Alexander H. Schmidt , Deborah Buk , Alexander Platz , Marcel R.M. van den Brink
{"title":"Cryopreservation for All Is No Option in Unrelated Stem Cell Transplantation. Comment on Dholaria B, et al. Securing the Graft During Pandemic: Are We Ready for Cryopreservation for All? Biol Blood Marrow Transplant. 2020;26:e145-e146.","authors":"Alexander H. Schmidt , Deborah Buk , Alexander Platz , Marcel R.M. van den Brink","doi":"10.1016/j.bbmt.2020.08.011","DOIUrl":"10.1016/j.bbmt.2020.08.011","url":null,"abstract":"","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":null,"pages":null},"PeriodicalIF":4.3,"publicationDate":"2020-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.08.011","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38296339","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Alexandra Gomez-Arteaga , Gunjan L. Shah , Raymond E. Baser , Michael Scordo , Josel D. Ruiz , Adam Bryant , Parastoo B. Dahi , Arnab Ghosh , Oscar B. Lahoud , Heather J. Landau , Ola Landgren , Brian C. Shaffer , Eric L. Smith , Guenther Koehne , Miguel-Angel Perales , Sergio A. Giralt , David J. Chung
{"title":"Prognostic Factors for Postrelapse Survival after ex Vivo CD34+-Selected (T Cell-Depleted) Allogeneic Hematopoietic Cell Transplantation in Multiple Myeloma","authors":"Alexandra Gomez-Arteaga , Gunjan L. Shah , Raymond E. Baser , Michael Scordo , Josel D. Ruiz , Adam Bryant , Parastoo B. Dahi , Arnab Ghosh , Oscar B. Lahoud , Heather J. Landau , Ola Landgren , Brian C. Shaffer , Eric L. Smith , Guenther Koehne , Miguel-Angel Perales , Sergio A. Giralt , David J. Chung","doi":"10.1016/j.bbmt.2020.07.016","DOIUrl":"10.1016/j.bbmt.2020.07.016","url":null,"abstract":"<div><p>Allogeneic hematopoietic cell transplantation (alloHCT) for multiple myeloma (MM), with its underlying graft-versus-tumor capacity, is a potentially curative approach for high-risk patients. Relapse is the main cause of treatment failure, but predictors for postrelapse survival are not well characterized. We conducted a retrospective analysis to evaluate predictors for postrelapse overall survival (OS) in 60 MM patients who progressed after myeloablative T cell-depleted alloHCT. The median patient age was 56 years, and 82% had high-risk cytogenetics. Patients received a median of 4 lines of therapy pre-HCT, and 88% achieved at least a partial response (PR) before alloHCT. Of the 38% who received preemptive post-HCT therapy, 13 received donor lymphocyte infusions (DLIs) and 10 received other interventions. Relapse was defined as very early (<6 months; 28%), early (6 to 24 months; 50%), or late (>24 months; 22%). At relapse, 27% presented with extramedullary disease (EMD). The median postrelapse overall survival (OS) by time to relapse was 4 months for the very early relapse group, 17 months for the early relapse group, and 72 months for the late relapse group (<em>P</em> = .002). Older age, relapse with EMD, <PR before alloHCT, <PR by day +100, and no maintenance were prognostic for inferior postrelapse OS on univariate analysis. On multivariate analysis adjusted for age and sex, very early relapse (hazard ratio [HR], 4.37; 95% confidence interval [CI], 1.42 to 13.5), relapse with EMD (HR, 5.20; 95% CI, 2.10 to 12.9), and DLI for relapse prevention (HR, .11; 95% CI, 2.10 to 12.9) were significant predictors for postrelapse survival. Despite their shared inherent high-risk status, patients with MM have significantly disparate post-HCT relapse courses, with some demonstrating long-term survival despite relapse.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":null,"pages":null},"PeriodicalIF":4.3,"publicationDate":"2020-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.07.016","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38195358","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Eduardo Rodríguez-Arbolí , Myriam Labopin , Johanna Tischer , Arne Brecht , Arnold Ganser , Jürgen Finke , Igor Wolfgang Blau , Nicolaus Kröger , Peter Kalhs , Edouard Forcade , Donald Bunjes , Alexandros Spyridonidis , Bipin Savani , Arnon Nagler , Mohamad Mohty
{"title":"FLAMSA-Based Reduced-Intensity Conditioning versus Myeloablative Conditioning in Younger Patients with Relapsed/Refractory Acute Myeloid Leukemia with Active Disease at the Time of Allogeneic Stem Cell Transplantation: An Analysis from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation","authors":"Eduardo Rodríguez-Arbolí , Myriam Labopin , Johanna Tischer , Arne Brecht , Arnold Ganser , Jürgen Finke , Igor Wolfgang Blau , Nicolaus Kröger , Peter Kalhs , Edouard Forcade , Donald Bunjes , Alexandros Spyridonidis , Bipin Savani , Arnon Nagler , Mohamad Mohty","doi":"10.1016/j.bbmt.2020.07.020","DOIUrl":"10.1016/j.bbmt.2020.07.020","url":null,"abstract":"<div><p>The use of myeloablative conditioning (MAC) in the setting of active relapsed/refractory (R/R) acute myeloid leukemia (AML) has been hindered by high historical rates of nonrelapse mortality (NRM). FLAMSA (fludarabine, Ara-C, and amsacrine) chemotherapy (CT) followed by reduced-intensity conditioning (RIC) has been proposed as an effective and potentially safer alternative in this scenario. As improvements in supportive care have contributed to decreasing NRM rates after MAC, a comparative reassessment of these two strategies was performed. This was a registry-based analysis by the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. Eligibility criteria included age 18 to 50 years, primary refractory, first or second relapsed active AML, first allogeneic stem cell transplantation from a matched sibling donor (MSD) or an unrelated donor (UD) performed between 2005 and 2018, MAC or FLAMSA-RIC. A total of 1018 patients were included. The median patient age was 39 years (range, 18 to 50). Two hundred and fifty-eight patients received busulfan (Bu)/cyclophosphamide (Cy), 314 received Cy/total body irradiation (TBI), 318 received FLAMSA-TBI, and 128 received FLAMSA-CT. The median duration of follow-up was 50 months. In univariate analysis, the 2-year relapse incidence (RI) (54%; 95% confidence interval (CI), 50%-57%), leukemia-free survival (LFS) (30%; 95% CI, 27%-33%), and refined graft-versus-host disease-free, relapse-free survival (GRFS) (21%; 95% CI, 18%-24%) were not significantly different between cohorts. Lower 2-year NRM was observed in the FLAMSA-CT group (7% versus 16% in Bu/Cy, 19% in Cy/TBI, and 18% in FLAMSA-TBI; <em>P</em> = .04), as well as increased 2-year overall survival (OS) (50% versus 33% in Bu/Cy, 34% in Cy/TBI, and 36% in FLAMSA-TBI; <em>P</em> = .03). These results were maintained in the multivariate analysis (hazard ratio [HR] for NRM: .40, <em>P</em> = .01; HR for OS: .65, <em>P</em> = .01; Bu/Cy as reference). These data suggest that FLAMSA-CT may be a preferred conditioning regimen in patients with active R/R AML due to lower NRM. Yet, the high relapse rates observed in our analyses emphasize the need for novel therapeutic strategies in this clinical setting.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":null,"pages":null},"PeriodicalIF":4.3,"publicationDate":"2020-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.07.020","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38205688","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Megan M. Herr, George L. Chen, Maureen Ross, Hillary Jacobson, Renee McKenzie, Laura Markel, Sophia R. Balderman, Christine M. Ho, Theresa Hahn, Philip L. McCarthy
{"title":"Identification of Neurotoxicity after Chimeric Antigen Receptor (CAR) T Cell Infusion without Deterioration in the Immune Effector Cell-Associated Encephalopathy (ICE) Score","authors":"Megan M. Herr, George L. Chen, Maureen Ross, Hillary Jacobson, Renee McKenzie, Laura Markel, Sophia R. Balderman, Christine M. Ho, Theresa Hahn, Philip L. McCarthy","doi":"10.1016/j.bbmt.2020.07.031","DOIUrl":"10.1016/j.bbmt.2020.07.031","url":null,"abstract":"<div><p>A consensus grading schema for cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) resulting from chimeric antigen receptor (CAR) T cell therapy was published in 2019. Although this consensus grading schema has been imperative in identifying and monitoring CRS and ICANS in our CAR T cell population, we observed patients exhibiting subtle neurotoxicity symptoms prior to a decrease in their immune effector cell-associated encephalopathy (ICE) score, which is one component of ICANS. Because we treat grade 1 ICANS at our institution, identification of early neurotoxicity symptoms is important. Additionally, we found changes in personality, occupational confusion, or inability to answer dichotomous questions were early signs of neurotoxicity. Therefore, we developed a 3-step command tool to supplement the ICE evaluation. We present 2 examples of patients who exhibited early neurotoxicity symptoms and led us to develop this tool and 1 in whom it was effective. We propose that CAR T cell patients are consistently followed by a clinical care provider who is familiar with the patient to recognize early changes in personality, behavior, and cognition. Additionally, we propose that the multistep command tool be used in conjunction with the ICE score to detect early symptoms of ICANS. Early intervention has the potential to prevent irreversible neurotoxicity.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":null,"pages":null},"PeriodicalIF":4.3,"publicationDate":"2020-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.07.031","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38212624","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"ASTCT Notes","authors":"","doi":"10.1016/j.bbmt.2020.10.012","DOIUrl":"https://doi.org/10.1016/j.bbmt.2020.10.012","url":null,"abstract":"","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":null,"pages":null},"PeriodicalIF":4.3,"publicationDate":"2020-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.10.012","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"137415573","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Gina Jiang , Jose-Mario Capo-Chichi , Aijun Liu , Eshetu G. Atenafu , Rajat Kumar , Mark D. Minden , Hong Chang
{"title":"Combination of FLT3-ITD Allelic Ratio, NPM1 Mutation, and Immunophenotypic Markers to Modulate Outcome Prediction in Patients with Normal Karyotype Acute Myelogenous Leukemia Undergoing Hematopoietic Stem Cell Transplantation","authors":"Gina Jiang , Jose-Mario Capo-Chichi , Aijun Liu , Eshetu G. Atenafu , Rajat Kumar , Mark D. Minden , Hong Chang","doi":"10.1016/j.bbmt.2020.07.017","DOIUrl":"10.1016/j.bbmt.2020.07.017","url":null,"abstract":"<div><p><em>NPM1</em> mutation status and the allelic ratio (AR) of <em>FLT3-</em>internal tandem duplication (FLT3-ITD) are routinely tested for disease risk stratification in patients with normal karyotype (NK) acute myelogenous leukemia (AML); however, the predictive impact of immunophenotypic markers on different <em>NPM1/FLT3</em> genotypes remains unclear. We performed a retrospective analysis of 423 patients with NK-AML subclassified into groups based on <em>NPM1/FLT3</em> genotype. Allogeneic hematopoietic stem cell transplantation (HSCT) was performed in 124 of 423 patients (29%) and was significantly associated with longer event-free survival (EFS) and overall survival (OS), except for patients with the favorable genotype, defined as mutated <em>NPM1</em> (<em>NPM1</em><sup>mut</sup>) combined with normal <em>FLT3</em> status (<em>FLT3</em>-ITD<sup>neg</sup>) or <em>FLT3</em>-ITD AR <.5 (<em>FLT3</em>-ITD<sup>low</sup>). A subset of AML patients bearing the favorable <em>NPM1</em><sup>mut</sup>/<em>FLT3</em>-ITD<sup>neg/low</sup> genotype share similar outcomes with AML patients who have the intermediate <em>FLT3/NPM1</em> genotype defined by normal <em>NPM1</em> (<em>NPM1</em><sup>wt</sup>) and <em>FLT3</em>-ITD<sup>neg/low</sup>. In these individuals, the lack of CD13 expression (CD13<sup>neg</sup>) was associated with shorter EFS (<em>P</em> = .041) and OS (<em>P</em> = .017). CD13<sup>neg</sup> was an independent predictor for shorter OS (hazard ratio, 1.985; <em>P</em> = .028).</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":null,"pages":null},"PeriodicalIF":4.3,"publicationDate":"2020-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.07.017","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38195357","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Edward W. Li , Anna Lee , Maryam Vaseghi-Shanjani , Alexander Anagnostopoulos , Gabriele Jagelaviciute , Elena Kum , Tanya Petraszko , Heidi Elmoazzen , David Allan , Warren Fingrut
{"title":"Development and Evaluation of a Whiteboard Video Series to Support the Education and Recruitment of Committed Unrelated Donors for Hematopoietic Stem Cell Transplantation","authors":"Edward W. Li , Anna Lee , Maryam Vaseghi-Shanjani , Alexander Anagnostopoulos , Gabriele Jagelaviciute , Elena Kum , Tanya Petraszko , Heidi Elmoazzen , David Allan , Warren Fingrut","doi":"10.1016/j.bbmt.2020.07.008","DOIUrl":"10.1016/j.bbmt.2020.07.008","url":null,"abstract":"<div><p>Whiteboard videos are a popular video format, allowing viewers to see drawings of concepts alongside explanatory text and speech. We hypothesized that whiteboard videos could support the education and recruitment of unrelated stem cell donors in Canada. A series of 5 sharable whiteboard videos about stem cell donation was produced and posted online in September 2018, including 1 full-length video (<span>https://youtu.be/V4fVBtxnWfM</span><svg><path></path></svg>) and 4 shorter videos titled “What Is Stem Cell Transplantation?” “How Does the Matching Process Work?” “How Are Stem Cells Donated?” and “How Can I Register as a Stem Cell Donor?” In the videos, metaphorical interpretations of stem cells as factories and genetic markers as barcode labels are employed to communicate complex concepts. The particular need for young, male, and ethnically diverse donors is reflected in the characters portrayed. Surveys demonstrated the videos (1) were used and valued by stakeholders in donor recruitment and (2) significantly improved objective and self-reported knowledge about stem cell donation and reduced donation-related ambivalence among viewers from the most-needed donor demographics. Use of the whiteboard videos was also associated with improved donor recruitment outcomes in Canada. Our work is relevant to donor registries and recruitment organizations worldwide that seek to improve their recruitment efforts.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":null,"pages":null},"PeriodicalIF":4.3,"publicationDate":"2020-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.07.008","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38159944","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Christianne Bourlon , Santiago Riviello-Goya , Aldo A Acosta-Medina , Rosa E. Caballero-Landinez , Angelica Manrique-Rubio , Kevin Teran-De-la-Sancha , Alfonso Gulias-Herrero , Maria T. Bourlon
{"title":"Outcomes and Challenges of Reproductive Health in Hematopoietic Stem Cell Transplantation Survivors","authors":"Christianne Bourlon , Santiago Riviello-Goya , Aldo A Acosta-Medina , Rosa E. Caballero-Landinez , Angelica Manrique-Rubio , Kevin Teran-De-la-Sancha , Alfonso Gulias-Herrero , Maria T. Bourlon","doi":"10.1016/j.bbmt.2020.07.007","DOIUrl":"10.1016/j.bbmt.2020.07.007","url":null,"abstract":"<div><p>Long-term therapy-related reproductive health side effects impact the quality of life of hematopoietic stem cell transplantation (HSCT) survivors. In this study, we evaluated the prevalence of gonadal dysfunction (GD) pre- and post-HSCT, analyzed factors associated with GD, and explored rates of fertility assessment (FA) and fertility preservation (FP) in a resource-limited setting. FA and outcomes of patients age ≤45 years undergoing HSCT between June 2000 and May 2018 were collected retrospectively. We included 213 patients with a median age of 26 years. Pre-HSCT FA was performed in 71.8%, with a GD rate of 17%. The rate of GD was not different between the sexes (females, 19.5% versus males, 16.1%; <em>P</em> = .616) and was only associated with increasing age. The rate of cryopreservation in the cohort was 3.3%. Almost one-half (47.7%) of post-HSCT patients completed FA and evidenced an increase in GD rate to 48.9%. Comparing pre-HSCT and post-HSCT GD rates, women had a significant increase (19.5% versus 81.4%; <em>P</em> < .001), whereas men did not (16.1% versus 20.4%; <em>P</em> = .76). These results were confirmed by a multiple imputation analysis accounting for missing data. Female sex, pre-HSCT cytotoxic therapy, myeloablative conditioning, and germ cell tumor (GCT) diagnosis were associated with post-HSCT GD. Reproductive health preservation can be positively impacted when FA and FP are prioritized at the initial diagnosis in HSCT candidates, particularly in women of older age and men with a diagnosis of GCT. The low FP success observed urges implementation of strategies that favor accessibility and improve quality of life of HSCT survivors in low- and middle-income countries.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":null,"pages":null},"PeriodicalIF":4.3,"publicationDate":"2020-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.07.007","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38154656","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Alpana Waghmare , Maheen Z. Abidi , Michael Boeckh , Roy F. Chemaly , Sanjeet Dadwal , Zeinab El Boghdadly , Mini Kamboj , Genovefa A. Papanicolaou , Steven A. Pergam , Zainab Shahid
{"title":"Guidelines for COVID-19 Management in Hematopoietic Cell Transplantation and Cellular Therapy Recipients","authors":"Alpana Waghmare , Maheen Z. Abidi , Michael Boeckh , Roy F. Chemaly , Sanjeet Dadwal , Zeinab El Boghdadly , Mini Kamboj , Genovefa A. Papanicolaou , Steven A. Pergam , Zainab Shahid","doi":"10.1016/j.bbmt.2020.07.027","DOIUrl":"10.1016/j.bbmt.2020.07.027","url":null,"abstract":"<div><p>There are currently limited data on the epidemiology, clinical manifestations, and optimal management of Coronavirus Disease 2019 (COVID-19) in hematopoietic cell transplantation and cellular therapy recipients. Given the experience with other respiratory viruses, we anticipate that patients may develop severe clinical disease and thus provide the following general principles for cancer centers across the nation. These guidelines were developed by members of the American Society for Transplantation and Cellular Therapy Infectious Diseases Special Interest Group. Specific practices may vary depending on local epidemiology and testing capacity, and the guidance provided in this document may change as new information becomes available.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":null,"pages":null},"PeriodicalIF":4.3,"publicationDate":"2020-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.07.027","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38213319","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Louise Bondeelle , Sylvie Chevret , Charlotte Hurabielle , Laila Samy , Tiphaine Goletto , Adrien Costantini , Flore Sicre de Fontbrune , David Michonneau , Gérard Socié , Abdellatif Tazi , Jean-David Bouaziz , Anne Bergeron
{"title":"Effect of Ruxolitinib on Lung Function after Allogeneic Stem Cell Transplantation","authors":"Louise Bondeelle , Sylvie Chevret , Charlotte Hurabielle , Laila Samy , Tiphaine Goletto , Adrien Costantini , Flore Sicre de Fontbrune , David Michonneau , Gérard Socié , Abdellatif Tazi , Jean-David Bouaziz , Anne Bergeron","doi":"10.1016/j.bbmt.2020.07.033","DOIUrl":"10.1016/j.bbmt.2020.07.033","url":null,"abstract":"<div><p>Ruxolitinib, a selective Janus kinase (JAK)1/2 inhibitor, has recently been proposed for steroid-refractory chronic graft-versus host disease (cGVHD) after allogeneic hematopoietic stem cell transplantation (HSCT), particularly in severe skin cGVHD. Lung function impairment is common in severe skin cGVHD through concomitant bronchiolitis obliterans syndrome (BOS) or restrictive lung disease (RLD) from skin sclerosis. To date, no treatment has shown a benefit on lung function in this context. We retrospectively assessed the effect of ruxolitinib on lung function in a cohort of 70 patients diagnosed with sclerotic-type skin cGVHD between March 2015 and April 2018. Among these patients, 36 received ruxolitinib. To handle confounding by indication bias, exposure groups were matched on the propensity score to receive ruxolitinib, incorporating age, myeloablative conditioning, total body irradiation, BOS, forced expiratory volume in 1 second (FEV<sub>1</sub>), forced vital capacity (FVC), and tobacco use at the time of cohort entry, as well as the time from transplantation. The 1:1 matching used a greedy-matching algorithm with replacement, with a caliper of 0.10. FVC and FEV<sub>1</sub> trajectories during follow-up were compared in the matched samples, using linear mixed-effects models. The median duration of follow-up of the 46 matched patients was 58 months (interquartile range, 32 to 84 months). Ten patients had an RLD (6 exposed, 4 unexposed), and 13 patients were diagnosed with BOS (8 exposed, 5 unexposed). FEV<sub>1</sub> decreased significantly over time independent of exposure to ruxolitinib (<em>P</em> < .0001). The FEV<sub>1</sub> trajectory was similar in the exposed patients and the unexposed patients (<em>P</em> = .11). In conclusion, ruxolitinib administration did not demonstrate any improvement in the course of respiratory function in allogeneic HSCT recipients with sclerotic-type skin cGVHD.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":null,"pages":null},"PeriodicalIF":4.3,"publicationDate":"2020-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.07.033","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38224692","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}