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JMML usually involves somatic and\/or germline mutations in the genes of the RAS pathway, including <jats:italic>PTPN11<\/jats:italic>,<jats:italic> NRAS<\/jats:italic>,<jats:italic> KRAS<\/jats:italic>,<jats:italic> NF1<\/jats:italic>, and <jats:italic>CBL<\/jats:italic>, in the leukemic cells. Almost all patients with JMML experience an aggressive clinical course, and hematopoietic stem cell transplantation (HSCT) is the only curative treatment. A certain proportion of patients with somatic <jats:italic>NRAS<\/jats:italic> and germline mutations in <jats:italic>CBL<\/jats:italic>, however, have spontaneous resolution. A suitable treatment after diagnosis and conditioning regimen prior to HSCT are yet to be determined, but several clinical trials have been initiated throughout the world to develop suitable pre\u2010 or post\u2010allogeneic HSCT treatments and new targeted therapies that are less toxic, to improve patient outcome.<\/jats:p>","DOI":"10.1111\/ped.13068","type":"journal-article","created":{"date-parts":[[2016,6,20]],"date-time":"2016-06-20T15:04:27Z","timestamp":1466435067000},"page":"681-690","source":"Crossref","is-referenced-by-count":33,"title":["Diagnosis and treatment of juvenile myelomonocytic leukemia"],"prefix":"10.1111","volume":"58","author":[{"given":"Kazuo","family":"Sakashita","sequence":"first","affiliation":[{"name":"Department of Pediatric Hematology and Oncology Nagano Children's Hospital Azumono Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kazuyuki","family":"Matsuda","sequence":"additional","affiliation":[{"name":"Department of Laboratory Medicine Shinshu University Hospital Matsumoto Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kenichi","family":"Koike","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Shinshu University School of Medicine Matsumoto Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2016,8,23]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.leukres.2008.08.022"},{"key":"e_1_2_9_3_1","first-page":"1569","article-title":"Childhood myelodysplastic syndrome in Denmark: incidence and predisposing conditions","volume":"9","author":"Hasle H","year":"1995","journal-title":"Leukemia"},{"key":"e_1_2_9_4_1","first-page":"1853","article-title":"Prognosis of 75 patients with juvenile myelomonocytic leukemia: prospective study by MDS committee in the Japanese Society of Pediatric Hematology","volume":"52","author":"Yoshida N","year":"2011","journal-title":"Rinsho Ketsueki"},{"key":"e_1_2_9_5_1","doi-asserted-by":"crossref","first-page":"2032","DOI":"10.1182\/blood.V92.6.2032","article-title":"Stimulation of mouse and human primitive hematopoiesis by murine embryonic aorta\u2010gonad\u2010mesonephros\u2010derived stromal cell lines","volume":"92","author":"Xu MJ","year":"1998","journal-title":"Blood"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1634\/stemcells.2007-0348"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1038\/leu.2014.239"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2009.07968.x"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2013-01-478412"},{"key":"e_1_2_9_10_1","volume-title":"WHO Classification of Tumors of Haematopoietic and Lymphoid Tissues","author":"World Health Organization","year":"2008"},{"key":"e_1_2_9_11_1","article-title":"Juvenile myelomonocytic leukemia: molecular pathogenesis informs current approaches to therapy and hematopoietic cell transplantation","volume":"25","author":"Dvorak CC","year":"2014","journal-title":"Front. 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