Article
Hematology
Mahasweta Gooptu, Rizwan Romee, Andrew St Martin, Mukta Arora, Monzr Al Malki, Joseph H. Antin, Christopher N. Bredeson, Claudio G. Brunstein, Saurabh Chhabra, Ephraim J. Fuchs, Nilanjan Ghosh, Michael R. Grunwald, Christopher G. Kanakry, Natasha Kekre, Jospeh P. McGuirk, Ian K. McNiece, Rohtesh S. Mehta, Marco Mielcarek, Fillipo Milano, Dipenkumar Modi, Ran Reshef, Scott R. Solomon, Mark A. Schroeder, Edmund K. Waller, Yoshiro Inamoto, Robert J. Soiffer, Mary Eapen
Summary: After comparing outcomes of 2036 haploidentical and 284 matched unrelated donor (MUD) transplantations with posttransplant cyclophosphamide (PTCy) graft-versus-host disease (GVHD) prophylaxis, it was found that MUD transplants had lower acute GVHD and nonrelapse mortality rates, as well as higher disease-free and overall survival, especially in recipients of reduced-intensity regimens. However, no significant differences were observed in patients receiving myeloablative conditioning regimens, highlighting the importance of donor-recipient HLA matching for allogeneic transplantation.
Article
Immunology
Fumiya Wada, Junya Kanda, Satoshi Yoshioka, Takayuki Ishikawa, Takashi Akasaka, Yasunori Ueda, Hirokazu Hirata, Yasuyuki Arai, Kazuhiro Yago, Naoyuki Anzai, Mitsumasa Watanabe, Takashi Ikeda, Akihito Yonezawa, Kazunori Imada, Mitsuru Itoh, Toshiyuki Kitano, Tomoharu Takeoka, Masakatsu Hishizawa, Masaharu Nohgawa, Nobuyoshi Arima, Kousuke Asagoe, Tadakazu Kondo, Akifumi Takaori-Kondo
Summary: Unrelated cord blood (UCB) and haploidentical related donor transplantation using posttransplant cyclophosphamide (PTCy-haplo) are alternative options for treating patients without a HLA-matched donor. This study compared the outcomes of UCB and PTCy-haplo transplantation in hematological malignancy patients and found that overall survival was comparable between the two groups. Neutrophil and platelet engraftment were delayed in the UCB group, but nonrelapse mortality risk and graft-versus-host disease incidence were similar between the groups. UCB transplantation showed a lower relapse rate in acute myeloid leukemia patients.
Article
Immunology
Motoi Yamashita, Shiori Eguchi, Dan Tomomasa, Takahiro Kamiya, Daiki Niizato, Noriko Mitsuiki, Takeshi Isoda, Hanako Funakoshi, Yuki Mizuno, Kentaro Okamoto, Tuan Minh Nguyen, Hidetoshi Takada, Masatoshi Takagi, Kohsuke Imai, Tomohiro Morio, Hirokazu Kanegane
Summary: Leukocyte adhesion deficiency type I (LAD-I) is a rare genetic immune disorder that can be cured with allogeneic hematopoietic cell transplant (HCT). In cases where ideal donors are unavailable, HLA-haploidentical HCT with posttransplant cyclophosphamide (PT-CY) can be a safe and effective alternative.
FRONTIERS IN IMMUNOLOGY
(2022)
Review
Hematology
Leonardo Javier Arcuri, Andreza Alice Feitosa Ribeiro, Nelson Hamerschlak, Mariana Nassif Kerbauy
Summary: Posttransplant cyclophosphamide (PTCy) has revolutionized haploidentical (Haplo) hematopoietic cell transplantation (HCT), showing comparable overall survival and lower incidences of graft-versus-host disease (GVHD) compared to unrelated donor (URD) with conventional GVHD prophylaxis. This raises the question of whether PTCy can be successfully incorporated into matched related donor (MRD) and URD HCT. This review discusses different approaches of using PTCy and its efficacy in various transplantation settings.
ANNALS OF HEMATOLOGY
(2023)
Article
Biophysics
Junichi Sugita, Tomohiko Kamimura, Takayuki Ishikawa, Shuichi Ota, Tetsuya Eto, Takashi Kuroha, Yasuhiko Miyazaki, Hiroaki Kumagai, Keitaro Matsuo, Koichi Akashi, Shuichi Taniguchi, Mine Harada, Takanori Teshima
Summary: The study showed that 80 mg/kg of posttransplant cyclophosphamide is a safe and effective option in HLA-haploidentical peripheral blood stem cell transplantation following reduced-intensity conditioning, leading to successful neutrophil engraftment and reducing the incidence of acute and chronic GVHD.
BONE MARROW TRANSPLANTATION
(2021)
Article
Hematology
Benedetta Rambaldi, Haesook T. Kim, Carol Reynolds, Sharmila Chamling Rai, Yohei Arihara, Tomohiro Kubo, Leutz Buon, Mahasweta Gooptu, John Koreth, Corey Cutler, Sarah Nikiforow, Vincent T. Ho, Edwin P. Alyea, Joseph H. Antin, Catherine J. Wu, Robert J. Soiffer, Jerome Ritz, Rizwan Romee
Summary: Administration of posttransplant cyclophosphamide has increased the number of patients undergoing HLA-haploidentical hematopoietic cell transplantation. Immune reconstitution in haplo-HCT patients showed delayed T-cell recovery skewed towards effector memory T cells and higher regulatory T cell ratios early after transplant. NK cell reconstitution also differed, with increased immature NK cells and enrichment of specific NK cell metadusters after haplo-HCT.
Article
Hematology
Ephraim J. Fuchs, Shannon R. McCurdy, Scott R. Solomon, Tao Wang, Megan R. Herr, Dipenkumar Modi, Michael R. Grunwald, Taiga Nishihori, Michelle Kuxhausen, Stephanie Fingerson, Caroline McKallor, Asad Bashey, Yvette L. Kasamon, Yung-Tsi Bolon, Ayman Saad, Joseph McGuirk, Sophie Paczesny, Shahinaz M. Gadalla, Steven G. E. Marsh, Bronwen E. Shaw, Stephen R. Spellman, Stephanie J. Lee, Effie W. Petersdorf
Summary: HLA factors play an important role in the success of haploidentical transplantation. Disease-free survival is associated with matching status at individual HLA loci, with HLA-DRB1 and -DPB1 mismatches and HLA-C,-B leader, and -DQB1 matching being favorable. Considering HLA factors may help optimize the selection of haploidentical related donors.
Article
Cell & Tissue Engineering
Fumiya Wada, Junya Kanda, Kimimori Kamijo, Masashi Nishikubo, Satoshi Yoshioka, Takayuki Ishikawa, Yasunori Ueda, Takashi Akasaka, Yasuyuki Arai, Kiyotaka Izumi, Hirokazu Hirata, Takashi Ikeda, Akihito Yonezawa, Naoyuki Anzai, Mitsumasa Watanabe, Kazunori Imada, Kazuhiro Yago, Naoki Tamura, Mitsuru Itoh, Yuki Masuo, Akane Kunitomi, Tomoharu Takeoka, Toshiyuki Kitano, Nobuyoshi Arima, Masakatsu Hishizawa, Kohsuke Asagoe, Tadakazu Kondo, Akifumi Takaori-Kondo, Kyoto Stem Cell Transplantat Grp
Summary: Haploidentical-related donor transplantation and cord blood transplantation are valid alternatives when HLA-matched donor transplantation is unavailable for patients with hematological malignancies. However, the effects of graft-versus-host disease on outcomes after these transplants have not been fully elucidated. This study evaluated the effects of acute and chronic GVHD on transplant outcomes after PTCy-haplo transplants and compared them with CBT and MDT.
CELL TRANSPLANTATION
(2023)
Article
Hematology
Daishi Nakagawa, Yoshimitsu Shimomura, Satoshi Mitsuyuki, Tomoyo Kubo, Masashi Nishikubo, Naoki Okada, Kimimori Kamijo, Ryusuke Yamamoto, Yuya Nagai, Nobuhiro Hiramoto, Satoshi Yoshioka, Noboru Yonetani, Takayuki Ishikawa
Summary: Prophylactic letermovir effectively prevents cytomegalovirus reactivation in patients undergoing haplo-HCT with posttransplant cyclophosphamide. The 100-day cumulative incidence of CMV reactivation was significantly lower in the letermovir group compared to the control group.
INTERNATIONAL JOURNAL OF HEMATOLOGY
(2023)
Article
Hematology
Viviane Jesus Torres de Lima, Anderson Felipe da Silva, Lucila Nassif Kerbauy, Mariana Nassif Kerbauy, Decio Lerner, Marta Colares, Andreza Alice Feitosa Ribeiro, Cinthya Feitosa da Silva, Nelson Hamerschlak, Leonardo Javier Arcuri
Summary: This study investigates risk factors for adverse outcomes after haplo-HCT with PTCy and finds that age, disease risk index, and donor selection are important factors affecting patient survival, progression-free survival, and relapse rates. Additionally, the study suggests that tacrolimus may be more effective than cyclosporine in preventing GVHD, while peripheral blood stem cells increase the risk of chronic GVHD.
ANNALS OF HEMATOLOGY
(2022)
Article
Biophysics
Max J. Rieger, Sebastian M. Stolz, Antonia M. Mueller, Rahel Schwotzer, Gayathri Nair, Dominik Schneidawind, Markus G. Manz, Urs Schanz
Summary: Hematopoietic cell transplantation from haploidentical donors (haploHCT) has proven to be a feasible and effective treatment for AML and MDS. The overall survival and progression-free survival rates are similar to those of HLA-identical MRD and MUD transplants, but haploHCT is associated with higher non-relapse mortality due to fatal infections.
BONE MARROW TRANSPLANTATION
(2023)
Article
Immunology
Paula Muniz, Mi Kwon, Diego Carbonell, Maria Chicano, Rebeca Bailen, Gillen Oarbeascoa, Julia Suarez-Gonzalez, Cristina Andres-Zayas, Javier Menarguez, Nieves Dorado, Ignacio Gomez-Centurion, Javier Anguita, Jose Luis Diez-Martin, Carolina Martinez-Laperche, Ismael Buno
Summary: HLA loss may be a mechanism of relapse after Haplo-HSCT, more common in female patients. Relapse with HLA loss occurs later, with more active disease and treatment history, requiring different treatment strategies.
FRONTIERS IN IMMUNOLOGY
(2021)
Article
Hematology
Amy E. DeZern, Marianna Zahurak, Heather J. Symons, Kenneth R. Cooke, Carol Ann Huff, Tania Jain, Lode J. Swinnen, Philip H. Imus, Nina D. Wagner-Johnston, Richard F. Ambinder, Mark Levis, Leo Luznik, Javier Bolanos-Meade, Ephraim J. Fuchs, Richard J. Jones, Robert A. Brodsky
Summary: Severe aplastic anemia (SAA) is a marrow failure disorder with high morbidity and mortality. This study found that reduced-intensity conditioning HLA-haploidentical bone marrow transplantation (BMT) with posttransplantation cyclophosphamide (PTCy)-based graft-versus-host (GVHD) prophylaxis resulted in 100% overall survival and minimal GVHD in patients with SAA. This approach not only avoids the adverse effects of immunosuppressive therapy, but also expands access to BMT across all populations.
Article
Hematology
Luisa Strocchio, Daria Pagliara, Mattia Algeri, Giuseppina Li Pira, Francesca Rossi, Valentina Bertaina, Giovanna Leone, Rita Maria Pinto, Marco Andreani, Emanuele Agolini, Katia Girardi, Stefania Gaspari, Lavinia Grapulin, Francesca del Bufalo, Antonio Novelli, Pietro Merli, Franco Locatelli
Summary: The use of HLA-haploidentical T-cell receptor and CD19(+) cell-depleted hematopoietic stem cell transplantation for Fanconi anemia patients resulted in high success rates, low incidences of acute and chronic graft-versus-host disease, and good overall survival rates.
Article
Biophysics
Michael H. Albert, Mehtap Sirin, Manfred Hoenig, Fabian Hauck, Catharina Schuetz, Rajat Bhattacharyya, Polina Stepensky, Elad Jacoby, Tayfun Gungor, Rita Beier, Ansgar Schulz
Summary: The study found that a post-transplantation cyclophosphamide-based HLA-haploidentical salvage HSCT after irradiation-free conditioning resulted in excellent engraftment and overall survival in children with non-malignant diseases.
BONE MARROW TRANSPLANTATION
(2021)
