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1. Prophylactic donor-derived CD19 CAR-T cell infusion for preventing relapse in high-risk B-ALL after allogeneic hematopoietic stem cell transplantation

Author

Lu, Wenyi, Lyu, Hairong, Xiao, Xia, Bai, Xue, Zhang, Meng, Wang, Jiaxi, Pu, Yedi, Meng, Juanxia, Zhang, Xiaomei, Zhu, Haibo, Yuan, Ting, Wang, Bing, Jin, Xin, Cao, Xinping, Wang, Zhao, Xie, Tianle, Meng, Haotian, Stepanov, Alexey V., Gabibov, Alexander G., An, Yuxin, Sun, Rui, Zhang, Yu, Maschan, Mikhail A., Zhu, Zunmin, Zhang, Hongkai, and Zhao, Mingfeng

Published
2024
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3. Targeting CD45 by gene-edited CAR T cells for leukemia eradication and hematopoietic stem cell transplantation preconditioning

Author

Stepanova, Valeria M., Volkov, Dmitry V., Osipova, Daria S., Wang, Wenjian, Hou, Yingqin, Pershin, Dmitry E., Fadeeva, Mariia S., Malakhova, Ekaterina A., Kulakovskaya, Elena A., Cuicui, Lui, Mingfeng, Zhao, Zhang, Hongkai, Xie, Jia, Zhang, Ding, Mamedov, Ilgar Z., Chernov, Alexandr S., Telegin, Georgij B., Rubtsov, Yuri P., Gabibov, Alexander G., Wu, Peng, Maschan, Michael A., and Stepanov, Alexey V.

Published
2024
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4. Vemurafenib combined with cladribine and cytarabine results in durable remission of pediatric BRAF V600E–positive LCH

Author

Evseev, Dmitry, Osipova, Daria, Kalinina, Irina, Raykina, Elena, Ignatova, Anna, Lyudovskikh, Evelina, Baidildina, Dina, Popov, Alexander, Zhogov, Vladimir, Semchenkova, Alexandra, Litvin, Eugeny, Kotskaya, Natalia, Cherniak, Ekaterina, Voronin, Kirill, Burtsev, Eugeny, Bronin, Gleb, Vlasova, Irina, Purbueva, Bazarma, Fink, Olesya, Pristanskova, Ekaterina, Dzhukaeva, Irina, Erega, Elena, Novichkova, Galina, Maschan, Alexey, and Maschan, Michael

Published
2023
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6. Extended characterization of anti-CD19 CAR T cell products manufactured at the point of care using the CliniMACS Prodigy system: comparison of donor sources and process duration

Author

Malakhova, Ekaterina, Pershin, Dmitriy, Kulakovskaya, Elena, Vedmedskaia, Viktoria, Fadeeva, Mariia, Lodoeva, Oyuna, Sozonova, Tatiana, Muzalevskii, Yakov, Kazachenok, Alexei, Belchikov, Vladislav, Shelikhova, Larisa, Molostova, Olga, Volkov, Dmitry, and Maschan, Michael

Published
2024
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8. Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia

Author

Goronkova, Olga, Novichkova, Galina, Salimova, Tatiana, Kalinina, Irina, Baidildina, Dina, Petrova, Ulyana, Antonova, Kristina, Sadovskaya, Maria, Suntsova, Elena, Evseev, Dmitry, Matveev, Victor, Venyov, Dmitry, Khachatryan, Lili, Litvinov, Dmitry, Pshonkin, Alexey, Ovsyannikova, Galina, Kotskaya, Natalia, Gobadze, Darina, Olshanskaya, Yulia, Popov, Alexander, Raykina, Elena, Mironenko, Olga, Voronin, Kirill, Purbueva, Bazarma, Boichenko, Elmira, Dinikina, Yulia, Guseynova, Evgeniya, Sherstnev, Dmitry, Kalinina, Elena, Mezentsev, Sergey, Streneva, Olga, Yudina, Natalia, Plaksina, Olga, Erega, Elena, Maschan, Michael, and Maschan, Alexey

Published
2023
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9. Potential value of high-throughput single-cell DNA sequencing of Juvenile myelomonocytic leukemia: report of two cases

Author

E. V. Volchkov, A. A. Khozyainova, M. Kh. Gurzhikhanova, I. V. Larionova, V. E. Matveev, D. A. Evseev, A. K. Ignatova, M. E. Menyailo, D. A. Venyov, R. S. Vorobev, A. A. Semchenkova, Yu. V. Olshanskaya, E. V. Denisov, and M. A. Maschan

Subjects
Biology (General), QH301-705.5
Abstract

Abstract Juvenile myelomonocytic leukemia (JMML) is a rare myeloproliferative disease of early childhood that develops due to mutations in the genes of the RAS-signaling pathway. Next-generation high throughput sequencing (NGS) enables identification of various secondary molecular genetic events that can facilitate JMML progression and transformation into secondary acute myeloid leukemia (sAML). The methods of single-cell DNA sequencing (scDNA-seq) enable overcoming limitations of bulk NGS and exploring genetic heterogeneity at the level of individual cells, which can help in a better understanding of the mechanisms leading to JMML progression and provide an opportunity to evaluate the response of leukemia to therapy. In the present work, we applied a two-step droplet microfluidics approach to detect DNA alterations among thousands of single cells and to analyze clonal dynamics in two JMML patients with sAML transformation before and after hematopoietic stem cell transplantation (HSCT). At the time of diagnosis both of our patients harbored only “canonical” mutations in the RAS signaling pathway genes detected by targeted DNA sequencing. Analysis of samples from the time of transformation JMML to sAML revealed additional genetic events that are potential drivers for disease progression in both patients. ScDNA-seq was able to measure of chimerism level and detect a residual tumor clone in the second patient after HSCT (sensitivity of less than 0.1% tumor cells). The data obtained demonstrate the value of scDNA-seq to assess the clonal evolution of JMML to sAML, response to therapy and engraftment monitoring.

Published
2023
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11. Switchable targeting of solid tumors by BsCAR T cells

Author

Stepanov, Alexey V., Kalinin, Roman S., Shipunova, Victoria O., Zhang, Ding, Xie, Jia, Rubtsov, Yuri P., Ukrainskaya, Valeria M., Schulga, Alexey, Konovalova, Elena V., Volkov, Dmitry V., Yaroshevich, Igor A., Moysenovich, Anastasiia M., Belogurov, Alexey A., Zhang, Hongkai, Telegin, Georgij B., Chernov, Alexandr S., Maschan, Mikhail A., Terekhov, Stanislav S., Wu, Peng, Deyev, Sergey M., Lerner, Richard A., Gabibov, Alexander G., and Altman, Sidney

Published
2022

12. Pediatric therapy-related hematologic neoplasms show enrichment for KMT2A rearrangement and lymphoblastic phenotype.

Author

Kovach, Alexandra E., Komova, Daria, Itov, Albert, Gaskova, Maria, Kalinina, Irina, Voronin, Kirill, Rumiantseva, Yulia, Karachunskii, Alexander, Maschan, Michael, Maschan, Alexey, Novichkova, Galina, Olshanskaya, Yulia, Bhojwani, Deepa, Raca, Gordana, and Zerkalenkova, Elena

Subjects
HEMATOLOGIC malignancies, RNA sequencing, CHROMOSOMES, PHENOTYPES, ANEUPLOIDY
Abstract

In children, therapy-related hematologic neoplasms (t-HN) are uncommon. Many are driven by genetic events independent of clonal hematopoiesis. We sought to understand the clinical and genetic factors of pediatric t-HN in a large independent cohort. Fifty-six t-HN were retrospectively identified. Chromosome microarray, next-generation and/or RNA sequencing were performed. Patients had primary hematologic, solid, or central nervous system tumors. t-HN included myeloid (t-MN) and lymphoblastic (t-ALL) phenotypes. Approximately half of the cases harbored KMTA2A rearrangement (KMT2Ar). Among t-HN without KMT2Ar, genetic drivers were heterogeneous, including diverse fusions or aneuploidy. Approximately 18% harbored 17p deletions and/or TP53 mutations. EFS/OS was not associated with t-HN lineage or KMT2Ar, but HSCT was associated with improved EFS and OS. We detail one of the largest cohorts to date of pediatric t-HN, confirming frequent KMT2Ar and t-ALL. [ABSTRACT FROM AUTHOR]

Published
2024
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13. The long-term efficacy and safety of nilotinib in pediatric patients with CML: a 5-year update of the DIALOG study

Author

Hijiya, Nobuko, Maschan, Alexey, Rizzari, Carmelo, Shimada, Hiroyuki, Dufour, Carlo, Goto, Hiroaki, Kang, Hyoung Jin, Guinipero, Terri, Karakas, Zeynep, Bautista, Francisco, Ducassou, Stéphane, Yoo, Keon Hee, Zwaan, Christian Michel, Millot, Frédéric, Patterson, Briana C., Samis, Jill, Izquierdo, Miguel, Titorenko, Ksenia, Li, Sai, and Sosothikul, Darintr

Published
2023
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14. Monitoring for virus-specific T-cell responses and viremia in allogeneic HSCT recipients: a survey from the EBMT Cellular Therapy & Immunobiology Working Party

Author

Greco, Raffaella, Hoogenboom, Jorinde D., Bonneville, Edouard F., Anagnostopoulos, Achilles, Cuoghi, Angela, Dalle, Jean-Hugues, Weissinger, Eva M., Lang, Peter, Galaverna, Federica, Martino, Massimo, Maschan, Alexei, Mauz-Körholz, Christine, Noviello, Maddalena, Passweg, Jakob, Peccatori, Jacopo, Rovira, Montserrat, Solano, Carlos, Veelken, Hendrik, Velardi, Andrea, Wagner-Drouet, Eva Maria, Zhang, Xi, Ciceri, Fabio, Bonini, Chiara, Vago, Luca, Ruggeri, Annalisa, and Chabannon, Christian

Published
2023
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15. Novel hemizygous CORO1A variant leads to combined immunodeficiency with defective platelet calcium signaling and cell mobility

Author

Anna Khoreva, MD, Kirill R. Butov, MD, Elena I. Nikolaeva, MD, Alexey Martyanov, PhD, Elena Kulakovskaya, MD, Dmitry Pershin, MD, Maxim Alexenko, MD, Maria Kurnikova, MD, Ruslan Abasov, MD, Elena Raykina, PhD, Dmitry Abramov, MD, Kristina Arnaudova, PhD, Yulia Rodina, PhD, Natalia Trubina, MD, Yulia Skvortsova, PhD, Dmitry Balashov, PhD, Anastasia Sveshnikova, PhD, Alexey Maschan, PhD, Galina Novichkova, PhD, Mikhail Panteleev, PhD, and Anna Shcherbina, PhD

Subjects
CORO1A, combined immunodeficiency, cytoskeleton, actin, granulomatous dermatitis, platelet, Immunologic diseases. Allergy, RC581-607
Abstract

Background: To date, fewer than 20 patients have been identified as having germline biallelic mutations in the coronin-1A gene (CORO1A) and its protein with clinical features of combined immunodeficiency characterized by T-cell lymphopenia ranging from the severe phenotype to the mild phenotype, recurrent infections, and lymphoproliferative disorders. However, the effects of CORO1A protein disruption on actin-dependent functions in primary cells have not been fully delineated. Objective: We sought to characterize the underlying defects of actin-dependent cellular functions in a female patient with combined immunodeficiency caused by a novel missense variant in the CORO1A gene in combination with a de novo heterozygous microdeletion of chromosome 16p11.2 and also to provide evidence of the pathogenicity of this gene mutation. Methods: To identify the genetic defect, next-generation sequencing followed by Sanger confirmation and array comparative genomic hybridization were performed. Western blot and quantitative PCR tests were used to assess the effects on the protein. Flow cytometry and live microscopy were performed to investigate cellular motility and immune cell counts and function. Results: We demonstrated that the CORO1A hemizygous variant c.19C>T, p. A7C induces significant decreases in cellular levels of the CORO1A protein while leaving mRNA concentrations unaffected. The observed mutation resulted in impaired natural killer cell cytotoxicity and platelet calcium signaling. In addition, primary granulocytes and mesenchymal cells showed significant defects in motility. Conclusion: Collectively, we added new data about the CORO1A gene as a key player in actin cytoskeleton dynamics and cell signaling. Our findings expand the clinical spectrum regarding CORO1A protein deficiency and confirm the importance of a personalized therapeutic approach for each patient.

Published
2024
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21. Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study

Author

Aiuti, Alessandro, Maschan, Alexei, Aljurf, Mahmoud, Gedde-Dahl, Tobias, Gurman, Gunhan, Bordon, Victoria, Kriván, Gergely, Locatelli, Franco, Porta, Fulvio, Valcárcel, David, Beguin, Yves, Faraci, Maura, Kröger, Nicolaus, Kulagin, Aleksandr, Shaw, Peter J., Veelken, Joan Hendrik, Diaz de Heredia, Cristina, Fagioli, Franca, Felber, Matthias, Gruhn, Bernd, Holter, Wolfgang, Rössig, Claudia, Sedlacek, Petr, Apperley, Jane, Ayas, Mouhab, Bodova, Ivana, Choi, Goda, Cornelissen, J.J., Sirvent, Anne, Khan, Anjum, Kupesiz, Alphan, Lenhoff, Stig, Ozdogu, Hakan, von der Weid, Nicolas, Rovira, Montserrat, Schots, Rik, Vinh, Donald C., Albert, Michael H., Sirait, Tiarlan, Eikema, Dirk-Jan, Bakunina, Katerina, Wehr, Claudia, Suarez, Felipe, Fox, Maria Laura, Mahlaoui, Nizar, Gennery, Andrew R., Lankester, Arjan C., Beier, Rita, Bernardo, Maria Ester, Bigley, Venetia, Lindemans, Caroline A., Burns, Siobhan O., Carpenter, Ben, Dybko, Jaroslaw, Güngör, Tayfun, Hauck, Fabian, Lum, Su Han, Balashov, Dmitry, Meisel, Roland, Moshous, Despina, Schulz, Ansgar, Speckmann, Carsten, Slatter, Mary A., Strahm, Brigitte, Uckan-Cetinkaya, Duygu, Meyts, Isabelle, Vallée, Tanja C., Wynn, Robert, Neven, Bénédicte, and Morris, Emma C.

Published
2022
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22. CD19 CAR T-cells for pediatric relapsed acute lymphoblastic leukemia with active CNS involvement: a retrospective international study

Author

Jacoby, Elad, Ghorashian, Sara, Vormoor, Britta, De Moerloose, Barbara, Bodmer, Nicole, Molostova, Olga, Yanir, Asaf D, Buechner, Jochen, Elhasid, Ronit, Bielorai, Bella, Rogosic, Srdan, Dourthe, Marie-Emilie, Maschan, Michael, Rossig, Claudia, Toren, Amos, von Stackelberg, Arend, Locatelli, Franco, Bader, Peter, Zimmermann, Martin, Bourquin, Jean Pierre, and Baruchel, Andre

Published
2022
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23. Novel LYST Variants Lead to Aberrant Splicing in a Patient with Chediak–Higashi Syndrome.

Author

Aleksenko, Maxim, Vlasova, Elena, Kieva, Amina, Abasov, Ruslan, Rodina, Yulia, Maschan, Michael, Shcherbina, Anna, and Raykina, Elena

Subjects
GENETIC variation, NUCLEOTIDE sequencing, GEL electrophoresis, COMPLEMENTARY DNA, SYMPTOMS
Abstract

Background: The advent of next-generation sequencing (NGS) has revolutionized the analysis of genetic data, enabling rapid identification of pathogenic variants in patients with inborn errors of immunity (IEI). Sometimes, the use of NGS-based technologies is associated with challenges in the evaluation of the clinical significance of novel genetic variants. Methods: In silico prediction tools, such as SpliceAI neural network, are often used as a first-tier approach for the primary examination of genetic variants of uncertain clinical significance. Such tools allow us to parse through genetic data and emphasize potential splice-altering variants. Further variant assessment requires precise RNA assessment by agarose gel electrophoresis and/or cDNA Sanger sequencing. Results: We found two novel heterozygous variants in the coding region of the LYST gene (c.10104G>T, c.10894A>G) in an individual with a typical clinical presentation of Chediak–Higashi syndrome (CHS). The SpliceAI neural network predicted both variants as probably splice-altering. cDNA assessment by agarose gel electrophoresis revealed the presence of abnormally shortened splicing products in each variant's case, and cDNA Sanger sequencing demonstrated that c.10104G>T and c.10894A>G substitutions resulted in a shortening of the 44 and 49 exons by 41 and 47 bp, respectively. Both mutations probably lead to a frameshift and the formation of a premature termination codon. This, in turn, may disrupt the structure and/or function of the LYST protein. Conclusions: We identified two novel variants in the LYST gene, predicted to be deleterious by the SpliceAI neural network. Agarose gel cDNA electrophoresis and cDNA Sanger sequencing allowed us to verify inappropriate splicing patterns and establish these variants as disease-causing. [ABSTRACT FROM AUTHOR]

Published
2025
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24. Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis

Author

Albert, Michael H., Slatter, Mary A., Gennery, Andrew R., Güngör, Tayfun, Bakunina, Katerina, Markovitch, Benyamin, Hazelaar, Sheree, Sirait, Tiarlan, Courteille, Virginie, Aiuti, Alessandro, Aleinikova, Olga V., Balashov, Dmitry, Bernardo, Maria Ester, Bodova, Ivana, Bruno, Benedicte, Cavazzana, Marina, Chiesa, Robert, Fischer, Alain, Hauck, Fabian, Ifversen, Marianne, Kałwak, Krzysztof, Klein, Christoph, Kulagin, Alexander, Kupesiz, Alphan, Kuskonmaz, Baris, Lindemans, Caroline A., Locatelli, Franco, Lum, Su Han, Maschan, Alexey, Meisel, Roland, Moshous, Despina, Porta, Fulvio, Sauer, Martin G., Sedlacek, Petr, Schulz, Ansgar, Suarez, Felipe, Vallée, Tanja C., Winiarski, Jacek H., Zecca, Marco, Neven, Bénédicte, Veys, Paul, and Lankester, Arjan C.

Published
2022
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25. Graft-versus-host disease and relapse/rejection-free survival after allogeneic transplantation for idiopathic severe aplastic anemia: a comprehensive analysis from the SAAWP of the EBMT

Author

Raynier Devillier, Dirk-Jan Eikema, Carlo Dufour, Mahmoud Aljurf, Depei Wu, Alexei Maschan, Alexander Kulagin, Constantijn J.M. Halkes, Matthew Collin, John Snowden, Cécile Renard, Arnold Ganser, Karl-Walter Sykora, Brenda E Gibson, Johan Maertens, Maija Itäla-Remes, Paola Corti, Jan Cornelissen, Martin Bornhäuser, Mercedes Colorado Araujo, Hakan Ozdogu, Antonio Risitano, Gerard Socie, and Regis Peffault de Latour

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

Survival after allogeneic hematopoietic stem cell transplantation (allo-HSCT) for severe idiopathic aplastic anemia (SAA) has improved in recent years, approaching 75% at 5 years. However, an SAA-adapted composite endpoint, graft-versus-host disease (GvHD) and relapse/rejection-free survival (GRFS), may more accurately assess patient outcomes beyond survival. We analyzed GRFS to identify risk factors and specific causes of GRFS failure. Our retrospective analysis from the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation included 479 patients with idiopathic SAA who underwent allo-HSCT in two conventional situations: i) upfront allo-HSCT from a matched related donor (MRD) (upfront cohort), and ii) allo-HSCT for relapsed or refractory SAA (rel/ref cohort). Relevant events for GRFS calculation included graft failure, grade 3-4 acute GvHD, extensive chronic GvHD, and death. In the upfront cohort (n=209), 5-year GRFS was 77%. Late allo-HSCT (i.e., >6 months after SAA diagnosis) was the main poor prognostic factor, specifically increasing the risk of death as the cause of GRFS failure (hazard ratio [HR]=4.08; 95% confidence interval [CI]: 1.41-11.83; P=0.010). In the rel/ref cohort (n=270), 5-year GRFS was 61%. Age was the main factor significantly increasing the risk of death (HR=1.04; 95% CI: 1.02-1.06; P

Published
2023
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26. Safety and efficacy of the low-dose memory (CD45RA-depleted) donor lymphocyte infusion in recipients of αβ T cell-depleted haploidentical grafts: results of a prospective randomized trial in high-risk childhood leukemia

Author

Dunaikina, Maria, Zhekhovtsova, Zhanna, Shelikhova, Larisa, Glushkova, Svetlana, Nikolaev, Ruslan, Blagov, Sergey, Khismatullina, Rimma, Balashov, Dmitriy, Kurnikova, Elena, Pershin, Dmitriy, Muzalevskii, Yakov, Kazachenok, Alexei, Osipova, Elena, Miakova, Natalia, Litvinov, Dmitriy, Novichkova, Galina, Maschan, Alexei, and Maschan, Michael

Published
2021
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29. T-cell tracking, safety, and effect of low-dose donor memory T-cell infusions after αβ T cell-depleted hematopoietic stem cell transplantation

Author

Blagov, Sergey, Zvyagin, Ivan V., Shelikhova, Larisa, Khismatullina, Rimma, Balashov, Dmitriy, Komech, Ekaterina, Fomchenkova, Viktoria, Shugay, Mikhail, Starichkova, Julia, Kurnikova, Elena, Pershin, Dmitriy, Fadeeva, Maria, Glushkova, Svetlana, Muzalevskii, Yakov, Kazachenok, Alexei, Efimenko, Maria, Osipova, Elena, Novichkova, Galina, Chudakov, Dmitriy, Maschan, Alexei, and Maschan, Michael

Published
2021
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30. A phase 2 study of nilotinib in pediatric patients with CML: long-term update on growth retardation and safety

Author

Hijiya, Nobuko, Maschan, Alexey, Rizzari, Carmelo, Shimada, Hiroyuki, Dufour, Carlo, Goto, Hiroaki, Kang, Hyoung Jin, Guinipero, Terri, Karakas, Zeynep, Bautista, Francisco, Ducassou, Stéphane, Yoo, Keon Hee, Zwaan, Christian Michel, Millot, Frédéric, Patterson, Briana, Samis, Jill, Aimone, Paola, Allepuz, Alex, Titorenko, Ksenia, and Sosothikul, Darintr

Published
2021
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34. Immunophenotypic changes in leukemic blasts in children with relapsed/refractory B-cell precursor acute lymphoblastic leukemia after treatment with CD19-directed chimeric antigen receptor (CAR)- expressing T cells

Author

Ekaterina Mikhailova, Olga Illarionova, Larisa Shelikhova, Elena Zerkalenkova, Olga Molostova, Yulia Olshanskaya, Galina Novichkova, Alexey Maschan, Michael Maschan, and Alexander Popov

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2021
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36. Cabozantinib in children with recurrent diffuse gliomas.

Author

Papusha, Ludmila, Zaytseva, Margarita, Senchenko, Maria, Sanakoeva, Agunda, Panferova, Agnesa, Sharanda, Tatiana, Vilesova, Irina, Tarakanova, Alexandra, Salnikova, Ekaterina, Usman, Natalia, Artemov, Anton, Karachunskiy, Alexander, Maschan, Alexey, Hwang, Eugene, Packer, Roger J., Novichkova, Galina, and Druy, Alexander

Published
2024
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37. Platelet functional abnormalities and clinical presentation in pediatric patients with germline RUNX1, ANKRD26, and ETV6 mutations

Author

Galina S. Ovsyannikova, Daria V. Fedorova, Ivan P. Tesakov, Alexey A. Martyanov, Anastasia A. Ignatova, Evgeniya A. Ponomarenko, Pavel A. Zharkov, Anna V. Pavlova, Elena V. Raykina, Michael A. Maschan, Mikhail A. Panteleev, Galina A. Novichkova, Anastasia N. Sveshnikova, and Nataliya S. Smetanina

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2022
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39. Features of Development and Course of Disseminated Intravascular Coagulation Syndrome During Surgical Interventions in Children with Oncological Diseases

Author

N. P. Leonov, V. V. Schukin, G. A. Novichkova, M. A. Maschan, F. I. Ataullakhanov, S. S. Yashin, A. M. Zeynalov, and Е. A. Spiridonova

Subjects
disseminated intravascular coagulation syndrome, multiple organ failure, bleeding, oncological pathology, thrombosis, coagulopathy, Medical emergencies. Critical care. Intensive care. First aid, RC86-88.9
Abstract

Coagulopathy always accompanies blood loss, and its transformation into disseminated intravascular coagulation syndrome (DIC) is associated with increased morbidity and mortality.Objective: to characterize the features of the development and course of DIC during bleeding, as well as identify the main predictors of its formation during surgical interventions in children with oncological diseases.Material and Methods. A retrospective study of children under 18 years of age with oncological pathology who received surgical treatment for the period from 2017 to 2019 years. Children who received blood transfusion and hemostatic therapy with intraoperative bleeding were selected. The resulting cohort (n=207) was divided into two groups using the modified ISTH assessment system: children with DIC (n=59), without DIC (n=148). Demographic, clinical, and laboratory factors were compared between groups. The final model of multivariate logistic regression included signs that were before the development of DIC on the second day after the operation and were selected as a result of univariate analysis (P

Published
2020
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41. Outcome of αβ T cell-depleted transplantation in children with high-risk acute myeloid leukemia, grafted in remission

Author

Maschan, Michael, Shelikhova, Larisa, Ilushina, Maria, Shekhovtsova, Zhanna, Khismatullina, Rimma, Kurnikova, Elena, Pershin, Dmitriy, Balashov, Dmitriy, Kalinina, Irina, Muzalevskii, Yakov, Kazachenok, Alexei, Zakharova, Viktoria, Olshanskaya, Yulia, Baidildina, Dina, Litvinov, Dmitriy, Novichkova, Galina, and Maschan, Alexei

Published
2020
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42. Platelet function and bleeding at different phases of childhood immune thrombocytopenia

Author

Ignatova, Anastasia A., Suntsova, Elena V., Pshonkin, Alexey V., Martyanov, Alexey A., Ponomarenko, Evgeniya A., Polokhov, Dmitry M., Fedorova, Daria V., Voronin, Kirill A., Kotskaya, Natalia N., Trubina, Natalia M., Krasilnikova, Marina V., Uzueva, Selima Sh., Serkova, Irina V., Ovsyannikova, Galina S., Romanova, Ksenia I., Hachatryan, Lili A., Kalinina, Irina I., Matveev, Viktor E., Korsantiya, Maya N., Smetanina, Natalia S., Evseev, Dmitry A., Sadovskaya, Maria N., Antonova, Kristina S., Khoreva, Anna L., Zharkov, Pavel A., Shcherbina, Anna, Sveshnikova, Anastasia N., Maschan, Aleksey A., Novichkova, Galina A., and Panteleev, Mikhail A.

Published
2021
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43. Multiple site place-of-care manufactured anti-CD19 CAR-T cells induce high remission rates in B-cell malignancy patients

Author

Maschan, Michael, Caimi, Paolo F., Reese-Koc, Jane, Sanchez, Gabriela Pacheco, Sharma, Ashish A., Molostova, Olga, Shelikhova, Larisa, Pershin, Dmitriy, Stepanov, Alexey, Muzalevskii, Yakov, Suzart, Vinicius G., Otegbeye, Folashade, Wald, David, Xiong, Ying, Wu, Darong, Knight, Adam, Oparaocha, Ibe, Ferencz, Beatrix, Roy, Andre, Worden, Andrew, Kruger, Winfried, Kadan, Michael, Schneider, Dina, Orentas, Rimas, Sekaly, Rafick-Pierre, de Lima, Marcos, and Dropulić, Boro

Published
2021
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44. Phase 2 study of nilotinib in pediatric patients with Philadelphia chromosome–positive chronic myeloid leukemia

Author

Hijiya, Nobuko, Maschan, Alexey, Rizzari, Carmelo, Shimada, Hiroyuki, Dufour, Carlo, Goto, Hiroaki, Kang, Hyoung Jin, Guinipero, Terri, Karakas, Zeynep, Bautista, Francisco, Ducassou, Stéphane, Yoo, Keon Hee, Zwaan, Christian Michel, Millot, Frédéric, Aimone, Paola, Allepuz, Alex, Quenet, Sara, Hourcade-Potelleret, Florence, Hertle, Sabine, and Sosothikul, Darintr

Published
2019
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45. Immunophenotypic changes of leukemic blasts in children with relapsed/refractory B-cell precursor acute lymphoblastic leukemia who have been treated with blinatumomab

Author

Ekaterina Mikhailova, Evgeny Gluhanyuk, Olga Illarionova, Elena Zerkalenkova, Svetlana Kashpor, Natalia Miakova, Yulia Diakonova, Yulia Olshanskaya, Larisa Shelikhova, Galina Novichkova, Michael Maschan, Alexey Maschan, and Alexander Popov

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2020
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46. Serotherapy-Free Regimen Improves Non-Relapse Mortality and Immune Recovery Among the Recipients of αβ TCell–Depleted Haploidentical Grafts: Retrospective Study in Childhood Leukemia

Author

Shelikhova, Larisa, Glushkova, Svetlana, Nikolaev, Ruslan, Dunaikina, Maria, Zhekhovtsova, Zhanna, Blagov, Sergey, Khismatullina, Rimma, Balashov, Dmitriy, Kurnikova, Elena, Pershin, Dmitriy, Muzalevskii, Yakov, Kazachenok, Alexei, Osipova, Elena, Trakhtman, Pavel, Maschan, Alexei, and Maschan, Michael

Published
2021
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47. Immunophenotypic but Not Genetic Changes Reclassify the Majority of Relapsed/Refractory Pediatric Cases of Early T-Cell Precursor Acute Lymphoblastic Leukemia.

Author

Demina, Irina, Dagestani, Aya, Borkovskaia, Aleksandra, Semchenkova, Alexandra, Soldatkina, Olga, Kashpor, Svetlana, Olshanskaya, Yulia, Roumiantseva, Julia, Karachunskiy, Alexander, Novichkova, Galina, Maschan, Michael, Zerkalenkova, Elena, and Popov, Alexander

Subjects
LYMPHOBLASTIC leukemia, ACUTE leukemia, ACUTE myeloid leukemia, T cell receptors, T cells, NUCLEOTIDE sequencing, RNA sequencing
Abstract

Early T-cell precursor acute lymphoblastic leukemia (ETP-ALL) develops from very early cells with the potential for both T-cell and myeloid differentiation. The ambiguous nature of leukemic blasts in ETP-ALL may lead to immunophenotypic alterations at relapse. Here, we address immunophenotypic alterations and related classification issues, as well as genetic features of relapsed pediatric ETP-ALL. Between 2017 and 2022, 7518 patients were diagnosed with acute leukemia (AL). In addition to conventional immunophenotyping, karyotyping, and FISH studies, we performed next-generation sequencing of the T-cell receptor clonal repertoire and reverse transcription PCR and RNA sequencing for patients with ETP-ALL at both initial diagnosis and relapse. Among a total of 534 patients diagnosed with T-cell ALL (7.1%), 60 had ETP-ALL (11.2%). Ten patients with ETP-ALL experienced relapse or progression on therapy (16.7%), with a median time to event of 5 months (ranging from two weeks to 5 years). Most relapses were classified as AL of ambiguous lineage (n = 5) and acute myeloid leukemia (AML) (n = 4). Major genetic markers of leukemic cells remained unchanged at relapse. Of the patients with relapse, four had polyclonal leukemic populations and a relapse with AML or bilineal mixed-phenotype AL (MPAL). Three patients had clonal TRD rearrangements and relapse with AML, undifferentiated AL, or retention of the ETP-ALL phenotype. ETP-ALL relapse requires careful clinical and laboratory diagnosis. Treatment decisions should rely mainly on initial examination data, taking into account both immunophenotypic and molecular/genetic characteristics. [ABSTRACT FROM AUTHOR]

Published
2024
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