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Diagnostic work-up for severe aplastic anemia in children: Consensus of the North American Pediatric Aplastic Anemia Consortium.
Shimano KA, Narla A, Rose MJ, Gloude NJ, Allen SW, Bergstrom K, Broglie L, Carella BA, Castillo P, de Jong JLO, Dror Y, Geddis AE, Huang JN, Lau BW, McGuinn C, Nakano TA, Overholt K, Rothman JA, Sharathkumar A, Shereck E, Vlachos A, Olson TS, Bertuch AA, Wlodarski MW, Shimamura A, Boklan J. Shimano KA, et al. Among authors: rose mj. Am J Hematol. 2021 Nov 1;96(11):1491-1504. doi: 10.1002/ajh.26310. Epub 2021 Aug 20. Am J Hematol. 2021. PMID: 34342889 Free article.
Hematopoietic cell transplantation for a child with OSTM1 osteopetrosis.
Overholt KM, Rose MJ, Joshi S, Herman GE, Bajwa R, Abu-Arja R, Rangarajan HG, Horwitz EM. Overholt KM, et al. Among authors: rose mj. Blood Adv. 2016 Dec 30;1(4):279-281. doi: 10.1182/bloodadvances.2016002345. eCollection 2017 Jan 10. Blood Adv. 2016. PMID: 29296943 Free PMC article.
Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study.
Grace RF, Bianchi P, van Beers EJ, Eber SW, Glader B, Yaish HM, Despotovic JM, Rothman JA, Sharma M, McNaull MM, Fermo E, Lezon-Geyda K, Morton DH, Neufeld EJ, Chonat S, Kollmar N, Knoll CM, Kuo K, Kwiatkowski JL, Pospíšilová D, Pastore YD, Thompson AA, Newburger PE, Ravindranath Y, Wang WC, Wlodarski MW, Wang H, Holzhauer S, Breakey VR, Kunz J, Sheth S, Rose MJ, Bradeen HA, Neu N, Guo D, Al-Sayegh H, London WB, Gallagher PG, Zanella A, Barcellini W. Grace RF, et al. Among authors: rose mj. Blood. 2018 May 17;131(20):2183-2192. doi: 10.1182/blood-2017-10-810796. Epub 2018 Mar 16. Blood. 2018. PMID: 29549173 Free article.
Physician decision making in selection of second-line treatments in immune thrombocytopenia in children.
Grace RF, Despotovic JM, Bennett CM, Bussel JB, Neier M, Neunert C, Crary SE, Pastore YD, Klaassen RJ, Rothman JA, Hege K, Breakey VR, Rose MJ, Shimano KA, Buchanan GR, Geddis A, Haley KM, Lorenzana A, Thompson A, Jeng M, Neufeld EJ, Brown T, Forbes PW, Lambert MP. Grace RF, et al. Among authors: rose mj. Am J Hematol. 2018 Jul;93(7):882-888. doi: 10.1002/ajh.25110. Epub 2018 May 6. Am J Hematol. 2018. PMID: 29659042 Free PMC article.
Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History Study.
van Beers EJ, van Straaten S, Morton DH, Barcellini W, Eber SW, Glader B, Yaish HM, Chonat S, Kwiatkowski JL, Rothman JA, Sharma M, Neufeld EJ, Sheth S, Despotovic JM, Kollmar N, Pospíšilová D, Knoll CM, Kuo K, Pastore YD, Thompson AA, Newburger PE, Ravindranath Y, Wang WC, Wlodarski MW, Wang H, Holzhauer S, Breakey VR, Verhovsek M, Kunz J, McNaull MA, Rose MJ, Bradeen HA, Addonizio K, Li A, Al-Sayegh H, London WB, Grace RF. van Beers EJ, et al. Among authors: rose mj. Haematologica. 2019 Feb;104(2):e51-e53. doi: 10.3324/haematol.2018.196295. Epub 2018 Sep 13. Haematologica. 2019. PMID: 30213831 Free PMC article. No abstract available.
Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes.
Grace RF, Shimano KA, Bhat R, Neunert C, Bussel JB, Klaassen RJ, Lambert MP, Rothman JA, Breakey VR, Hege K, Bennett CM, Rose MJ, Haley KM, Buchanan GR, Geddis A, Lorenzana A, Jeng M, Pastore YD, Crary SE, Neier M, Neufeld EJ, Neu N, Forbes PW, Despotovic JM. Grace RF, et al. Among authors: rose mj. Am J Hematol. 2019 Jul;94(7):741-750. doi: 10.1002/ajh.25479. Epub 2019 Apr 29. Am J Hematol. 2019. PMID: 30945320 Free PMC article. Clinical Trial.
Characterization of the severe phenotype of pyruvate kinase deficiency.
Al-Samkari H, van Beers EJ, Morton DH, Barcellini W, Eber SW, Glader B, Yaish HM, Chonat S, Kuo KHM, Kollmar N, Despotovic JM, Pospíšilová D, Knoll CM, Kwiatkowski JL, Pastore YD, Thompson AA, Wlodarski MW, Ravindranath Y, Rothman JA, Wang H, Holzhauer S, Breakey VR, Verhovsek MM, Kunz J, Sheth S, Sharma M, Rose MJ, Bradeen HA, McNaull MN, Addonizio K, Al-Sayegh H, London WB, Grace RF. Al-Samkari H, et al. Among authors: rose mj. Am J Hematol. 2020 Oct;95(10):E281-E285. doi: 10.1002/ajh.25926. Epub 2020 Aug 6. Am J Hematol. 2020. PMID: 32619047 Free article. No abstract available.
SLC25A38 congenital sideroblastic anemia: Phenotypes and genotypes of 31 individuals from 24 families, including 11 novel mutations, and a review of the literature.
Heeney MM, Berhe S, Campagna DR, Oved JH, Kurre P, Shaw PJ, Teo J, Shanap MA, Hassab HM, Glader BE, Shah S, Yoshimi A, Ameri A, Antin JH, Boudreaux J, Briones M, Dickerson KE, Fernandez CV, Farah R, Hasle H, Keel SB, Olson TS, Powers JM, Rose MJ, Shimamura A, Bottomley SS, Fleming MD. Heeney MM, et al. Among authors: rose mj. Hum Mutat. 2021 Nov;42(11):1367-1383. doi: 10.1002/humu.24267. Epub 2021 Aug 5. Hum Mutat. 2021. PMID: 34298585 Free PMC article. Review.
199 results