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As a journalist, you can create a free Muck Rack account to customize your profile, list your contact preferences, and upload a portfolio of your best work.Articles
Tisagenlecleucel for post-transplant relapse in young acute lymphoblastic leukemia patients: European real-world determinants of outcome
Abstract This multicenter real-world study identifies critical determinants of outcome for tisagenlecleucel (tisa-cel) in treating post-HSCT relapse in 220 children/young adults with B-ALL from 31 European centers. Median follow-up was 30.0 months, with a 43.6% 2-year event-free survival (EFS), 67.2% overall-survival (OS), and 57.1% incidence of CAR-T failure.
Age-dependent phenotypic and molecular evolution of pediatric MDS arising from GATA2 deficiency - Blood Cancer Journal
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Abstract GATA2 deficiency is an autosomal dominant transcriptopathy disorder with high risk for myelodysplastic syndrome (MDS). To elucidate genotype-phenotype associations and identify new genetic risk factors for MDS, we analyzed 218 individuals with germline heterozygous GATA2 variants. We observed striking age-dependent incidence patterns in GATA2-related MDS (GATA2-MDS), with MDS being absent in infants, rare before age 6 years, and steeply increasing in older children.
By Lili Kotmayer, Guolian Kang, Ayami Yoshimi, Rebecca Voss, Barbara De Moerloose, Henrik Hasle, Kirsi Jahnukainen, Riccardo Masetti, Jochen Buechner, Marek Ussowicz, Paula Kjöllerström, Dominik Turkiewicz, Markus Schmugge, Victoria Okhomina, Cristian Sotomayor, Claudia Wehr, Nathan Gray, Alessandra Giorgetti, Charlotte M. Niemeyer, Luca Vinci
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Nature
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Management of children and adolescents with chronic myeloid leukemia in chronic phase: International pediatric chronic myeloid leukemia expert panel recommendations | Leukemia
Abstract The treatment strategy for children and adolescents with chronic myeloid leukemia in the chronic phase (CML-CP) has evolved from allogeneic hematopoietic stem cell transplantation (HSCT) to tyrosine kinase inhibitors (TKIs).
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