Review
Oncology
Amar H. Kelkar, Joseph H. Antin, Roman M. Shapiro
Summary: This review summarizes the most common long-term complications of hematopoietic cell transplantation (HCT) and emphasizes the importance of recognizing and managing these complications in survivorship care.
FRONTIERS IN ONCOLOGY
(2023)
Article
Hematology
Rajshekhar Chakraborty, Jean Yi, Lisa Rybicki, Jaime Preussler, Abhinav Deol, Alison Loren, Bipin Savani, Heather S. L. Jim, Jan Cerny, Jana Reynolds, Jennifer Whitten, John R. Wingard, Joseph P. McGuirk, Joseph Uberti, Nandita Khera, Patrick Stiff, Samantha M. Jaglowski, Shahrukh Hashmi, Shernan G. Holtan, Steven Devine, Theresa Hahn, Victoria L. Whalen, Wael Saber, William Wood, K. Scott Baker, Karen Syrjala, Navneet S. Majhail
Summary: Survivors of autologous hematopoietic cell transplantation (AHCT) for multiple myeloma in stable remission do not have clinically meaningful worsening in physical functioning compared to the general population. However, they may experience distress related to health burden, uncertainty, and finances. Adherence to preventive care guidelines is high, but adherence to exercise and diet guidelines is relatively low.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Letter
Clinical Neurology
Robert A. Gross, Yan Yuan, Chris Keran, Janis M. Miyasaki
Summary: The study shows that aHSCT can prevent disability worsening in the majority of multiple sclerosis (MS) patients and induce durable improvement in disability, especially in patients with relapsing-remitting MS. The use of the BEAM + ATG conditioning protocol is associated with a more pronounced suppression of clinical relapses and MRI inflammatory activity.
Article
Hematology
Isabel C. Yoon, Nicholas A. Bascou, Michele D. Poe, Paul Szabolcs, Maria L. Escolar
Summary: HSCT can prolong the lifespan and improve the functional abilities of late-infantile patients with Krabbe disease, especially those who undergo transplantation before symptom onset. The cognitive outcomes were better for patients with disease onset at >12 months compared to untreated patients. The findings support reclassifying late-infantile Krabbe disease to symptom onset at 12 to 36 months of age.
Article
Hematology
Roxane Labrosse, Julia I. Chu, Myriam A. Armant, John K. Everett, Danilo Pellin, Niharika Kareddy, Andrew L. Frelinger, Lauren A. Henderson, Amy E. O'Connell, Amlan Biswas, Jet Coenen-van der Spek, Alexandra Miggelbrink, Claudia Fiorini, Hriju Adhikari, Charles C. Berry, Vito Adrian Cantu, Johnson Fong, Jason Jaroslavsky, Derin F. Karadeniz, Quan-Zhen Li, Shantan Reddy, Aoife M. Roche, Chengsong Zhu, Jennifer S. Whangbo, Colleen Dansereau, Brenda Mackinnon, Emily Morris, Stephanie M. Koo, Wendy B. London, Safa Baris, Ahmet Ozen, Elif Karakoc-Aydiner, Jenny M. Despotovic, Lisa R. Forbes Satter, Akihiko Saitoh, Yuta Aizawa, Alejandra King, Mai Anh Thi Nguyen, Vy Do Uyen Vu, Scott B. Snapper, Anne Galy, Luigi D. Notarangelo, Frederic D. Bushman, David A. Williams, Sung-Yun Pai
Summary: Wiskott-Aldrich syndrome (WAS) is a rare X-linked disorder characterized by immunodeficiency, eczema, microthrombocytopenia, autoimmunity, and lymphoid malignancies. In a phase 1/2 clinical trial, gene therapy using a self-inactivating lentiviral vector was performed on 5 patients with severe WAS, resulting in positive outcomes and improvements in clinical and laboratory manifestations.
Article
Hematology
Karen L. Syrjala, Helene Schoemans, Jean C. Yi, Shelby L. Langer, Ahona Mukherjee, Lynn Onstad, Stephanie J. Lee
Summary: This study investigated sexual activity and function in hematopoietic cell transplantation (HCT) survivors, identifying common sexual dysfunction in both men and women regardless of time since treatment. Women were more likely than men to report sexual inactivity, as well as lower sexual function among sexually active individuals.
TRANSPLANTATION AND CELLULAR THERAPY
(2021)
Review
Oncology
Nandita Khera
Summary: With the increasing awareness about survivorship issues, studies on late complications in HCT survivors are moving beyond description to examining pathogenesis and identifying biomarkers. The ultimate goal is to decrease the incidence of these complications and improve post-HCT management through improved health care delivery models and interventions targeting late effects.
CURRENT HEMATOLOGIC MALIGNANCY REPORTS
(2023)
Review
Hematology
Zachariah DeFilipp, Mehrdad Hefazi, Yi-Bin Chen, Bruce R. Blazar
Summary: This article discusses the application of transplantation in the treatment of congenital or acquired nonmalignant blood diseases. Unlike patients with hematologic malignancies, the priority for nonmalignant blood disease patients is to avoid graft-versus-host disease, and emerging therapeutic methods hold promise for advancing transplantation care.
Article
Oncology
Sanghee Hong, Ruta Brazauskas, Kyle M. Hebert, Siddhartha Ganguly, Hisham Abdel-Azim, Miguel Angel Diaz, Sara Beattie, Stefan O. Ciurea, David Szwajcer, Sherif M. Badawy, Alois A. Gratwohl, Charles LeMaistre, Mahmoud D. S. M. Aljurf, Richard F. Olsson, Neel S. Bhatt, Nosha Farhadfar, Jean A. Yared, Ayami Yoshimi, Sachiko Seo, Usama Gergis, Amer M. Beitinjaneh, Akshay Sharma, Hillard Lazarus, Jason Law, Matthew Ulrickson, Hasan Hashem, Helene Schoemans, Jan Cerny, David Rizzieri, Bipin N. Savani, Rammurti T. Kamble, Bronwen E. Shaw, Nandita Khera, William A. Wood, Shahrukh Hashmi, Theresa Hahn, Stephanie J. Lee, J. Douglas Rizzo, Navneet S. Majhail, Wael Saber
Summary: This study analyzed the impact of community health status on allogeneic HCT outcomes using data from CHRR and CIBMTR, finding that patients residing in counties with a worse community health status have inferior survival due to increased risk of NRM after allogeneic HCT. There was no significant association between the community health status of the transplant center location and allogeneic HCT outcomes.
Article
Biophysics
Maria Cancio, Alexandre G. Troullioud Lucas, Marc Bierings, Elizabeth Klein, Moniek A. de Witte, Frans J. Smiers, Dorine Bresters, Jaap Jan Boelens, Stephanie E. Smetsers
Summary: Allogeneic hematopoietic cell transplantation (HCT) is the only cure for the hematologic manifestations of Fanconi anemia (FA). This study analyzed the outcomes of pediatric and young adult patients who underwent HCT between 2007 and 2020. The results showed that age, HLA mismatch, and year of HCT were predictors of overall survival, event-free survival, and treatment-related mortality.
BONE MARROW TRANSPLANTATION
(2023)
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
Hematology
Robert Chiesa, Jaap Jan Boelens, Christine N. Duncan, Jorn-Sven Kuhl, Caroline Sevin, Neena Kapoor, Vinod K. Prasad, Caroline A. Lindemans, Simon A. Jones, Hernan M. Amartino, Mattia Algeri, Nancy Bunin, Cristina Diaz-de-Heredia, Daniel J. Loes, Esther Shamir, Alison Timm, Elizabeth McNeil, Andrew C. Dietz, Paul J. Orchard
Summary: A study found that allogeneic hematopoietic stem cell transplantation can stabilize neurologic function and improve survival in patients with early cerebral adrenoleukodystrophy. The donor source, donor match, conditioning regimen, and disease stage all have an impact on the outcomes of the transplantation. However, there are associated risks such as transplant-related mortality, graft failure, and graft-versus-host disease.
Review
Hematology
Jacinta Perram, David M. Ross, Donal McLornan, Krisstina Gowin, Nicolas Kroger, Vikas Gupta, Clinton Lewis, Nico Gagelmann, Nada Hamad
Summary: Myelofibrosis is a clonal myeloproliferative neoplasm with limited and ineffective treatment options. The transplantation eligibility criteria for MF patients need to be refined, considering the heterogeneity in disease grade.
AMERICAN JOURNAL OF HEMATOLOGY
(2022)
Article
Hematology
Ryota Hamada, Tsugumi Asano, Masanobu Murao, Junsuke Miyasaka, Michiko Matsushita, Taishi Kajimoto, Ayumi Otagaki, Manabu Nankaku, Yasuyuki Arai, Junya Kanda, Tadakazu Kondo, Akifumi Takaori-Kondo, Ryosuke Ikeguchi, Shuichi Matsuda
Summary: This retrospective cohort study used computed tomography scans to evaluate the quality and quantity of skeletal muscle in patients undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). The study found that radiographic density (RD) was an independent risk factor for poor overall survival, while psoas muscle mass index (PMI) was not significant. Decreased RD and reduced 6-min walking distance before transplantation were significant factors in increased nonrelapse mortality.
TRANSPLANTATION AND CELLULAR THERAPY
(2022)
Correction
Clinical Neurology
G. Boffa, L. Massacesi, M. Inglese
Summary: Despite equal numbers of women and men completing neurology residencies, the majority of AAN members are still men. Based on historical imbalance, it is estimated that women and men will be equal in number among neurologist AAN membership by 2047.
Article
Oncology
Yazeed Sawalha, Tomas Radivoyevitch, Xuefei Jia, Katherine Tullio, Robert M. Dean, Brad Pohlman, Brian T. Hill, Matt Kalaycio, Navneet S. Majhail, Deepa Jagadeesh
Summary: Younger patients with private insurance and receiving treatment at academic facilities are more likely to undergo autologous stem cell transplantation for mantle cell lymphoma, which is associated with improved survival rates.
LEUKEMIA & LYMPHOMA
(2022)
Letter
Biophysics
Pranay Hegde, Lisa Rybicki, Sheila Serafino, Christina Ferraro, Matt Kalaycio, Ronald Sobecks, Aaron T. Gerds, Sharon Caroniti, Donna Corrigan, Kayla Giannetti, Jamie Elberson, Brittany Hodgeman, Jamie Starn, Jane Dabney, Linda McLellan, Navneet S. Majhail, Betty K. Hamilton
BONE MARROW TRANSPLANTATION
(2022)
Letter
Biophysics
Julia H. Joo, Sanghee Hong, Lisa A. Rybicki, Betty K. Hamilton, Navneet S. Majhail
BONE MARROW TRANSPLANTATION
(2022)
Article
Biophysics
Joseph E. Maakaron, Mei-Jie Zhang, Karen Chen, Sunil Abhyankar, Vijaya Raj Bhatt, Saurabh Chhabra, Najla El Jurdi, Sherif S. Farag, Fiona He, Mark Juckett, Marcos de Lima, Navneet Majhail, Marjolein van der Poel, Ayman Saad, Bipin Savani, Celalettin Ustun, Edmund K. Waller, Mark Litzow, Partow Kebriaei, Christopher S. Hourigan, Wael Saber, Daniel Weisdorf
Summary: A study on 1321 AML patients aged 60 and older found that age alone is not a barrier to successful HCT in CR1, and efforts should focus on minimizing residual disease and better donor selection.
BONE MARROW TRANSPLANTATION
(2022)
Letter
Biophysics
Pranay S. Hegde, Lisa Rybicki, Sheila Serafino, Laura Bernhard, Donna Corrigan, Faiz Anwer, Matt Kalaycio, Ronald M. Sobecks, Deepa Jagadeesh, Brian T. Hill, Robert M. Dean, Jack Khouri, Allison M. Winter, Brad Pohlman, Navneet S. Majhail, Betty K. Hamilton
BONE MARROW TRANSPLANTATION
(2022)
Letter
Biophysics
Agrima Mian, Wei Wei, Betty K. Hamilton, Allison M. Winter, Jack Khouri, Brad Pohlman, Aaron T. Gerds, Deepa Jagadeesh, Faiz Anwer, Matt Kalaycio, Robert M. Dean, Ronald Sobecks, Paolo F. Caimi, Brian T. Hill, Navneet S. Majhail
BONE MARROW TRANSPLANTATION
(2023)
Article
Health Care Sciences & Services
Charleen Hsuan, Brendan G. Carr, David Vanness, Yinan Wang, Douglas L. Leslie, Eleanor Dunham, Jeannette A. Rogowski
Summary: Current payment policies neglect hospital transfers for emergency conditions, worsening disparities. There is no existing framework that provides a patient-centered, population-based perspective for the structure of hospital transfer networks. The hospital transfer network equity-quality model highlights the factors that determine the structure of hospital transfer networks, including structural inequity and racism.
Article
Hematology
Brittany Knick Ragon, Mithun Vinod Shah, Anita D'Souza, Noel Estrada-Merly, Lohith Gowda, Gemlyn George, Marcos de Lima, Shahrukh Hashmi, Mohamed A. Kharfan-Dabaja, Navneet S. Majhail, Rahul Banerjee, Ayman Saad, Gerhard C. Hildebrandt, Hira Mian, Muhammad Bilal Abid, Minoo Battiwalla, Lazaros J. Lekakis, Sagar S. Patel, Hemant S. Murthy, Yago Nieto, Christopher Strouse, Sherif M. Badawy, Samer Al Hadidi, Bhagirathbhai Dholaria, Mahmoud Aljurf, David H. Vesole, Cindy H. Lee, Attaphol Pawarode, Usama Gergis, Kevin C. Miller, Leona A. Holmberg, Aimaz Afrough, Melhem Solh, Pashna N. Munshi, Taiga Nishihori, Larry D. Anderson Jr, Baldeep Wirk, Gurbakhash Kaur, Muzaffar H. Qazilbash, Nina Shah, Shaji K. Kumar, Saad Z. Usmani
Summary: The use of proteasome inhibitor and immunomodulatory drug-based combinations, followed by high-dose melphalan and autologous hematopoietic stem cell transplantation (auto-HSCT), has significantly improved the overall survival (OS) in multiple myeloma (MM) patients. However, the use of lenalidomide maintenance after auto-HSCT has been associated with an increased risk of second primary malignancies (SPM) and second hematological malignancies (SHM). This study evaluated the impact of SPM and SHM on progression-free survival (PFS) and OS in MM patients after auto-HSCT using registry data.
Article
Psychology, Multidisciplinary
Jillian C. Strayhorn, Linda M. Collins, David J. Vanness
Summary: In the current practice of intervention science, a component screening approach (CSA) is widely used for selecting intervention components based on estimated main effects and interactions. However, we propose an alternative posterior expected value approach based on Bayesian decision theory, which offers easier application and greater flexibility in intervention optimization. Through Monte Carlo simulations, we found that both the posterior expected value approach and CSA outperformed random component selection and classical treatment package approaches. The posterior expected value approach showed slightly more consistent and accurate results compared to CSA across a range of simulated optimization trials, indicating its potential for decision-making in the multiphase optimization strategy (MOST).
PSYCHOLOGICAL METHODS
(2023)
Article
Surgery
Audrey Stevens, Jennie Meier, Archana Bhat, Sara J. Knight, David J. Vanness, Courtney Balentine
Summary: This study compared survival outcomes between lobectomy and total thyroidectomy in patients with papillary thyroid cancer, while addressing the bias from unmeasured confounding variables. The results showed no significant differences in overall survival or survival in subgroup analyses, indicating that total thyroidectomy does not offer a survival advantage over lobectomy regardless of tumor size, patient age, or overall risk of death.
Article
Hematology
Rahul Banerjee, Amar H. Kelkar, Urshila Durani, Theodora Anagnostou, Miki Nishitani, Kanwaldeep Mallhi, Navneet S. Majhail, Aaron C. Logan
Summary: Recent guidelines from the American Society for Transplantation and Cellular Therapy have aimed to establish expectations for participants in blood and marrow transplantation (BMT) and cellular therapy (CT) fellowships. A survey was conducted to examine the demographics, motivations, and experiences of recent participants in BMT/CT fellowships. The results showed that the fellowship training had a positive impact on participants' comfort with BMT/CT management and the majority would highly recommend this training to others.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Article
Hematology
Sagar S. Patel, Sanghee Hong, Lisa Rybicki, Stephanie Farlow, Jane Dabney, Matt Kalaycio, Ronald Sobecks, Navneet S. Majhail, Betty K. Hamilton
Summary: Acute graft-versus-host disease (GVHD) is a major complication after allogeneic hematopoietic cell transplantation (HCT), causing significant physical and psychosocial symptoms. This pilot study aimed to assess the feasibility of using patient-reported outcome (PRO) measures to evaluate symptom burden and quality of life (QOL) in acute GVHD. The study utilized surveys from various PRO measures and found that patients with acute GVHD had lower QOL scores compared to those without or with mild GVHD. The PRO-CTCAE captured several common symptoms of acute GVHD. Challenges in implementing PRO measures in acute GVHD were identified. Further research is needed to make PROs feasible in this patient population.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Article
Hematology
Rajshekhar Chakraborty, Jean Yi, Lisa Rybicki, Jaime Preussler, Abhinav Deol, Alison Loren, Bipin Savani, Heather S. L. Jim, Jan Cerny, Jana Reynolds, Jennifer Whitten, John R. Wingard, Joseph P. McGuirk, Joseph Uberti, Nandita Khera, Patrick Stiff, Samantha M. Jaglowski, Shahrukh Hashmi, Shernan G. Holtan, Steven Devine, Theresa Hahn, Victoria L. Whalen, Wael Saber, William Wood, K. Scott Baker, Karen Syrjala, Navneet S. Majhail
Summary: Survivors of autologous hematopoietic cell transplantation (AHCT) for multiple myeloma in stable remission do not have clinically meaningful worsening in physical functioning compared to the general population. However, they may experience distress related to health burden, uncertainty, and finances. Adherence to preventive care guidelines is high, but adherence to exercise and diet guidelines is relatively low.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Article
Hematology
Abraham S. Kanate, Navneet Majhail, Zachariah Defilipp, Binod Dhakal, Bhagirathbhai Dholaria, Betty Hamilton, Alex F. Herrera, Yoshihiro Inamoto, Tania Jain, Miguel -Angel Perales, Paul A. Carpenter, Mehdi Hamadani
Summary: The American Society for Transplantation and Cellular Therapy (ASTCT) has published guidelines on CAR-T therapy indications, categorizing them as standard of care and recommending only FDA-approved indications. These guidelines will be periodically updated based on new evidence.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Article
Hematology
Sanghee Hong, Lisa Rybicki, Linda Mclellan, Jane Dabney, Aaron T. Gerds, Seth J. Rotz, Matt Kalaycio, Rabi Hanna, Betty K. Hamilton, Navneet S. Majhail, Ronald M. Sobecks
Summary: This study compared the HRQOL between haplo-HCT and other donor sources for HCT, showing no difference in some aspects but differences in specific situations. Haplo-HCT demonstrated better emotional and functional well-being in certain conditions.
TRANSPLANTATION AND CELLULAR THERAPY
(2022)