Article
Oncology
Bhagirathbhai Dholaria, Myriam Labopin, Jaime Sanz, Annalisa Ruggeri, Jan Cornelissen, Helene Labussiere-Wallet, Didier Blaise, Edouard Forcade, Patrice Chevallier, Anna Grassi, Ludmila Zubarovskaya, Jurgen Kuball, Patrice Ceballos, Fabio Ciceri, Frederic Baron, Bipin N. Savani, Arnon Nagler, Mohamad Mohty
Summary: This study compared the outcomes of CBT and single-allele-mismatched MMUD allo-HCT with PTCy in acute myeloid leukemia. The results showed that CBT was associated with higher incidence of graft failure and non-relapse mortality compared to MMUD, leading to lower leukemia-free survival, overall survival, and GVHD-free, relapse-free survival. In conclusion, MMUD allo-HCT with PTCy may be a preferred choice over CBT for patients without a fully matched donor.
JOURNAL OF HEMATOLOGY & ONCOLOGY
(2021)
Article
Medicine, General & Internal
Theresa Hahn, Junke Wang, Leah M. Preus, Ezgi Karaesmen, Abbas Rizvi, Alyssa Clay-Gilmour, Qianqian Zhu, Yiwen Wang, Li Yan, Song Liu, Daniel O. Stram, Loreall Pooler, Xin Sheng, Christopher A. Haiman, David Van den Berg, Amy Webb, Guy Brock, Stephen R. Spellman, Kenan Onel, Philip L. McCarthy, Marcelo C. Pasquini, Lara E. Sucheston-Campbell
Summary: Through genome-wide analysis, novel non-HLA genetic loci were found to be associated with 1-year survival after unrelated donor BMT.
Article
Hematology
Mahmoud R. Gaballa, Pinaki Banerjee, Denai R. Milton, Xianli Jiang, Christina Ganesh, Sajad Khazal, Vandana Nandivada, Sanjida Islam, Mecit Kaplan, May Daher, Rafet Basar, Amin Alousi, Rohtesh Mehta, Gheath Alatrash, Issa Khouri, Betul Oran, David Marin, Uday Popat, Amanda Olson, Priti Tewari, Nitin Jain, Elias Jabbour, Farhad Ravandi, Hagop Kantarjian, Ken Chen, Richard Champlin, Elizabeth Shpall, Katayoun Rezvani, Partow Kebriaei
Summary: This study examined the feasibility and efficacy of blinatumomab in high-risk acute lymphoblastic leukemia patients after allogeneic hematopoietic cell transplantation. The results showed that blinatumomab treatment was feasible and effective in reducing the risk of relapse, with specific T-cell profiles associated with treatment response.
Article
Oncology
Mahta Mardani, Maryam Behfar, Leila Jafari, Rashin Mohseni, Parisa Naji, Pouria Salajegheh, Ghazaleh Donyadideh, Amir Ali Hamidieh
Summary: This study aimed to compare the transplant outcomes between non-HID and HID transplant in pediatric patients with ALL. The results showed that in the setting of non-TBI conditioning, the PBSC-HID transplant outcomes were comparable to those of non-HIDs in terms of overall survival, incidence of acute and chronic graft-versus-host disease, and relapse rate. However, patients receiving HID transplants had a higher risk of cytomegalovirus infection than non-HIDs.
PEDIATRIC BLOOD & CANCER
(2023)
Review
Oncology
Francesco Tamiro, Andrew P. Weng, Vincenzo Giambra
Summary: Leukemia-initiating cells (LIC) are unique cells in different types of leukemia that have self-renewing capabilities and produce tumors, which are functionally distinct from bulk leukemia cells. Current conventional treatments are not effective in eliminating LICs, hence innovative therapeutics targeting LICs hold promise for developing an effective cure for ALL.
Review
Immunology
Mohammad Hassan Hodroj, Iman Abou Dalle, Nour Moukalled, Jean El Cheikh, Mohamad Mohty, Ali Bazarbachi
Summary: The outcome of B-cell acute lymphoblastic leukemia has improved with multi-agent chemotherapy and immunotherapeutic agents. However, relapse post-transplant is still common. This review discusses strategies such as tyrosine kinase inhibitors, innovative agents, and cellular therapy to prevent and overcome relapse post allo-HCT in ALL patients.
FRONTIERS IN IMMUNOLOGY
(2023)
Article
Hematology
Zaid H. Abdel Rahman, Ricardo D. Parrondo, Michael G. Heckman, Mikolaj Wieczorek, Kevin C. Miller, Hassan Alkhateeb, Lisa Z. Sproat, Hemant Murthy, William J. Hogan, Mohamed A. Kharfan-Dabaja, Jess F. Peterson, Linda B. Baughn, Nicole Hoppman, Mark R. Litzow, Rhett P. Ketterling, Patricia T. Greipp, James M. Foran
Summary: Patients with therapy-related acute lymphoblastic leukaemia (tr-ALL) differed significantly from de novo ALL patients in terms of age, gender, cytogenetic abnormalities, etc. Patients with tr-ALL were less likely to achieve complete remission, but had similar overall survival rates after diagnosis and allogeneic hematopoietic cell transplantation.
BRITISH JOURNAL OF HAEMATOLOGY
(2022)
Article
Oncology
Dominic Schenone, Jeffrey R. Andolina, Brooks Rademacher, Thomas J. Fountaine, Elena Edwards, Leti Nunez, Michelle Qiu, Sonakshi Sharma, Craig A. Mullen
Summary: The study suggests that cytokines and cyclosporine may play a significant role in mediating the graft versus leukemia effect. ALL cells exposed to cytokines and cyclosporine remained susceptible to T and NK cell killing, and also showed increased cell death even without exposure to killer cells.
ANTICANCER RESEARCH
(2021)
Article
Pediatrics
JinFang Zhang, LingJi Zeng, YuLian Wang, JianWei Pan, XingDong Li, Bei Feng, Quan Yang
Summary: The study found that TTN and NOTCH1 gene mutations were associated with glucocorticoid resistance in pediatric acute lymphoblastic leukemia (ALL). Additionally, TTN and MUC16 mutations were associated with a younger age at diagnosis, while NOTCH1 mutations were associated with T-cell ALL in female patients.
FRONTIERS IN PEDIATRICS
(2022)
Article
Pharmacology & Pharmacy
Gabriela Burgueno-Rodriguez, Yessika Mendez, Natalia Olano, Magdalena Schelotto, Luis Castillo, Ana Maria Soler, Julio da Luz
Summary: This study aims to identify genetic variants related to drugs used in the treatment of pediatric acute lymphoblastic leukemia in Uruguay and analyze their contribution to adverse effects. The study found that mucositis, Cushing syndrome, and neurotoxicity were linked to specific genetic variants and individual genetic ancestry.
FRONTIERS IN PHARMACOLOGY
(2023)
Article
Immunology
Edyta Heropolitanska-Pliszka, Barbara Piatosa, Anna Szmydki-Baran, Karolina Kuczborska, Karolina Miarka-Walczyk, Agata Pastorczak, Wojciech Mlynarski, Lukasz Sedek, Tomasz Szczepanski, Marek Ussowicz
Summary: This study reports a successful bone marrow transplantation in a carrier of a novel GATA2 pathogenic variant who had previously been treated for B-cell precursor acute lymphoblastic leukemia (BCP-ALL). The study highlights the importance of genetic counseling and family screening prior to selecting related hematopoietic stem cell transplantation (HSCT) donors.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Oncology
Sebastian Giebel, Myriam Labopin, Urpu Salmenniemi, Gerard Socie, Sergey Bondarenko, Didier Blaise, Nicolaus Kroeger, Jan Vydra, Anna Grassi, Francesca Bonifazi, Tomasz Czerw, Achilles Anagnostopoulos, Bruno Lioure, Annalisa Ruggeri, Bipin Savani, Alexandros Spyridonidis, Jaime Sanz, Zinaida Peric, Arnon Nagler, Fabio Ciceri, Mohamad Mohty
Summary: This study compared the efficacy of using rabbit antithymocyte globulin (ATG) and posttransplant cyclophosphamide (PTCY) as immunosuppressive strategies for prophylaxis of graft-versus-host disease (GVHD) in acute lymphoblastic leukemia (ALL) patients. The study found that while the PTCY group had a reduced risk of chronic GVHD, it also had inferior leukemia-free survival compared to the ATG group.
Article
Cell Biology
Qing Hao, Minyuan Cao, Chunlan Zhang, Dandan Yin, Yuelan Wang, Yuanxin Ye, Shan Zhao, Yunfan Yang, Ke-Ling Chen, Binwu Ying, Lanlan Wang, Yiguan Zhang, Caigang Xu, Yiping Zhu, Yu Wu, Ju Gao, Jun-Ning Zhao, Yan Zhang, Xiaoxi Lu
Summary: This study conducted a genome-wide association study in Chinese patients with ALL to estimate the impact of age on genetic susceptibility, identifying key inherited predisposition loci such as ARID5B and GATA3. The analysis highlighted the differentiation of genetic risk between pediatric and adult patients, providing insights into genetic susceptibility to ALL in Chinese patients.
Article
Hematology
Byung-Sik Cho, Gi-June Min, Silvia Park, Sung-Soo Park, Seung-Hwan Shin, Seung-Ah Yahng, Young-Woo Jeon, Jae-Ho Yoon, Sung-Eun Lee, Ki-Seong Eom, Yoo-Jin Kim, Seok Lee, Chang-Ki Min, Seok-Goo Cho, Dong-Wook Kim, Jong Wook Lee, Myungshin Kim, Yonggoo Kim, Hee-Je Kim
Summary: This prospective study demonstrated the non-inferiority of Haplo-HSCT to MUD-HSCT for AML in remission, and validated the role of WT1 quantification as an MRD marker. Both groups had similar outcomes in terms of overall and disease-free survival, relapse, non-relapse mortality, and GVHD-free/relapse-free survival.
AMERICAN JOURNAL OF HEMATOLOGY
(2021)
Review
Oncology
Cristina Tecchio, Anna Russignan, Mauro Krampera
Summary: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) benefits adult ALL patients, but those with MRD may require interventions with side effects. The significance of MFC in ALL treatment is widely recognized, but there is limited data on MRD monitoring in allo-HSCT settings. This article aims to summarize and discuss the role of MFC detection of MRD in adult ALL patients undergoing allo-HSCT, including sensitivity, timing, and relation to other MRD assessment techniques.
FRONTIERS IN ONCOLOGY
(2023)
Article
Hematology
Erica Brivio, Franco Locatelli, Marta Lopez-Yurda, Andrea Malone, Cristina Diaz-de-Heredia, Bella Bielorai, Claudia Rossig, Vincent H. J. van der Velden, Anneke C. J. Ammerlaan, Adriana Thano, Inge M. van der Sluis, Monique L. den Boer, Ying Chen, Barbara Sleight, Benoit Brethon, Karsten Nysom, Lucie Sramkova, Ingrid Ora, Luciana Vinti, Christiane Chen-Santel, Christian Michel Zwaan
Summary: This phase 1 study investigated the efficacy and safety of inotuzumab ozogamicin in pediatric patients with acute lymphoblastic leukemia, establishing the recommended phase 2 dose as 1.8 mg/m(2). The treatment showed promising efficacy and good tolerability, with potential clinical implications.
Article
Hematology
Juan Torres Canizales, Cristina Ferreras, Antonia Pascual, Laura Alonso, Alexandra Regueiro, Mercedes Plaza, Jose Maria Perez Hurtado, Ana Benito, Jose M. Couselo, Jose L. Fuster, Mariana Diaz-Almiron, David Bueno, Yasmina Mozo, Alicia Gomez Lopez, Jose L. Vicario, Antonio Balas, Luisa Sisinni, Cristina Diaz de Heredia, Antonio Perez-Martinez
Summary: The study retrospectively analyzed 31 haplo-HSCT procedures performed at 7 Spanish centers, showing a 27.4% graft failure rate, 34.6% acute GvHD rate, 16.7% chronic GvHD rate, and a high 30.8% transplantation-related mortality rate.
EUROPEAN JOURNAL OF HAEMATOLOGY
(2021)
Review
Infectious Diseases
Ibai Los-Arcos, Gloria Iacoboni, Manuela Aguilar-Guisado, Laia Alsina-Manrique, Cristina Diaz de Heredia, Claudia Fortuny-Guasch, Irene Garcia-Cadenas, Carolina Garcia-Vidal, Marta Gonzalez-Vicent, Rafael Hernani, Mi Kwon, Marina Machado, Xavier Martinez-Gomez, Valentin Ortiz Maldonado, Carolina Pinto Pla, Jose Luis Pinana, Virginia Pomar, Juan Luis Reguera-Ortega, Miguel Salavert, Pere Soler-Palacin, Lourdes Vazquez-Lopez, Pere Barba, Isabel Ruiz-Camps
Summary: CAR-T cell therapy is a promising treatment for B-cell malignancies, with two products approved for aggressive B-cell lymphoma and acute lymphoblastic leukemia. Patients receiving CAR-T therapy often develop fever post-infusion, mainly due to cytokine release syndrome.
Article
Biophysics
L. Murillo-Sanjuan, M. Gonzalez-Vicent, B. Argiles Aparicio, I Badell Serra, A. Rodriguez Villa, M. L. Uria Oficialdegui, M. Lopez-Duarte, C. Belendez-Bieler, A. Sastre Urgelles, J. Sevilla Navarro, C. Diaz-de-Heredia
Summary: Hematopoietic stem cell transplantation (HSCT) is an effective curative option for hematological manifestations in Fanconi anemia (FA) patients, with a high success rate and overall survival of 73% at 5 years post-transplant. However, patients with myelodysplastic syndrome (MDS) have a lower survival rate compared to those with bone marrow failure (BMF).
BONE MARROW TRANSPLANTATION
(2021)
Article
Pediatrics
Victor Galan Gomez, Lydia de la Fuente Regano, Antonia Rodriguez Villa, Cristina Diaz de Heredia Rubio, Marta Gonzalez Vicent, Isabel Badell Serra, Jose Maria Fernandez, Antonia Isabel Pascual Martinez, Jose Maria Perez Hurtado, Monica Lopez Duarte, M. Soledad Maldonado Regalado, Antonio Perez-Martinez
Summary: Despite advances in drug treatments and hematopoietic stem cell transplantation, the prognosis for patients with Ph-ALL remains unfavorable. A multicentric study analyzed the role of allogeneic HSCT in Ph-ALL patients and found that HSCT with ATG-based conditioning in first complete remission (CR) improved overall survival (OS). HSCT in first CR with ATG-based conditioning, along with the development of acute graft versus host disease (aGvHD), increased event-free survival (EFS). Treatment with imatinib and undetectable minimal residual disease (MRD) prior to HSCT, combined with aGvHD, reduced the risk of relapse (RR). Younger age at HSCT, first CR, and ATG-based conditioning were associated with lower transplant-related mortality (TRM). Therefore, HSCT should be considered for Ph-ALL patients who achieve first CR and receive ATG-based conditioning.
ANALES DE PEDIATRIA
(2022)
Article
Oncology
L. Murillo-Sanjuan, J. Balmana, A. de Pablo Garcia-Cuenca, J. Lorente Guerrero, M. L. Uria Oficialdegui, E. Carrasco, C. Diaz-de-Heredia
Summary: HSCT is a curative option for FA patients with hematological manifestations, but does not prevent solid tumors such as head-and-neck SCC. Long-term follow-up showed a high cumulative incidence of SCC in FA patients, highlighting the importance of multidisciplinary cancer screening programs.
CLINICAL & TRANSLATIONAL ONCOLOGY
(2022)
Article
Microbiology
Jose Maria Valle-T-Figueras, Berta Renedo Miro, Maria Isabel Benitez Carabante, Cristina Diaz-de-Heredia, Jaume Vima Bofarull, Natalia Mendoza-Palomar, Maria Teresa Martin-Gomez, Pere Soler-Palacin
Summary: Voriconazole plasma concentrations in children are highly variable, especially in patients aged 5-12 years. The new dosing recommendations have led to an increased rate of adequate PC in therapeutic drug monitoring, but still a portion falls outside the therapeutic range. Factors influencing PC include severe hypoalbuminemia and elevated C-reactive protein levels.
Article
Allergy
Arjan C. Lankester, Benedicte Neven, Nizar Mahlaoui, Erik G. J. von Asmuth, Virginie Courteille, Mikael Alligon, Michael H. Albert, Isabelle Badell Serra, Peter Bader, Dmitry Balashov, Rita Beier, Yves Bertrand, Stephane Blanche, Victoria Bordon, Robbert G. Bredius, Andrew Cant, Marina Cavazzana, Cristina Diaz-de-Heredia, Figen Dogu, Karoline Ehlert, Natacha Entz-Werle, Anders Fasth, Francesca Ferrua, Alina Ferster, Renata Formankova, Wilhelm Friedrich, Marta Gonzalez-Vicent, Jolanta Gozdzik, Tayfun Gungor, Manfred Hoenig, Aydan Ikinciogullari, Krzysztof Kalwak, Savas Kansoy, Alphan Kupesiz, Arnalda Lanfranchi, Caroline A. Lindemans, Roland Meisel, Gerard Michel, Nuno A. A. Miranda, Jose Moraleda, Despina Moshous, Herbert Pichler, Kanchan Rao, Petr Sedlacek, Mary Slatter, Elena Soncini, Carsten Speckmann, Mikael Sundin, Amos Toren, Kim Vettenranta, Austen Worth, Mehmet A. Yesilipek, Marco Zecca, Fulvio Porta, Ansgar Schulz, Paul Veys, Alain Fischer, Andrew R. Gennery
Summary: This comprehensive multicenter analysis examined the outcomes of hematopoietic stem cell transplantation (HSCT) in patients with severe combined immunodeficiency (SCID) and analyzed the factors affecting immune reconstitution (IR) and long-term clinical outcomes. The results showed improved overall survival (OS) and event-free survival (EFS) in all genotypes and donor types. Pretransplantation infections and the use of mismatched related donors were associated with less favorable OS and EFS. The study also identified factors such as specific genetic mutations, conditioning regimen, matched donor HSCT, and levels of naive CD4 T lymphocytes as independent predictors of favorable clinical and immunologic outcomes.
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2022)
Review
Pediatrics
Matthias Woelfl, Muna Qayed, Maria Isabel Benitez Carabante, Tomas Sykora, Halvard Bonig, Anita Lawitschka, Cristina Diaz-de-Heredia
Summary: Acute graft-versus-host disease (aGvHD) is a major complication following hematopoietic stem cell transplantation (HSCT), and moderate-severity aGvHD is associated with a graft-versus-leukemia effect. To optimize the benefits of transplantation, reducing non-relapse mortality caused by severe GvHD is crucial.
FRONTIERS IN PEDIATRICS
(2022)
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
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.
Article
Medicine, General & Internal
Barbara Ochoa-Fernandez, Victor Galan-Gomez, Carmen Mestre, Marta Gonzalez-Vicent, Antonia Pascual, Laura Alonso, Alexandra Regueiro, Mercedes Plaza, Jose Maria Perez Hurtado, Ana Benito, Jose Luis Fuster, David Bueno, Yasmina Mozo, Jose Luis Vicario, Antonio Balas, Luisa Sisinni, Cristina Diaz de Heredia, Antonio Perez-Martinez
Summary: This study retrospectively analyzed pediatric haplo-HSCT activity in Spain and found that although the method has many advantages, there is still room for improvement in treatment outcomes. The results suggest that MD and NMD patients have different limitations during the transplantation process, and there is no significant difference in 5-year overall survival rate. It is important to establish reference centers to accumulate experience, especially in treating rare diseases.
Article
Hematology
Brian D. Friend, Larisa Broglie, Brent R. Logan, Saurabh Chhabra, Caitrin Bupp, Gary Schiller, Amer Beitinjaneh, Miguel Angel Diaz Perez, Gregory M. T. Guilcher, Hasan Hashem, Gerhard C. Hildebrandt, Maxwell M. Krem, Hillard M. Lazarus, Taiga Nishihori, Roomi Nusrat, Seth J. Rotz, Baldeep Wirk, Matthew Wieduwilt, Marcelo Pasquini, Bipin N. Savani, Edward A. Stadtmauer, Mohamed L. Sorror, Monica S. Thakar
Summary: This study evaluated and developed new risk scoring tools for pediatric and young adult patients by expanding the definitions of Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI). The expanded ymHCT-CI and simplified ymHCT-CI, with broader definitions, were found to be more relevant in assessing the risk of non-relapse mortality in these patient populations. Further prospective studies are needed to validate the use of these novel comorbidity indexes in younger patients.
TRANSPLANTATION AND CELLULAR THERAPY
(2023)
Article
Hematology
Neel S. Bhatt, Akshay Sharma, Andrew St Martin, Muhammad Bilal Abid, Valerie Brown, Miguel Angel Diaz Perez, Haydar Frangoul, Shahinaz M. Gadalla, Megan M. Herr, Maxwell M. Krem, Hillard M. Lazarus, Michael J. Martens, Parinda A. Mehta, Taiga Nishihori, Tim Prestidge, Michael A. Pulsipher, Hemalatha G. Rangarajan, Kirsten M. Williams, Lena E. Winestone, Dwight E. Yin, Marcie L. Riches, Christopher E. Dandoy, Jeffery J. Auletta
Summary: This study describes the outcomes of pediatric hematopoietic stem cell transplant (HSCT) recipients after COVID-19. Although most cases were mild, some patients required supplemental oxygen or mechanical ventilation. The overall survival rate was relatively high, but the mortality rate was still increased compared to adult HSCT recipients. The study also found that patients with HSCT comorbidities were more likely to be diagnosed with COVID-19.
TRANSPLANTATION AND CELLULAR THERAPY
(2022)
Article
Hematology
Carrie L. Kitko, Hisham Abdel-Azim, Paul A. Carpenter, Jean-Hugues Dalle, Cristina Diaz-de-Heredia, Stefania Gaspari, Andrew R. Gennery, Rupert Handgretinger, Anita Lawitschka
Summary: Extracorporeal photopheresis (ECP) is an effective and safe treatment for pediatric patients with steroid-refractory acute graft-versus-host disease (SR-aGVHD).
TRANSPLANTATION AND CELLULAR THERAPY
(2022)
