Article
Respiratory System
Robert F. Tamburro, Kenneth R. Cooke, Stella M. Davies, Samuel Goldfarb, James S. Hagood, Ashok Srinivasan, Marie E. Steiner, Dennis Stokes, Nancy DiFronzo, Nahed El-Kassar, Nonniekaye Shelburne, Aruna Natarajan
Summary: Approximately 2,500 pediatric hematopoietic cell transplants, mostly allogeneic, are performed annually in the United States for life-threatening malignant and nonmalignant conditions. Post-HCT complications, with pulmonary dysfunction as the leading cause of nonrelapse mortality, limit successful outcomes. A multidisciplinary group of 33 experts convened to address knowledge gaps and research strategies to improve outcomes.
ANNALS OF THE AMERICAN THORACIC SOCIETY
(2021)
Review
Immunology
Tirthankar Sen, Rajkumar P. Thummer
Summary: The human microbiota plays a significant role in organ transplantation outcomes and rejection risk, with complex interactions existing between different microbiotas and the human body.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Immunology
Alexandra Laberko, Anna Mukhina, Elena Machneva, Olga Pashchenko, Tatiana Bykova, Larisa Vahonina, Gleb Bronin, Yulia Skvortsova, Elena Skorobogatova, Irina Kondratenko, Larisa Fechina, Anna Shcherbina, Ludmila Zubarovskaya, Dmitry Balashov, Alexander Rumiantsev
Summary: This study collected and analyzed data on HSCT activity in IEI patients in Russia. The results showed that the number of IEI patients receiving HSCT has increased significantly over the years, and there has been a change in the distribution of IEI diagnoses. The study suggests the need for additional transplant beds for IEI in Russia.
JOURNAL OF CLINICAL IMMUNOLOGY
(2023)
Article
Oncology
Eileen Danaher Hacker, Rae Lee Richards, Mohammad Abu Zaid, Shu-Yu Chung, Susan Perkins, Sherif Shafik Farag
Summary: This pilot study tested the acceptability, feasibility, and preliminary effects of a free-living physical activity intervention for MM patients after HCT. The results showed that the intervention was acceptable and feasible, but there were some declines in symptoms and physical performance. This study provides implications for nurses to help patients increase physical activity.
Review
Oncology
Divyanshu Mohananey, Alexandra Sarau, Rohit Kumar, David Lewandowski, Sol M. Abreu-Sosa, Sunita Nathan, Tochukwu M. Okwuosa
Summary: HSCT is a standard treatment for malignancies, with cardiovascular disease and risk factors becoming significant issues for morbidity and mortality in this patient population. Studies show beneficial effects of physical activity in HSCT patients, although data may be limited. Further research is needed on the use of cardiac rehabilitation in this population.
JACC: CARDIOONCOLOGY
(2021)
Article
Medicine, General & Internal
B. Gentner, F. Tucci, S. Galimberti, F. Fumagalli, M. De Pellegrin, P. Silvani, C. Camesasca, S. Pontesilli, S. Darin, F. Ciotti, M. Sarzana, G. Consiglieri, C. Filisetti, G. Forni, L. Passerini, D. Tomasoni, D. Cesana, A. Calabria, G. Spinozzi, M-P Cicalese, V Calbi, M. Migliavacca, F. Barzaghi, F. Ferrua, V Gallo, S. Miglietta, E. Zonari, P. S. Cheruku, C. Forni, M. Facchini, A. Corti, M. Gabaldo, S. Zancan, S. Gasperini, A. Rovelli, J. J. Boelens, S. A. Jones, R. Wynn, C. Baldoli, E. Montini, S. Gregori, F. Ciceri, M. G. Valsecchi, G. la Marca, R. Parini, L. Naldini, A. Aiuti, M-E Bernardo
Summary: Hematopoietic gene therapy for Hurler syndrome in patients resulted in extensive metabolic correction in both peripheral tissues and the central nervous system, showing promising treatment outcomes.
NEW ENGLAND JOURNAL OF MEDICINE
(2021)
Review
Oncology
Jessica Stokes, Megan S. Molina, Emely A. Hoffman, Richard J. Simpson, Emmanuel Katsanis
Summary: Bendamustine, a chemotherapeutic agent used to treat various cancers, has shown promising immunomodulatory effects in the context of allogeneic hematopoietic cell transplantation. It has been found to reduce graft-versus-host disease while enhancing graft-versus-leukemia effects, making it a potential candidate for improving clinical outcomes in transplant patients. Further studies are underway to explore the full range of its immunomodulatory effects and potential applications in clinical settings.
Article
Immunology
Rebecca Isabel Wurm-Kuczera, Judith Buentzel, Julia Felicitas Leni Koenig, Tobias Legler, Jan-Jakob Valk, Justin Hasenkamp, Wolfram Jung, Jan-Gerd Rademacher, Peter Korsten, Gerald Georg Wulf
Summary: Sarcoidosis is a disease characterized by altered immune response and inflammatory granulomatous tissue infiltration. It may occur during immune reconstitution after HSCT, with glucocorticoid treatment resolving symptoms effectively. The HLA allele association reflects that observed in the general population.
FRONTIERS IN IMMUNOLOGY
(2021)
Letter
Gastroenterology & Hepatology
Mohammad Haneef Khan, Guillaume C. Onyeaghala, Armin Rashidi, Shernan G. Holtan, Alexander Khoruts, Ajay Israni, Pamala A. Jacobson, Christopher Staley
Summary: The activity of intestinal microbiota-produced beta-glucuronidase plays a crucial role in the metabolism of the immunosuppressant mycophenolate mofetil. Differences in beta-glucuronidase activity between transplant types may account for variations in dosing requirements.
Article
Multidisciplinary Sciences
Laura Breda, Tyler E. Papp, Michael P. Triebwasser, Amir Yadegari, Megan T. Fedorky, Naoto Tanaka, Osheiza Abdulmalik, Giulia Pavani, Yongping Wang, Stephan A. Grupp, Stella T. Chou, Houping Ni, Barbara L. Mui, Ying K. Tam, Drew Weissman, Stefano Rivella, Hamideh Parhiz
Summary: This study developed a nanoparticle system that can target hematopoietic stem cells in vivo and achieved correction of diseased cells through mRNA delivery. Furthermore, this system enables non-genotoxic conditioning for stem cells, providing a new approach for HSCT.
Article
Oncology
Mary Eapen, Ruta Brazauskas, David A. Williams, Mark C. Walters, Andrew St Martin, Benjamin L. Jacobs, Joseph H. Antin, Kira Bona, Sonali Chaudhury, Victoria H. Coleman-Cowger, Nancy L. DiFronzo, Erica B. Esrick, Joshua J. Field, Courtney D. Fitzhugh, Julie Kanter, Neena Kapoor, Donald B. Kohn, Lakshmanan Krishnamurti, Wendy B. London, Michael A. Pulsipher, Sohel Talib, Alexis A. Thompson, Edmund K. Waller, Ted Wun, Mary M. Horowitz
Summary: This study investigates the incidence and risk factors for secondary neoplasm after transplantation for sickle cell disease. The results show that the 10-year incidence of leukemia/MDS was 1.7% and of any secondary neoplasm was 2.4%. Low-intensity regimens were associated with higher risks for leukemia/MDS or any secondary neoplasm compared with more intense regimens.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Review
Immunology
Xiaofeng Zhou, Bethany B. Moore
Summary: Pulmonary infections are a major concern for hematopoietic cell transplantation recipients, with mouse models playing a key role in understanding the impacts of these infections. Studies have shown that post-transplant infectious complications adversely affect innate immunity, and previous infections may contribute to noninfectious pulmonary issues post-HCT.
FRONTIERS IN IMMUNOLOGY
(2021)
Review
Immunology
Motoko Koyama, Geoffrey R. Hill
Summary: Allogeneic stem cell transplantation is an effective therapy for hematopoietic malignancies, relying on donor T cells and NK cells for the graft-versus-leukemia effect. However, graft-versus-host disease remains a major complication, highlighting the importance of understanding alloantigen presentation to separate beneficial effects from detrimental ones. Mouse models and tools play a key role in quantifying alloantigen presentation post-alloSCT.
FRONTIERS IN IMMUNOLOGY
(2021)
Article
Immunology
Panpan Zhu, Yibo Wu, Dawei Cui, Jimin Shi, Jian Yu, Yanmin Zhao, Xiaoyu Lai, Lizhen Liu, Jue Xie, He Huang, Yi Luo
Summary: Post-HSCT PRCA is a rare complication in major ABO-incompatible HSCT, with donor type being an independent factor associated with its development. Most patients with PRCA can fully recover, and recipients of haploidentical donors have a lower risk of post-HSCT PRCA.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Allergy
Rebecca A. Marsh, Kyle Hebert, Soyoung Kim, Christopher C. Dvorak, Victor M. Aquino, K. Scott Baker, Deepak Chellapandian, Blachy Davila Saldana, Christine N. Duncan, Michael J. Eckrich, George E. Georges, Timothy S. Olson, Michael A. Pulsipher, Shalini Shenoy, Elizabeth Stenger, Mark Vander Lugt, Lolie C. Yu, Andrew R. Gennery, Mary Eapen
Summary: Allogeneic hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis (HLH) disorders is associated with substantial morbidity and mortality. The study found that conditioning regimens using flu/melphalan and flu/melphalan/thiotepa had better outcomes compared to other regimens in terms of post-transplant complications and survival rates in HLH patients.
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2022)
Article
Public, Environmental & Occupational Health
Mingqian Lin, Liliana E. Pezzin, Ali Mohamedi, Ankit Kansagra, Anita D'Souza
Summary: Despite known racial disparities in other plasma cell disorders, little is known about racial differences in the incidence of AL amyloidosis. This study aimed to assess the reporting of patients' race in published clinical research on AL amyloidosis in the USA. The results showed underreporting of race/ethnicity, with single institution studies being less likely to report race. Standardized collection and reporting of racial/ethnic demographics are needed to identify disparities and promote equitable care.
JOURNAL OF RACIAL AND ETHNIC HEALTH DISPARITIES
(2023)
Article
Hematology
Anita D'Souza, Ruta Brazauskas, Edward A. Stadtmauer, Marcelo C. Pasquini, Parameswaran Hari, Asad Bashey, Natalie Callander, Steven Devine, Yvonne Efebera, Siddhartha Ganguly, Cristina Gasparetto, Nancy Geller, Mary M. Horowitz, John Koreth, Heather Landau, Claudio Brunstein, Philip McCarthy, Muzaffar H. Qazilbash, Sergio Giralt, Amrita Krishnan, Kathryn E. Flynn
Summary: Early autologous hematopoietic cell transplantation is the standard treatment for multiple myeloma. This study aimed to investigate the long-term symptom burden and quality of life after transplantation. The results showed that patients achieved excellent recovery of quality of life scores by 1 year post-transplant, but many patients continued to experience moderate to severe symptoms.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Article
Hematology
Rachel Cusatis, Michael J. Martens, Ryotaro Nakamura, Corey S. Cutler, Wael Saber, Stephanie J. Lee, Brent R. Logan, Bronwen E. Shaw, Alyssa Gregory, Anita D'Souza, Betty K. Hamilton, Mary M. Horowitz, Kathryn E. Flynn
Summary: For older adults with MDS, the survival advantage associated with allogeneic hematopoietic cell transplantation (alloHCT) does not come at the cost of worse quality of life, reassuring those who prefer a curative approach to treating MDS.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Editorial Material
Hematology
Mary Eapen
Summary: This article reports on the outcomes of allogeneic hematopoietic stem cell transplantation (allo-HSCT) and conservative management for adult patients with inborn errors of immunity (IEI).
Letter
Biophysics
Anita D'Souza, Ruta Brazauskas, Bi Qing Teng, Grant Yun, Hannah Uttley, Jing Dong, Michael B. Dwinell, Marcelo C. Pasquini, Sergio Giralt, Heather Landau, Edward Stadtmauer, Amrita Krishnan, Siegfried Janz
BONE MARROW TRANSPLANTATION
(2023)
Letter
Oncology
Anita D'Souza, Shaji Kumar
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Article
Oncology
Mary Eapen, Ruta Brazauskas, David A. Williams, Mark C. Walters, Andrew St Martin, Benjamin L. Jacobs, Joseph H. Antin, Kira Bona, Sonali Chaudhury, Victoria H. Coleman-Cowger, Nancy L. DiFronzo, Erica B. Esrick, Joshua J. Field, Courtney D. Fitzhugh, Julie Kanter, Neena Kapoor, Donald B. Kohn, Lakshmanan Krishnamurti, Wendy B. London, Michael A. Pulsipher, Sohel Talib, Alexis A. Thompson, Edmund K. Waller, Ted Wun, Mary M. Horowitz
Summary: This study investigates the incidence and risk factors for secondary neoplasm after transplantation for sickle cell disease. The results show that the 10-year incidence of leukemia/MDS was 1.7% and of any secondary neoplasm was 2.4%. Low-intensity regimens were associated with higher risks for leukemia/MDS or any secondary neoplasm compared with more intense regimens.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Article
Health Care Sciences & Services
Anita D'Souza, Aniko Szabo, Idayat Akinola, Muriel Finkel, Kathryn E. Flynn
Summary: We conducted a cross-sectional study to characterize health-related quality of life and symptom burden in individuals living with light chain (AL) amyloidosis. HRQL was measured using PROMIS and PRO-CTCAE questionnaires. The results showed that AL amyloidosis patients have significant impairments in physical function, fatigue, and social roles compared to the general population. High symptom burden was observed in both long-term survivors and those not on active therapy. Therefore, patient-reported outcomes should be measured regularly to provide the best supportive care for all AL amyloidosis patients.
QUALITY OF LIFE RESEARCH
(2023)
Article
Health Care Sciences & Services
Tiffany P. Quock, Anita D'Souza, Michael S. Broder, Katalin Bognar, Eunice Chang, Marian H. Tarbox
Summary: This study describes the clinical and economic burden of hospitalizations for amyloid light chain (AL) amyloidosis. The results show that delayed diagnosis of the disease is common and is associated with poor prognosis, increased healthcare utilization, and higher costs. Patients with both cardiac and renal involvement have a higher mortality rate, and the hospitalization costs and duration for AL amyloidosis are significantly higher.
JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH
(2023)
Article
Hematology
Meera Mohan, Rajshekhar Chakraborty, Susan Bal, Anoma Nellore, Muhamed Baljevic, Anita D'Souza, Peter G. Pappas, Jesus G. Berdeja, Natalie Callander, Luciano J. Costa
Summary: CAR T-cell and bispecific antibody therapies have shown remarkable efficacy in multiple myeloma patients, but they come with a significant risk of severe infections due to various factors. As these therapies have been recently approved, it is crucial to establish practical guidelines for infection monitoring and prevention until robust data from clinical trials become available. A panel of experienced investigators from COMMIT developed consensus recommendations for mitigating infections associated with CAR T-cell and bispecific antibody therapies in multiple myeloma patients.
BRITISH JOURNAL OF HAEMATOLOGY
(2023)
Letter
Oncology
Mary Eapen, Ruta Brazaukas
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Editorial Material
Oncology
Robert Peter Gale, Wolfgang Hinterberger, Neal S. Young, Andrew R. Gennery, Christopher C. Dvorak, Kyle M. Hebert, Michael Heim, Larisa Broglie, Mary Eapen
Article
Oncology
Marc S. Raab, Yael C. Cohen, Fredrik Schjesvold, Kimberly Aardalen, Adwait Oka, Andrew Spencer, Martin Wermke, Anita D. Souza, Jonathan L. Kaufman, Anna Maria Cafro, Enrique M. Ocio, Noriko Doki, Kristin Henson, Gina Trabucco, Ana Carrion, Florent C. Bender, Pierre-Eric Juif, Adonai Fessehatsion, Liqiong Fan, Jeffrey P. Stonehouse, John W. Blankenship, Brian Granda, Serena De Vita, Haihui Lu
Summary: BCMA is a promising target for multiple myeloma treatment, and therapies targeting BCMA have shown high response rates. WVT078, an anti-BCMA/anti-CD3 bispecific antibody, has demonstrated acceptable safety and preliminary evidence of clinical activity in ongoing clinical trials.
Article
Hematology
Anita D'Souza, Aniko Szabo, Idayat Akinola, Muriel Finkel, Kathryn E. Flynn
Summary: This study aimed to assess the impact of organ involvement on patient-reported outcomes (PROs) in light chain (AL) amyloidosis. PROs were evaluated using the KCCQ-12, PROMIS-29 + 2, and SF-36. The results showed that fatigue, physical function, social function, and global physical health were the most discriminative factors for assessing the severity of organ involvement.
EUROPEAN JOURNAL OF HAEMATOLOGY
(2023)
Letter
Oncology
Rajshekhar Chakraborty, Divaya Bhutani, Mathew S. Maurer, Meera Mohan, Suzanne Lentzsch, Anita D'Souza
BLOOD CANCER JOURNAL
(2023)