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1975 4
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938 results

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Quoted phrase not found in phrase index: "Bone marrow failure syndrome 6"
Page 1
The Genetic Landscape of Diamond-Blackfan Anemia.
Ulirsch JC, Verboon JM, Kazerounian S, Guo MH, Yuan D, Ludwig LS, Handsaker RE, Abdulhay NJ, Fiorini C, Genovese G, Lim ET, Cheng A, Cummings BB, Chao KR, Beggs AH, Genetti CA, Sieff CA, Newburger PE, Niewiadomska E, Matysiak M, Vlachos A, Lipton JM, Atsidaftos E, Glader B, Narla A, Gleizes PE, O'Donohue MF, Montel-Lehry N, Amor DJ, McCarroll SA, O'Donnell-Luria AH, Gupta N, Gabriel SB, MacArthur DG, Lander ES, Lek M, Da Costa L, Nathan DG, Korostelev AA, Do R, Sankaran VG, Gazda HT. Ulirsch JC, et al. Am J Hum Genet. 2018 Dec 6;103(6):930-947. doi: 10.1016/j.ajhg.2018.10.027. Epub 2018 Nov 29. Am J Hum Genet. 2018. PMID: 30503522 Free PMC article.
Diamond-Blackfan anemia (DBA) is a rare bone marrow failure disorder that affects 7 out of 1,000,000 live births and has been associated with mutations in components of the ribosome. ...Leveraging the size of our cohort, we observed robust genotype-phe …
Diamond-Blackfan anemia (DBA) is a rare bone marrow failure disorder that affects 7 out of 1,000,000 live births and ha …
Clinical and Molecular Determinants of Clonal Evolution in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria.
Gurnari C, Pagliuca S, Prata PH, Galimard JE, Catto LFB, Larcher L, Sebert M, Allain V, Patel BJ, Durmaz A, Pinto AL, Inacio MCB, Hernandez L, Dhedin N, Caillat-Zucman S, Clappier E, Sicre de Fontbrune F, Voso MT, Visconte V, Peffault de Latour R, Soulier J, Calado RT, Socié G, Maciejewski JP. Gurnari C, et al. J Clin Oncol. 2023 Jan 1;41(1):132-142. doi: 10.1200/JCO.22.00710. Epub 2022 Sep 2. J Clin Oncol. 2023. PMID: 36054881 Free PMC article.
PATIENTS AND METHODS: We studied a multicenter, retrospective cohort of 1,008 patients (median follow-up 8.6 years) with AA and PNH to assess clinical and molecular determinants of clonal evolution. ...The 5-year overall survival reached 40% and was independently associate …
PATIENTS AND METHODS: We studied a multicenter, retrospective cohort of 1,008 patients (median follow-up 8.6 years) with AA and PNH t …
Baseline clinical characteristics and disease burden in patients with paroxysmal nocturnal hemoglobinuria (PNH): updated analysis from the International PNH Registry.
Schrezenmeier H, Röth A, Araten DJ, Kanakura Y, Larratt L, Shammo JM, Wilson A, Shayan G, Maciejewski JP. Schrezenmeier H, et al. Ann Hematol. 2020 Jul;99(7):1505-1514. doi: 10.1007/s00277-020-04052-z. Epub 2020 May 10. Ann Hematol. 2020. PMID: 32390114 Free PMC article.
Outcomes assessed in the current analysis included proportions of patients with high disease activity (HDA), history of major adverse vascular events (MAVEs; including thrombotic events [TEs]), bone marrow failure (BMF), red blood cell (RBC) transfusions, and …
Outcomes assessed in the current analysis included proportions of patients with high disease activity (HDA), history of major adverse vascul …
Genetic testing in severe aplastic anemia is required for optimal hematopoietic cell transplant outcomes.
McReynolds LJ, Rafati M, Wang Y, Ballew BJ, Kim J, Williams VV, Zhou W, Hendricks RM, Dagnall C, Freedman ND, Carter B, Strollo S, Hicks B, Zhu B, Jones K, Paczesny S, Marsh SGE, Spellman SR, He M, Wang T, Lee SJ, Savage SA, Gadalla SM. McReynolds LJ, et al. Blood. 2022 Aug 25;140(8):909-921. doi: 10.1182/blood.2022016508. Blood. 2022. PMID: 35776903 Free PMC article.
Patients with severe aplastic anemia (SAA) can have an unrecognized inherited bone marrow failure syndrome (IBMFS) because of phenotypic heterogeneity. ...We identified 113 P/LP single-nucleotide variants or small insertions/deletions and 10 copy numbe …
Patients with severe aplastic anemia (SAA) can have an unrecognized inherited bone marrow failure syndrome (IBMF …
Hepatitis-Associated Aplastic Anemia.
Alshaibani A, Dufour C, Risitano A, de Latour R, Aljurf M. Alshaibani A, et al. Hematol Oncol Stem Cell Ther. 2022 Jun 1;15(2):8-12. doi: 10.1016/j.hemonc.2020.10.001. Hematol Oncol Stem Cell Ther. 2022. PMID: 33197413 Review.
Hepatitis-associated aplastic anemia (HAAA) is a rare illness, characterized by onset of pancytopenia with a hypoplastic bone marrow that traditionally occurs within 6 months of an increase in serum aminotransferases. HAAA is observed in 1% to 5% of al …
Hepatitis-associated aplastic anemia (HAAA) is a rare illness, characterized by onset of pancytopenia with a hypoplastic bone marr …
Horse versus rabbit antithymocyte globulin in acquired aplastic anemia.
Scheinberg P, Nunez O, Weinstein B, Scheinberg P, Biancotto A, Wu CO, Young NS. Scheinberg P, et al. N Engl J Med. 2011 Aug 4;365(5):430-8. doi: 10.1056/NEJMoa1103975. N Engl J Med. 2011. PMID: 21812672 Free PMC article. Clinical Trial.
BACKGROUND: In severe acquired aplastic anemia, hematopoietic failure is the result of immune-mediated destruction of bone marrow stem and progenitor cells. ...RESULTS: A large, unexpected difference was observed in the rate of hematologic response at …
BACKGROUND: In severe acquired aplastic anemia, hematopoietic failure is the result of immune-mediated destruction of bone …
Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia.
Goronkova O, Novichkova G, Salimova T, Kalinina I, Baidildina D, Petrova U, Antonova K, Sadovskaya M, Suntsova E, Evseev D, Matveev V, Venyov D, Khachatryan L, Litvinov D, Pshonkin A, Ovsyannikova G, Kotskaya N, Gobadze D, Olshanskaya Y, Popov A, Raykina E, Mironenko O, Voronin K, Purbueva B, Boichenko E, Dinikina Y, Guseynova E, Sherstnev D, Kalinina E, Mezentsev S, Streneva O, Yudina N, Plaksina O, Erega E, Maschan M, Maschan A. Goronkova O, et al. Blood Adv. 2023 Mar 28;7(6):953-962. doi: 10.1182/bloodadvances.2021006716. Blood Adv. 2023. PMID: 35446936 Free PMC article. Clinical Trial.
In severity subgroups, the ORR was 89% vs 57% (P = .028) in favor of IST + ELTR in SAA, but it did not differ in patients with vSAA (52% vs 50%; P = .902). At 6 months after the crossover, 61% of initial ELTR(-) patients achieved a response compared with 17% of initial ELT …
In severity subgroups, the ORR was 89% vs 57% (P = .028) in favor of IST + ELTR in SAA, but it did not differ in patients with vSAA (52% vs …
New-onset aplastic anemia after SARS-CoV-2 vaccination.
Chatzikalil E, Kattamis A, Diamantopoulos P, Solomou EE. Chatzikalil E, et al. Int J Hematol. 2023 Dec;118(6):667-681. doi: 10.1007/s12185-023-03666-z. Epub 2023 Sep 28. Int J Hematol. 2023. PMID: 37768509 Review.
Reporting cases of such uncommon post-vaccination events could help clinicians to consider aplastic anemia when pancytopenia is observed after vaccination. The benefits of SARS-Cov-2 vaccination are established, and reports of rare events serve only to increase awareness i …
Reporting cases of such uncommon post-vaccination events could help clinicians to consider aplastic anemia when pancytopenia is observed …
Diamond-Blackfan anemia.
Krijanovski OI, Sieff CA. Krijanovski OI, et al. Hematol Oncol Clin North Am. 1997 Dec;11(6):1061-77. doi: 10.1016/s0889-8588(05)70483-4. Hematol Oncol Clin North Am. 1997. PMID: 9443046 Review.
The disease is characterized by a moderate-to-severe macrocytic anemia, occasional neutropenia or thrombocytosis, a normocellular bone marrow with erythroid hypoplasia, and an increased risk of developing leukemia. ...Nonresponders usually are transfusion dependent, …
The disease is characterized by a moderate-to-severe macrocytic anemia, occasional neutropenia or thrombocytosis, a normocellular bone …
Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study.
Fattizzo B, Gurnari C, Cassanello G, Bortolotti M, Awada H, Giammarco S, Consonni D, Sica S, Gandhi S, Trikha R, Large J, Salter S, Maciejewski JP, Barcellini W, Kulasekararaj AG. Fattizzo B, et al. Leukemia. 2023 Dec;37(12):2479-2485. doi: 10.1038/s41375-023-02047-z. Epub 2023 Oct 4. Leukemia. 2023. PMID: 37794100 Free PMC article.
Non-severe aplastic anemia is a rare bone marrow failure disorder characterized by variable degrees and combination of cytopenias, with limited data on management and outcome. ...The majority required treatment with cyclosporine (CyA) alone (N = 84) or in com …
Non-severe aplastic anemia is a rare bone marrow failure disorder characterized by variable degrees and combination of …
938 results