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EDITORIAL
Didier Blaise
No abstract text is available yet for this article.
April 25, 2024: New England Journal of Medicine
#2
JOURNAL ARTICLE
Yongxian Hu, Mingming Zhang, Tingting Yang, Zhuomao Mo, Guoqing Wei, Ruirui Jing, Houli Zhao, Rongrong Chen, Cheng Zu, Tianning Gu, Pingnan Xiao, Ruimin Hong, Jingjing Feng, Shan Fu, Delin Kong, Huijun Xu, Jiazhen Cui, Simao Huang, Bin Liang, Xiaolin Yuan, Qu Cui, Hongshan Guo, Yunxian Yu, Youqin Feng, Chunxiang Jin, Jiangtao Ren, Alex H Chang, Dongrui Wang, He Huang
BACKGROUND: Patients with relapsed or refractory hematologic cancers have a poor prognosis. Chimeric antigen receptor (CAR) T-cell therapy as a bridge to allogeneic hematopoietic stem-cell transplantation (HSCT) has the potential for long-term tumor elimination. However, pre-HSCT myeloablation and graft-versus-host disease (GVHD) prophylaxis agents have toxic effects and could eradicate residual CAR T cells and compromise antitumor effects. Whether the integration of CAR T-cell therapy and allogeneic HSCT can preserve CAR T-cell function and improve tumor control is unclear...
April 25, 2024: New England Journal of Medicine
#3
Bibi Shahin Shamsian, Nader Momtazmanesh, Hedyeh Saneifard, Seyed Mohammad Taghi Hosseini Tabatabaei, Mohammadreza Jafari, Zahra Khafaf Pour, Kawthar Jasim Mohammad Rida Al-Hussieni, Mahnaz Jamee, Sharareh Kamfar
BACKGROUND: Osteopetrosis is a group of geneticall heterogeneous disorders resulting from impaired osteoclast function and bone resorption. The identification of specific genetic mutations can yield important prognostic and therapeutic implications. Herein, we present the diagnosis and successful application of hematopoietic stem cell transplantation (HSCT) in a patient with osteopetrosis caused by carbonic anhydrase II deficiency (Intermediate osteopetrosis). CASE PRESENTATION: Herein, we describe a 2...
May 2024: Pediatric Transplantation
#4
JOURNAL ARTICLE
Ariadna Pérez, Juan Montoro, Pedro Chorão, Dolores Gómez, Manuel Guerreiro, Estela Giménez, Marta Villalba, Jaime Sanz, Rafael Hernani, Juan Carlos Hernández-Boluda, Ignacio Lorenzo, David Navarro, Carlos Solano, Per Ljungman, José Luis Piñana
BACKGROUND: This retrospective study focused on analyzing community-acquired respiratory virus (CARV) infections, in particular human parainfluenza virus (hPIV) after allogeneic stem cell transplant (allo-SCT) in adults recipients. It aimed to assess the impact of ribavirin treatment, clinical characteristics, and risk factors associated with lower respiratory tract disease (LRTD) progression and all-cause mortality. PATIENTS AND METHODS: The study included 230 allo-SCT recipients diagnosed with hPIV between December 2013 and June 2023...
April 24, 2024: Infection
#5
REVIEW
Navigating the Tumor Microenvironment: Mesenchymal Stem Cell-Mediated Delivery of Anticancer Agents.
Muhammad Ahsan Waqar, Muhammad Zaman, Muhammad ShafeeqUrRahman, Rabeel Khan, Imtiaz Majeed
Scientific knowledge of cancer has advanced greatly throughout the years, with most recent studies findings includes many hallmarks that capture disease's multifaceted character. One of the novel approach utilized for the delivery of anti-cancer agents includes mesenchymal stem cell mediated drug delivery. Mesenchymal stem cells (MSCs) are non-hematopoietic progenitor cells that may be extracted from bone marrow, tooth pulp, adipose tissue and placenta/umbilical cord blood dealing with adult stem cells. MSCs are mostly involved in regeneration of tissue, they have also been shown to preferentially migrate to location of several types of tumor in-vivo ...
April 23, 2024: Journal of Drug Targeting
#6
JOURNAL ARTICLE
Meziane Brizini, Bruno Michon, Marc-Antoine Bédard
Multiple myeloma is a rare disease in pediatrics, where about 30 cases are described under 15 years old. It is even rarer when atypical multiple myeloma occurs in the context of autoimmunity. This case describes a 9-year-old female with autoimmune lymphoproliferative-like disease and combined immune deficiency that developed acute kidney failure with monoclonal peak associated with RAC2 and TNFRSF9 variants. An adapted protocol from the backbone adult multiple myeloma standard of care with the addition of an allogeneic hematopoietic stem cell transplant was used...
April 23, 2024: Journal of Pediatric Hematology/oncology
#7
JOURNAL ARTICLE
Vasil Toskov, Annamaria Cseh, Alexander Claviez, Beatrice Drexler, Natalia Rotari, Stephan Schwarz-Furlan, Matthias Braun, Peter Bader, Peter Lang, Rita Beier, Bernhard Erdlenbruch, Monika Führer, Miriam Erlacher, Charlotte M Niemeyer, Brigitte Strahm, Ayami Yoshimi
We report 5 children with bone marrow failure (BMF) after primary varicella zoster virus (VZV) infection or VZV vaccination, highlighting the highly variable course. Two patients were treated with intravenous immunoglobulins; one had a slow hematologic recovery, and the other was rescued by allogeneic hematopoietic stem cell transplantation (HSCT). Of the 2 patients treated with immunosuppressive therapy with antithymocyte globulin and cyclosporine, one had a complete response, and the other was transplanted for nonresponse...
April 23, 2024: Journal of Pediatric Hematology/oncology
#8
REVIEW
Hongtao Li, Yaxin Zheng, Kehai Gao, Chen Tian
To evaluate whether patients with multiple myeloma (MM) could benefit from tandem autologous hematopoietic stem cell transplantation (auto-HSCT), PubMed, Embase, Web of Science and Cochrane Library databases were systematically searched, and 10 eligible studies were included after data extraction and quality evaluation. Meta-analysis showed that compared to single autologous hematopoietic stem cell transplantation, tandem auto-HSCT does not improve OS, EFS or efficacy in MM patients, and may even lead to higher treatment-related mortality (TRM)...
December 2024: Hematology (Amsterdam, Netherlands)
#9
JOURNAL ARTICLE
Bianca Serio, Gabriella Storti, Matteo D'Addona, Lidia Santoro, Camilla Frieri, Danilo De Novellis, Luana Marano, Giovanna De Santis, Roberto Guariglia, Ilenia Manfra, Eleonora Urciuoli, Serena Luponio, Serena Marotta, Denise Morini, Michela Rizzo, Fausto Palmieri, Nicola Cantore, Valentina Giudice, Antonio Maria Risitano, Carmine Selleri
Background: Post-transplant cyclophosphamide (PTCY) is widely used as graft versus host disease (GvHD) prophylaxis in allogeneic hematopoietic stem cell transplant (HSCT) recipients, with reported clinical benefits in patients who underwent transplant from a matched unrelated donor (MUD). However, real-life data on clinical efficacy and safety of PTCY in haploidentical and MUD transplantations are still poor. Methods: In our real-life retrospective observational study, we included a total of 40 consecutive adult patients who underwent haploidentical or MUD HSCT for various hematological malignancies and who received PTCY ( n = 24) or ATG ( n = 16) as GvHD prophylaxis at Hematology Units from hospitals of Salerno and Avellino, Italy, and clinical outcomes were compared...
April 17, 2024: Hematology Reports
#10
REVIEW
Beatrice Xuan Ho, Adrian Kee Keong Teo, Natasha Hui Jin Ng
Human allogeneic pancreatic islet transplantation is a life-changing treatment for patients with severe Type 1 Diabetes (T1D) who suffer from hypoglycemia unawareness and high risk of severe hypoglycemia. However, intensive immunosuppression is required to prevent immune rejection of the graft, that may in turn lead to undesirable side effects such as toxicity to the islet cells, kidney toxicity, occurrence of opportunistic infections, and malignancies. The shortage of cadaveric human islet donors further limits islet transplantation as a treatment option for widespread adoption...
2024: Frontiers in Immunology
#11
JOURNAL ARTICLE
Yahan Li, Fansheng Kong, Guanchen Bai, Yujie Jiang, Wenlu Zhang, Xue Sun, Xiaohui Sui, Ying Li, Mei Ding, Dai Yuan, Xin Wang, Xiaosheng Fang
BACKGROUND: Eltrombopag has demonstrated efficacy in treating low platelet (PLT) levels, but it remains unclear whether eltrombopag can promote PLT engraftment after hematopoietic stem cell transplantation (HSCT). METHODS: Forty-one HSCT patients received eltrombopag 50 mg/d from +1 day until PLT >50 × 109 /L or 1 month after HSCT. Fifty-one patients in the same period received thrombopoietin (TPO) to promote PLT graft after HSCT and served as a control group...
2024: Frontiers in Immunology
#12
JOURNAL ARTICLE
Ivan Pasic, Tommy Alfaro Moya, Mats Remberger, Carol Chen, Armin Gerbitz, Dennis Dong Hwan Kim, Rajat Kumar, Wilson Lam, Arjun Datt Law, Jeffrey H Lipton, Fotios V Michelis, Igor Novitzky-Basso, Auro Viswabandya, Jonas Mattsson
Treosulfan has shown promise in allogeneic hematopoietic cell transplantation (HCT) for its myeloablative properties and low toxicity. In this single-center retrospective propensity score-matched cohort study we compared treosulfan- and busulfan-based conditioning in allogeneic HCT for patients with myelodysplastic syndrome (MDS). This study included 138 adults who underwent allogeneic HCT for MDS or chronic myelomonocytic leukemia (CMML) at Princess Margaret Hospital, Toronto 2015-2022. Using propensity score matching, we compared transplant outcomes between two well-matched cohorts who received conditioning with either fludarabine-treosulfan (FT) (n=46) or fludarabine-busulfan with total body irradiation (FBT200) (n=92)...
April 20, 2024: Transplantation and cellular therapy
#13
REVIEW
Agnieszka Piekarska, Katarzyna Pawelec, Anna Szmigielska-Kapłon, Marek Ussowicz
Acquired aplastic anemia (AA) is an immune-mediated bone marrow (BM) failure where marrow disruption is driven by a cytotoxic T-cell-mediated autoimmune attack against hematopoietic stem cells. The key diagnostic challenge in children, but also in adults, is to exclude the possible underlying congenital condition and myelodysplasia. The choice of treatment options, either allogeneic hematopoietic cell transplantation (alloHCT) or immunosuppressive therapy (IST), depends on the patient's age, comorbidities, and access to a suitable donor and effective therapeutic agents...
2024: Frontiers in Immunology
#14
REVIEW
Chi-Cheng Li, Xavier Cheng-Hong Tsai, Wei-Han Huang, Tso-Fu Wang
Hematopoietic stem cell transplantation (HSCT) can cure malignant and nonmalignant hematological disorders. From 1983 to 2022, Taiwan performed more than 10,000 HSCT transplants. The Taiwan Blood and Marrow Transplantation Registry collects clinical information to gather everyone's experience and promote the advances of HSCT in Taiwan to gather everyone's experience and promote advances of HSCT in Taiwan. Compared with matched sibling donors, transplants from matched unrelated donors exhibited a trend of superior survival...
2024: Tzu chi medical journal
#15
John Donald Marra, Eugenio Galli, Sabrina Giammarco, Elisabetta Metafuni, Gessica Minnella, Federica Fosso, Sara Marietti, Simona Sica, Federica Sorà, Patrizia Chiusolo
While there is intense interest in the production of allogeneic CAR-T cells from umbilical cord units, little is known about the reactivity and persistence of CAR-T cells of umbilical origin. We report the case of a patient at our hematological center with multiple relapsing Ph+ B-ALL, notably a Blinatunomab non-responder, who underwent therapy with Brexucabtagene Autoleucel following relapse on Ponatinib post-allogeneic hematopoietic stem cell transplantation. The patient achieved a rapid CAR-T expansion and durable remission presenting in good clinical conditions 6 months post-CAR-T infusion, without manifestations of graft-versus-host disease...
April 21, 2024: European Journal of Haematology
#16
REVIEW
Tahereh Rostami, Mohammad Reza Rostami, Amir Hossein Mirhosseini, Saeed Mohammadi, Mohsen Nikbakht, Hediyeh Alemi, Naghmeh Khavandgar, Soroush Rad, Ghasem Janbabai, Seied Asadollah Mousavi, Azadeh Kiumarsi, Amir Kasaeian
BACKGROUND: Graft failure (GF) is a rare but serious complication after allogeneic hematopoietic stem cell transplantation (HSCT). Prevention of graft failure remains the most advisable approach as there is no clear recommendation for the best strategies for reversing this complication. Administration of growth factor, additional hematopoietic progenitor boost, or a salvage HSCT are current modalities recommended for the treatment of GF. Autologous recovery without evidence of disease relapse occurs rarely in patients with GF, and in the absence of autologous recovery, further salvage transplantation following a second conditioning regimen is a potential treatment option that offers the best chances of long-term disease-free survival...
April 22, 2024: Stem Cell Research & Therapy
#17
JOURNAL ARTICLE
Glenda M Davison, Jessica J Opie, Saarah F G Davids, Rygana Mohammed, Nicolas Novitzky
BACKGROUND: Allogeneic stem cell transplantation (SCT) is a critical therapy for haematological malignancy but may lead to acute and chronic graft versus host disease (GvHD). T-cell depletion with alemtuzumab, either in vivo or ex vivo, reduces the incidence of GvHD but is a risk factor for disease relapse and poor immune reconstitution. Natural killer (NK) cells are the first lymphocytes to recover. Classical NK cells make up >90% of the normal circulating population and can directly kill neoplastic or virally infected cells while the regulatory subset makes up <10%, secretes cytokines and is not cytotoxic...
April 17, 2024: Transplant Immunology
#18
JOURNAL ARTICLE
Takaaki Konuma, Kotaro Miyao, Hideki Nakasone, Fumihiko Ouchi, Takahiro Fukuda, Masatsugu Tanaka, Yukiyasu Ozawa, Shuichi Ota, Toshiro Kawakita, Naoyuki Uchida, Masashi Sawa, Yuta Katayama, Nobuhiro Hiramoto, Tetsuya Eto, Tatsuo Ichinohe, Yoshiko Atsuta, Junya Kanda
BACKGROUND: Mobilized peripheral blood stem cells (PBSC) have been widely used instead of bone marrow (BM) as the graft source for allogeneic hematopoietic cell transplantation (HCT). Although early studies demonstrated no significant differences in survival between PBSC transplantation (PBSCT) and BM transplantation (BMT) from human leukocyte antigen (HLA)-identical sibling donors to adults with hematological malignancies, recent results have been unclear. OBJECTIVE: The objective of this retrospective study was to compare overall survival (OS), relapse, non-relapse mortality (NRM), hematopoietic recovery and graft-versus-host disease (GVHD) between PBSCT and BMT according to the time period of HCT (2003-2008, 2009-2014, or 2015-2020)...
March 30, 2024: Cytotherapy
#19
JOURNAL ARTICLE
Sara Torres-Esquius, Francisco Beas, Tzu Hua Chen-Liang, Helena Pomares, Marta Santiago, Nicolás Díaz Varela, Alessandro Liquori, Francisca Hernandez, Blanca Xicoy, Lourdes Hermosín, Montserrat Arnan, Bárbara Tazón-Vega, Adoración Blanco, José Cervera, María Diez-Campelo, María Luisa Lozano, David Valcárcel, Francesc Bosch, Maria Julia Montoro, Andrés Jerez
Patients aged 50 or above diagnosed with myeloid neoplasms (MNs) are typically not candidates for germline testing. However, approximately 8% carry pathogenic germline variants. Allogeneic haematopoietic stem cell transplantation (alloHSCT) remains an option for those aged over 50; neglecting germline testing could mask the risk for relative donor cell-derived MN. We propose a germline-augmented somatic panel (GASP), combining MN predisposition genes with a myeloid somatic panel for timely germline variant identification when initial testing is not indicated...
April 19, 2024: British Journal of Haematology
#20
MULTICENTER STUDY
Michael D Keller, Patrick J Hanley, Yueh-Yun Chi, Paibel Aguayo-Hiraldo, Christopher C Dvorak, Michael R Verneris, Donald B Kohn, Sung-Yun Pai, Blachy J Dávila Saldaña, Benjamin Hanisch, Troy C Quigg, Roberta H Adams, Ann Dahlberg, Shanmuganathan Chandrakasan, Hasibul Hasan, Jemily Malvar, Mariah A Jensen-Wachspress, Christopher A Lazarski, Gelina Sani, John M Idso, Haili Lang, Pamela Chansky, Chase D McCann, Jay Tanna, Allistair A Abraham, Jennifer L Webb, Abeer Shibli, Amy K Keating, Prakash Satwani, Pawel Muranski, Erin Hall, Michael J Eckrich, Evan Shereck, Holly Miller, Ewelina Mamcarz, Rajni Agarwal, Satiro N De Oliveira, Mark T Vander Lugt, Christen L Ebens, Victor M Aquino, Jeffrey J Bednarski, Julia Chu, Suhag Parikh, Jennifer Whangbo, Michail Lionakis, Elias T Zambidis, Elizabeth Gourdine, Catherine M Bollard, Michael A Pulsipher
Viral infections remain a major risk in immunocompromised pediatric patients, and virus-specific T cell (VST) therapy has been successful for treatment of refractory viral infections in prior studies. We performed a phase II multicenter study (NCT03475212) for the treatment of pediatric patients with inborn errors of immunity and/or post allogeneic hematopoietic stem cell transplant with refractory viral infections using partially-HLA matched VSTs targeting cytomegalovirus, Epstein-Barr virus, or adenovirus...
April 18, 2024: Nature Communications
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