Comparison among China, Korea, and Japan.” J Gerontol B Psychol Sci Soc Sci Apr 23. pii: gbaa042. [Epub ahead of print].

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OBJECTIVES: Heterogeneity in successful aging has been found across countries. Yet, comparable evidence is sparse except in North America and Europe. Extending prior research, this study examined the prevalence and correlates of successful aging in East Asian: China, Korea, and Japan. METHOD: We used harmonized datasets from national surveys. A total of 6,479 participants (aged between 65 and 75) were analyzed. Using Rowe and Kahn’s (1987, 1997) model, successful aging was defined as having no major diseases, no difficulty performing activities of daily living, obtaining a median or higher score on tests of cognitive function, and being actively engaged. RESULTS: The average prevalence of successful agers was 17.6%. There were variations in the global and specific measures of successful aging within and across countries, even after controlling for individual sociodemographic factors (age, gender, and education). The odds of aging successfully was highest in Japan and lowest in China, especially in the rural areas. Being younger and males were associated with a higher likelihood of successful agers in both global and specific measures. DISCUSSION: This study observed heterogeneity in successful aging in East Asia. To identify policy implications, future research should explore potential societal factors influencing individuals’ opportunities for successful aging.

Mukherjee, S., E. Stroberg, F. Wang, L. Morales, Y. Shan, A. Rao, J. H. Huang, E. Wu and E. Fonkem (2020). “SMARCB1 Gene Mutation Predisposes to Earlier Development of Glioblastoma: A Case Report of Familial GBM.” J Neuropathol Exp Neurol 79(5): 562-565.

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Glioblastoma (GBM) is the most aggressive adult brain tumor. While GBM typically occurs sporadically, familial GBM can be associated with certain hereditary disorders and isolated familial GBMs in the absence of syndrome are rare. Relevant hereditary factors have remained elusive in these cases. Understanding specific genetic abnormality may potentially lead to better treatment strategies in these patients. Here, we analyzed GBM tissue from our patient and 2 afflicted family members, with next generation sequencing to better understand the genetic alterations associated with this disease development. DNA was extracted and sequenced and the data were then analyzed. Results revealed 2 common mutations in afflicted family members; PDGFRA and HRAS. In addition, both siblings showed a mutation of the SMARCB1 gene. The sister of our patient exhibited a homozygous mutation, while our patient had heterozygous mutation of this gene in the tumor tissue. This result suggests that mutation of SMARCB1, either alone or in the presence of PDGFRA and HRAS mutations, is associated with earlier onset GBM.

Minzenmayer, A., R. N. Miranda, P. Powell and P. Parekh (2020). “An unusual case of cutaneous Waldenstrom macroglobulinemia with the MYD88 L265P mutation.” J Cutan Pathol Apr 26. [Epub ahead of print].

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Waldenstrom macroglobulinemia is a lymphoplasmacytic lymphoma with bone marrow involvement and a monoclonal IgM gammopathy. Infiltration of the skin by neoplastic cells is very rare, and it can be difficult to distinguish from marginal zone lymphoma. The MYD88 L265P mutation is strongly associated with Waldenstrom macroglobulinemia, and it may be helpful in differentiating the two disorders, although the presence of this mutation is not specific, and other factors must be considered when making the final diagnosis. We present a diagnostically challenging case of cutaneous Waldenstrom macroglobulinemia in which the MYD88 L265P mutation was identified in the skin but not in the bone marrow, due to a low tumor burden. This article is protected by copyright. All rights reserved.

Mehta, R. S., S. G. Holtan, T. Wang, M. T. Hemmer, S. R. Spellman, M. Arora, D. R. Couriel, A. M. Alousi, J. Pidala, H. Abdel-Azim, V. Agrawal, I. Ahmed, A. S. Al-Homsi, M. Aljurf, J. H. Antin, M. Askar, J. J. Auletta, V. R. Bhatt, L. Chee, S. Chhabra, A. Daly, Z. DeFilipp, J. Gajewski, R. P. Gale, U. Gergis, P. Hematti, G. C. Hildebrandt, W. J. Hogan, Y. Inamoto, R. Martino, N. S. Majhail, D. I. Marks, T. Nishihori, R. F. Olsson, A. Pawarode, M. A. Diaz, T. Prestidge, H. G. Rangarajan, O. Ringden, A. Saad, B. N. Savani, H. Schoemans, S. Seo, K. R. Schultz, M. Solh, T. Spitzer, J. Storek, T. Teshima, L. F. Verdonck, B. Wirk, J. A. Yared, J. Y. Cahn and D. J. Weisdorf (2020). “Composite GRFS and CRFS Outcomes After Adult Alternative Donor HCT.” J Clin Oncol May 4:JCO1900396. [Epub ahead of print]: Jco1900396.

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PURPOSE: There is no consensus on the best choice of an alternative donor (umbilical cord blood [UCB], haploidentical, one-antigen mismatched [7/8]-bone marrow [BM], or 7/8-peripheral blood [PB]) for hematopoietic cell transplantation (HCT) for patients lacking an HLA-matched related or unrelated donor. METHODS: We report composite end points of graft-versus-host disease (GVHD)-free relapse-free survival (GRFS) and chronic GVHD (cGVHD)-free relapse-free survival (CRFS) in 2,198 patients who underwent UCB (n = 838), haploidentical (n = 159), 7/8-BM (n = 241), or 7/8-PB (n = 960) HCT. All groups were divided by myeloablative conditioning (MAC) intensity or reduced intensity conditioning (RIC), except haploidentical group in which most received RIC. To account for multiple testing, P < .0071 in multivariable analysis and P < .00025 in direct pairwise comparisons were considered statistically significant. RESULTS: In multivariable analysis, haploidentical group had the best GRFS, CRFS, and overall survival (OS). In the direct pairwise comparison of other groups, among those who received MAC, there was no difference in GRFS or CRFS among UCB, 7/8-BM, and 7/8-PB with serotherapy (alemtuzumab or antithymocyte globulin) groups. In contrast, the 7/8-PB without serotherapy group had significantly inferior GRFS, higher cGVHD, and a trend toward worse CRFS (hazard ratio [HR], 1.38; 95% CI, 1.13 to 1.69; P = .002) than the 7/8-BM group and higher cGVHD and trend toward inferior CRFS (HR, 1.36; 95% CI, 1.14 to 1.63; P = .0006) than the UCB group. Among patients with RIC, all groups had significantly inferior GRFS and CRFS compared with the haploidentical group. CONCLUSION: Recognizing the limitations of a registry retrospective analysis and the possibility of center selection bias in choosing donors, our data support the use of UCB, 7/8-BM, or 7/8-PB (with serotherapy) grafts for patients undergoing MAC HCT and haploidentical grafts for patients undergoing RIC HCT. The haploidentical group had the best GRFS, CRFS, and OS of all groups.