Review
Health Care Sciences & Services
Archana Ramgopal, Shiva Sridar, Jignesh Dalal, Ramasubramanian Kalpatthi
Summary: Thrombotic microangiopathy (TMA) is a rare but serious complication of hematopoietic stem cell transplantation (HSCT) in children. This study aimed to estimate the incidence and analyze the risk factors of TMA in HSCT recipients, finding that HHV6 is not only a risk factor for TMA, but also associated with increased mortality in these patients.
JOURNAL OF PERSONALIZED MEDICINE
(2021)
Article
Immunology
Hiroshi Okamura, Hirohisa Nakamae, Takero Shindo, Katsuki Ohtani, Yoshihiko Hidaka, Yasufumi Ohtsuka, Yosuke Makuuchi, Masatomo Kuno, Teruhito Takakuwa, Naonori Harada, Mitsutaka Nishimoto, Yasuhiro Nakashima, Hideo Koh, Asao Hirose, Mika Nakamae, Nobutaka Wakamiya, Masayuki Hino, Norimitsu Inoue
Summary: The study found that plasma levels of Ba protein play a sensitive and predictive role as a biomarker in transplant-associated thrombotic microangiopathy, while complement-related genetic variants do not predict the development of TA-TMA.
FRONTIERS IN IMMUNOLOGY
(2021)
Review
Biochemistry & Molecular Biology
Ioanna Lazana
Summary: Transplant-associated thrombotic microangiopathy (TA-TMA) is a significant contributor to morbidity and mortality after allogenic hematopoietic stem cell transplantation (allo-HSCT). The diagnosis of TA-TMA is challenging due to lack of consensus diagnostic criteria and common clinical features mimicking other diseases. Therapeutic plasma exchange (TPE) has been traditionally used, but the efficacy is doubtful. Complement inhibitors, such as eculizumab, have shown promising results in clinical trials. Future goals include identifying specific risk factors and developing better treatment options.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Editorial Material
Hematology
Anthony Sabulski, Sonata Jodele
Summary: Transplant-associated thrombotic microangiopathy is a life-threatening complication of haematopoietic stem cell transplant, and some patients do not respond to current treatment methods. Qi et al. show that HIF-1α may be a previously unrecognized driver of this endothelial injury syndrome.
BRITISH JOURNAL OF HAEMATOLOGY
(2022)
Review
Biophysics
Joanna A. Young, Christopher R. Pallas, Mary Ann Knovich
Summary: TA-TMA is a recognized complication of HSCT with high morbidity and mortality. Complement inhibition has been explored as a therapeutic option, with Eculizumab and narsoplimab showing effectiveness. Early recognition and treatment may lead to improved outcomes.
BONE MARROW TRANSPLANTATION
(2021)
Article
Hematology
Ambreen Pandrowala, Parth Ganatra, V. P. Krishnan, Ajay Narayan Sharma, Saroj Chavan, Minnie Bodhanwala, Bharat Agarwal, Prashant Hiwarkar
Summary: TA-TMA is an endothelial injury syndrome linked to the overactivation of complement pathways. Inhibition of the lectin pathway with Narsoplimab can be effective in treating TA-TMA.
THROMBOSIS JOURNAL
(2023)
Article
Immunology
Vojtech Petr, Dorottya Csuka, Petra Hruba, Agnes Szilagyi, Marek Kollar, Antonij Slavcev, Zoltan Prohaszka, Ondrej Viklicky
Summary: De novo thrombotic microangiopathy (TMA) is associated with poor kidney graft survival, and there is a recipient-driven process with suspected genetic background. Carriers of the MCPggaac haplotype have a higher risk of graft loss, and longer cold ischemia time is associated with worse graft survival.
FRONTIERS IN IMMUNOLOGY
(2022)
Review
Immunology
Rui Zhang, Meng Zhou, Jiaqian Qi, Wenjing Miao, Ziyan Zhang, Depei Wu, Yue Han
Summary: The study evaluated the efficacy and safety of Eculizumab for TA-TMA, finding that it can improve survival rate and overall response rate in patients, with the most common adverse event being infection. However, more high-quality studies are needed to further validate these findings.
FRONTIERS IN IMMUNOLOGY
(2021)
Article
Immunology
Sonia Elhadad, David Redmond, Jenny Huang, Adrian Tan, Jeffrey Laurence
Summary: This study investigates the involvement of neutrophil intra-cytoplasmic complement activation-induced endothelial cell injury in transplant-associated thrombotic microangiopathy (TA-TMA), and demonstrates that the anti-MASP2 monoclonal antibody narsoplimab can effectively suppress this process.
CLINICAL AND EXPERIMENTAL IMMUNOLOGY
(2023)
Article
Pediatrics
Laura Gomez-Ganda, Maria Isabel Benitez-Carabante, Aurora Fernandez-Polo, Marina Munoz-Lopez, Berta Renedo-Miro, Gema Ariceta, Cristina Diaz De Heredia
Summary: In pediatric patients diagnosed with high-risk TA-TMA after HSCT, treatment with eculizumab led to improvement in renal function, proteinuria, and hypertension. The patients achieved resolution of TA-TMA after treatment with eculizumab for varying durations, and no increased risk of infection was observed with proper vaccine and antibiotic prophylaxis.
FRONTIERS IN PEDIATRICS
(2021)
Article
Hematology
Anthony Sabulski, Grace Arcuri, Sara Szabo, Marguerite M. Care, Christopher E. Dandoy, Stella M. Davies, Sonata Jodele
Summary: TA-TMA and aHUS are complement-mediated TMAs. CNS involvement is rarely reported in TA-TMA, suggesting it may be underdiagnosed. We studied 13 recipients of pediatric HCTs with TA-TMA and found that vascular injury in the brain was common, with severe cases correlating with neurologic symptoms. Classic TMA histology and similar imaging abnormalities were observed. A study of 100 HCT recipients also showed that TA-TMA patients were more likely to develop neurologic symptoms. Based on these findings, we propose considering TA-TMA-directed therapy for low to moderate-risk TA-TMA patients with neurologic complications.
Article
Surgery
Mikhail M. Kanunnikov, Zhemal Z. Rakhmanova, Nikita V. Levkovsky, Aliya I. Vafina, Oleg V. Goloshapov, Tatiana S. Shchegoleva, Julia J. Vlasova, Olesya V. Paina, Elena V. Morozova, Ludmilla S. Zubarovskaya, Alexander D. Kulagin, Ivan S. Moiseev
Summary: Transplant-associated thrombotic microangiopathy (TA-TMA) is a specific complication of allogeneic hematopoietic cell transplantation, and replacing calcineurin inhibitors with sirolimus may lead to better outcomes for patients with TA-TMA.
CLINICAL TRANSPLANTATION
(2021)
Review
Immunology
Michelle L. L. Schoettler, Harshil Bhatt, Sumithira Vasu
Summary: Transplant-associated thrombotic microangiopathy (TA-TMA) is a complication of hematopoietic cellular therapy (HCT) with significant morbidity and mortality. Although the disease is associated with endothelial damage and complement activation, specific diagnostic biomarkers have not been identified. This study reviewed the diagnostic, early risk, and prognostic biomarkers of TA-TMA, finding that sC5b-9 was the most robust biomarker. However, further research is needed to validate and establish cut-off points for biomarkers in separate cohorts.
FRONTIERS IN IMMUNOLOGY
(2023)
Review
Hematology
Sonata Jodele, Anthony Sabulski
Summary: TA-TMA is a severe complication of stem cell transplantation, characterized by endothelial dysfunction leading to hemolytic anemia and microthrombi formation. Early identification and targeted interventions are crucial for improving outcomes, with ongoing research focusing on complement blocking therapy and identifying reasons for treatment failure.
EXPERT REVIEW OF HEMATOLOGY
(2021)
Review
Immunology
Hamed Azhdari Tehrani, Maryam Darnahal, Mohammad Vaezi, Shirin Haghighi
Summary: This study reported four cases of COVID-19 patients presenting with symptoms of TTP, with peripheral blood smears showing numerous schistocytes, low ADAMTS13 antigen activity level and elevated antibody level, and positive COVID-19 PCR tests for all patients.
INTERNATIONAL IMMUNOPHARMACOLOGY
(2021)
Article
Allergy
Roxane Labrosse, Ines Boufaied, Benoite Bourdin, Saideep Gona, Haley E. Randolph, Brent R. Logan, Sara Bourbonnais, Chloe Berthe, Wendy Chan, Rebecca H. Buckley, Roberta E. Parrott, Geoffrey D. E. Cuvelier, Neena Kapoor, Sharat Chandra, Blachy J. Davila Saldana, Hesham Eissa, Fred D. Goldman, Jennifer Heimall, Richard O'Reilly, Sonali Chaudhury, Edward A. Kolb, Shalini Shenoy, Linda M. Griffith, Michael Pulsipher, Donald B. Kohn, Luigi D. Notarangelo, Sung-Yun Pai, Morton J. Cowan, Christopher C. Dvorak, Ellie Haddad, Jennifer M. Puck, Luis B. Barreiro, Helene Decaluwe
Summary: Severe combined immunodeficiency (SCID) patients may experience CD4+ T-cell lymphopenia and T-cell exhaustion after transplantation, leading to incomplete immune reconstitution and poor long-term outcomes.
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2023)
Article
Allergy
Christopher C. Dvorak, Elie Haddad, Jennifer Heimall, Elizabeth Dunn, Morton J. Cowan, Sung-Yun Pai, Neena Kapoor, Lisa Forbes Satter, Rebecca H. Buckley, Richard J. O'Reilly, Sharat Chandra, Jeffrey J. Bednarski, Olatundun Williams, Ahmad Rayes, Theodore B. Moore, Christen L. Ebens, Blachy J. Davila Saldana, Aleksandra Petrovic, Deepak Chellapandian, Geoffrey D. E. Cuvelier, Mark T. Vander Lugt, Emi H. Caywood, Shanmuganathan Chandrakasan, Hesham Eissa, Frederick D. Goldman, Evan Shereck, Victor M. Aquino, Kenneth B. Desantes, Lisa M. Madden, Holly K. Miller, Lolie Yu, Larisa Broglie, Alfred Gillio, Ami J. Shah, Alan P. Knutsen, Jeffrey P. Andolina, Avni Y. Joshi, Paul Szabolcs, Malika Kapadia, Caridad A. Martinez, Roberta E. Parrot, Kathleen E. Sullivan, Susan E. Prockop, Roshini S. Abraham, Monica S. Thakar, Jennifer W. Leiding, Donald B. Kohn, Michael A. Pulsipher, Linda M. Griffith, Luigi D. Notarangelo, Jennifer M. Puck
Summary: Shearer et al in 2014 established criteria for the diagnosis and classification of severe combined immunodeficiency (SCID), but due to advancements in screening and genetic sequencing, revision of the criteria was necessary. The PIDTC 2022 Definitions provide a more precise description of SCID and its subtypes, facilitating analysis of SCID characteristics and outcomes.
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2023)
Article
Immunology
Keith Sacco, Hye Sun Kuehn, Tomoki Kawai, Nouf Alsaati, Lauren Smith, Blachy Davila, Vanessa Bundy, Douglas B. Kuhns, Kerry Dobbs, Ottavia Delmonte, Luigi D. Notarangelo, Sergio D. Rosenzweig, Michael D. Keller
Summary: This study describes a rare case of autoimmunity and autoinflammation caused by a heterozygous gain-of-function variant in the IKBKB gene. The variant is associated with impaired cell degradation and increased macrophage activation, providing important insights into a novel clinical phenotype.
JOURNAL OF CLINICAL IMMUNOLOGY
(2023)
Meeting Abstract
Immunology
Lena Winestone, Brent R. Logan, Xuerong Liu, Rebecca H. Buckley, Richard J. O'Reilly, Linda M. Griffith, Jennifer Puck, Christopher C. Dvorak, Morton J. Cowan, Elie Haddad
CLINICAL IMMUNOLOGY
(2023)
Meeting Abstract
Immunology
Monica Thakar, Brent R. Logan, Jennifer Puck, Elizabeth Dunn, Rebecca Buckley, Morton Cowan, Sung-Yun Pai, Jennifer Heimall, Michael Pulsipher, Linda Griffith, Elie Haddad, Christopher C. Dvorak, Luigi Notarangelo
CLINICAL IMMUNOLOGY
(2023)
Article
Medicine, General & Internal
Monica S. Thakar, Brent R. Logan, Jennifer M. Puck, Elizabeth A. Dunn, Rebecca H. Buckley, Morton J. Cowan, Richard J. O'Reilly, Neena Kapoor, Lisa Forbes Satter, Sung-Yun Pai, Jennifer Heimall, Sharat Chandra, Christen L. Ebens, Deepak Chellapandian, Olatundun Williams, Lauri M. Burroughs, Blachy Davila Saldana, Ahmad Rayes, Lisa M. Madden, Shanmuganathan Chandrakasan, Jeffrey J. Bednarski, Kenneth B. Desantes, Geoffrey D. E. Cuvelier, Pierre Teira, Alfred P. Gillio, Hesham Eissa, Alan P. Knutsen, Frederick Goldman, Victor M. Aquino, Evan B. Shereck, Theodore B. Moore, Emi H. Caywood, Mark T. Vander Lugt, Jacob Rozmus, Larisa Broglie, Lolie C. Yu, Ami J. Shah, Jeffrey R. Andolina, Xuerong Liu, Roberta E. Parrott, Jasmeen Dara, Susan Prockop, Caridad A. Martinez, Malika Kapadia, Soma C. Jyonouchi, Kathleen E. Sullivan, Jack J. Bleesing, Sonali Chaudhury, Aleksandra Petrovic, Michael Keller, Troy C. Quigg, Suhag Parikh, Shalini Shenoy, Christine Seroogy, Tamar Rubin, Helene Decaluwe, John M. Routes, Troy R. Torgerson, Jennifer W. Leiding, Michael A. Pulsipher, Donald B. Kohn, Linda M. Griffith, Elie Haddad, Christopher C. Dvorak, Luigi D. Notarangelo
Summary: This study found that population-based newborn screening plays a crucial role in early detection and prompt treatment of SCID. The overall 5-year survival rate has significantly increased with the implementation of newborn screening, and the promotion of this screening method can benefit public health programs globally.
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
Natasha N. Frederick, James L. Klosky, Lillian Meacham, Gwendolyn P. Quinn, Joanne F. Kelvin, Brooke Cherven, David R. Freyer, Christopher C. Dvorak, Julienne Brackett, Sameeya Ahmed-Winston, Elyse Bryson, H. Irene Su, Eric J. Chow, Jennifer Levine
Summary: This study investigated the fertility discussion practices in 220 Children's Oncology Group member institutions. It found that fertility discussions were not routine and males were more likely to be offered appropriate options compared to females. There were no specific criteria for offering fertility preservation options for females, while there were clearer criteria for males.
JCO ONCOLOGY PRACTICE
(2023)
Article
Medicine, General & Internal
Troy Yi, Jeffrey Steinberg, Scott Olson, Howaida El-Said, Jun Mo, Eric Anderson, Nicholas Gloude, Deborah Schiff
Summary: HV-LPD is a rare cutaneous disease caused by chronic active Epstein-Barr virus, characterized by sun-induced vesicular lesions. Arterial aneurysm is a rare but potentially fatal complication of CAEBV and HV-LPD.
CLINICAL CASE REPORTS
(2023)
Article
Oncology
Michelle L. L. Schoettler, Christopher E. E. Dandoy, Anora Harris, Marilynn Chan, Keiko M. M. Tarquinio, Sonata Jodele, Muna Qayed, Benjamin Watkins, Pradip Kamat, Toni Petrillo, Jeremy Obordo, Christine S. S. Higham, Christopher C. C. Dvorak, Adrianna Westbrook, Matt S. S. Zinter, Kirsten M. M. Williams
Summary: This study evaluated the effect of different treatments on the outcomes of DAH patients and analyzed data from consecutive HCT patients from January 2018 to August 2022. The study found that using steroids, inhaled tranexamic acid, and inhaled recombinant activated factor VII can reduce the risk of death. Prospective studies are needed to validate these findings.
FRONTIERS IN ONCOLOGY
(2023)
Article
Hematology
Jeffery J. Auletta, Jianqun Kou, Min Chen, Yung-Tsi Bolon, Larisa Broglie, Caitrin Bupp, Debra Christianson, Rachel N. Cusatis, Steven M. Devine, Mary Eapen, Mehdi Hamadani, Mary Hengen, Stephanie J. Lee, Amy Moskop, Kristin M. Page, Marcelo C. Pasquini, Waleska S. Perez, Rachel Phelan, Marcie L. Riches, J. Douglas Rizzo, Wael Saber, Stephen R. Spellman, Heather E. Stefanski, Patricia Steinert, Eileen Tuschl, Rafeek Yusuf, Mei-Ji Zhang, Bronwen E. Shaw
Summary: The use of HLA-mismatched donors could increase access to allogeneic hematopoietic cell transplantation for ethnically diverse patients in the United States. This study aimed to determine if the use of mismatched donor platforms and novel GVHD prophylaxis regimens have improved outcomes for diverse patients, and if the outcomes are comparable to those of non-Hispanic White patients.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Article
Hematology
Tristan E. Knight, Kwang Woo Ahn, Kyle M. Hebert, Rasha Atshan, Donna A. Wall, Kanhatai Chiengthong, Seth J. Rotz, Ellen Fraint, Hemalatha G. Rangarajan, Jeffery J. Auletta, Akshay Sharma, Carrie L. Kitko, Hasan Hashem, Kirsten M. Williams, Baldeep Wirk, Christopher C. Dvorak, Kasiani C. Myers, Michael A. Pulsipher, Anne B. Warwick, Nahal Rose Lalefar, Kirk R. Schultz, Muna Qayed, Larisa Broglie, Mary Eapen, Gregory A. Yanik
Summary: Consolidation with autologous hematopoietic stem cell transplantation (HSCT) has improved survival for patients with central nervous system tumors (CNSTs). This study analyzed the relationship between the CD34(+) dose and clinical outcomes, and found that increasing CD34(+) cell dose was associated with significantly improved overall survival (OS), progression-free survival (PFS), and lower relapse rates in children undergoing autologous HSCT for CNSTs.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Article
Hematology
Brian H. Johnstone, John R. Woods, W. Scott Goebel, Dongsheng Gu, Chieh-Han Lin, Hannah M. Miller, Kelsey G. Musall, Aubrey M. Sherry, Barbara J. Bailey, Emily Sims, Anthony L. Sinn, Karen E. Pollok, Stephen Spellman, Jeffery J. Auletta, Erik J. Woods
Summary: Despite the availability of graft sources for allogeneic hematopoietic cell transplantation, there is still a significant shortage of suitable unrelated donor grafts. Obtaining hematopoietic progenitor cells from vertebral bodies of deceased organ donors could provide a solution to the obstacles associated with using grafts from living donors or umbilical cord blood. Cryopreserved bone marrow hematopoietic progenitor cells from deceased donors have been found to be equivalent in quality and function to those from living donors, making them a viable graft source for clinical transplantation.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Article
Hematology
Fareed Khawaja, Genovefa Papanicolaou, Sanjeet Dadwal, Steven A. Pergam, John R. Wingard, Zeinab El Boghdadly, Maheen Z. Abidi, Alpana Waghmare, Zainab Shahid, Laura Michaels, Joshua A. Hill, Mini Kamboj, Michael Boeckh, Jeffery J. Auletta, Roy F. Chemaly
Summary: COVID-19 disproportionately affects immunocompromised and elderly patients. Hematopoietic cell transplantation and CAR T-cell recipients may have suboptimal immune response to COVID-19 vaccines. Guidelines for optimizing vaccine use in these patients are provided by professional societies.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)