Long-term outcome for Down syndrome patients with hematopoietic disorders
Although Down syndrome (DS) patients have a higher risk of developing transient myeloproliferative disorder (TMD) and acute leukemia, very little data is available on long-term outcome in Taiwanese patients. The current study was designed to determine the clinical characteristics and treatment outco...
| Main Authors: | , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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Elsevier
2016-02-01
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| Series: | Journal of the Formosan Medical Association |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S0929664615000601 |
| _version_ | 1818616674157330432 |
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| author | Meng-Ju Li Ni-Chung Lee Yung-Li Yang Hsiu-Ju Yen Hsiu-Hao Chang Yin-Hsiu Chien Meng-Yao Lu Shiann-Tarng Jou Kai-Hsin Lin Wuh-Liang Hwu Dong-Tsamn Lin |
| author_facet | Meng-Ju Li Ni-Chung Lee Yung-Li Yang Hsiu-Ju Yen Hsiu-Hao Chang Yin-Hsiu Chien Meng-Yao Lu Shiann-Tarng Jou Kai-Hsin Lin Wuh-Liang Hwu Dong-Tsamn Lin |
| author_sort | Meng-Ju Li |
| collection | DOAJ |
| description | Although Down syndrome (DS) patients have a higher risk of developing transient myeloproliferative disorder (TMD) and acute leukemia, very little data is available on long-term outcome in Taiwanese patients. The current study was designed to determine the clinical characteristics and treatment outcome of DS patients with TMD or acute leukemia (AL).
Methods: In 25 consecutive DS patients with TMD or AL enrolled from 1990 to 2012, clinical manifestations and treatment protocols were investigated and GATA1 (GATA binding protein 1) mutations were identified. Among 16 DS-acute myeloid leukemia (DS-AML) patients, clinical outcomes were compared between survivors and nonsurvivors.
Results: Most of our DS patients had TMD (32%), acute megakaryoblastic leukemia (24%), or acute erythromegakaryoblastic leukemia (16%). The median follow-up time was 22.5 months (1–230 months). The age was younger and the hemoglobin (Hb) level and platelet count were higher in TMD patients than in leukemia patients. Among DS-AML patients, the Hb level was higher in survivors than nonsurvivors (8.8 ± 2.7 g/dL vs. 5.8 ± 2.4 g/dL; p = 0.044) and the age was older in relapsed patients than in nonrelapsed patients (43.8 ± 18 months old vs. 21.6 ± 8.6 months old; p = 0.025). The 3-year overall survival (OS) rate was 44%, higher in patients receiving appropriate chemotherapy than in those receiving inadequate treatment (63.6% vs. 0%, p = 0.001), and higher in those diagnosed with TMD or AL after 2008 than before 2008 (33.3% vs. 75%; p = 0.119).
Conclusion: Outcome in DS-AML patients is optimal if appropriate treatment is provided. With modification of the treatment strategy in 2008, OS increased in Taiwan. |
| first_indexed | 2024-12-16T16:53:33Z |
| format | Article |
| id | doaj.art-4a700b093f7648f8b86591004d9c9343 |
| institution | Directory Open Access Journal |
| issn | 0929-6646 |
| language | English |
| last_indexed | 2024-12-16T16:53:33Z |
| publishDate | 2016-02-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Journal of the Formosan Medical Association |
| spelling | doaj.art-4a700b093f7648f8b86591004d9c93432022-12-21T22:23:56ZengElsevierJournal of the Formosan Medical Association0929-66462016-02-011152949910.1016/j.jfma.2015.01.009Long-term outcome for Down syndrome patients with hematopoietic disordersMeng-Ju Li0Ni-Chung Lee1Yung-Li Yang2Hsiu-Ju Yen3Hsiu-Hao Chang4Yin-Hsiu Chien5Meng-Yao Lu6Shiann-Tarng Jou7Kai-Hsin Lin8Wuh-Liang Hwu9Dong-Tsamn Lin10Department of Pediatrics, National Taiwan University Hospital Hsin-Chu Branch, Hsinchu, TaiwanDepartment of Pediatrics, National Taiwan University Hospital and College of Medicine, National Taiwan University, Taipei, TaiwanDepartment of Pediatrics, National Taiwan University Hospital and College of Medicine, National Taiwan University, Taipei, TaiwanDepartment of Pediatrics, Taipei Veterans General Hospital, Taipei, TaiwanDepartment of Pediatrics, National Taiwan University Hospital and College of Medicine, National Taiwan University, Taipei, TaiwanDepartment of Pediatrics, National Taiwan University Hospital and College of Medicine, National Taiwan University, Taipei, TaiwanDepartment of Pediatrics, National Taiwan University Hospital and College of Medicine, National Taiwan University, Taipei, TaiwanDepartment of Pediatrics, National Taiwan University Hospital and College of Medicine, National Taiwan University, Taipei, TaiwanDepartment of Pediatrics, National Taiwan University Hospital and College of Medicine, National Taiwan University, Taipei, TaiwanDepartment of Pediatrics, National Taiwan University Hospital and College of Medicine, National Taiwan University, Taipei, TaiwanDepartment of Pediatrics, National Taiwan University Hospital and College of Medicine, National Taiwan University, Taipei, TaiwanAlthough Down syndrome (DS) patients have a higher risk of developing transient myeloproliferative disorder (TMD) and acute leukemia, very little data is available on long-term outcome in Taiwanese patients. The current study was designed to determine the clinical characteristics and treatment outcome of DS patients with TMD or acute leukemia (AL). Methods: In 25 consecutive DS patients with TMD or AL enrolled from 1990 to 2012, clinical manifestations and treatment protocols were investigated and GATA1 (GATA binding protein 1) mutations were identified. Among 16 DS-acute myeloid leukemia (DS-AML) patients, clinical outcomes were compared between survivors and nonsurvivors. Results: Most of our DS patients had TMD (32%), acute megakaryoblastic leukemia (24%), or acute erythromegakaryoblastic leukemia (16%). The median follow-up time was 22.5 months (1–230 months). The age was younger and the hemoglobin (Hb) level and platelet count were higher in TMD patients than in leukemia patients. Among DS-AML patients, the Hb level was higher in survivors than nonsurvivors (8.8 ± 2.7 g/dL vs. 5.8 ± 2.4 g/dL; p = 0.044) and the age was older in relapsed patients than in nonrelapsed patients (43.8 ± 18 months old vs. 21.6 ± 8.6 months old; p = 0.025). The 3-year overall survival (OS) rate was 44%, higher in patients receiving appropriate chemotherapy than in those receiving inadequate treatment (63.6% vs. 0%, p = 0.001), and higher in those diagnosed with TMD or AL after 2008 than before 2008 (33.3% vs. 75%; p = 0.119). Conclusion: Outcome in DS-AML patients is optimal if appropriate treatment is provided. With modification of the treatment strategy in 2008, OS increased in Taiwan.http://www.sciencedirect.com/science/article/pii/S0929664615000601acute myeloid leukemiaDown syndromeGATA1myeloproliferative disorderstranscription factor |
| spellingShingle | Meng-Ju Li Ni-Chung Lee Yung-Li Yang Hsiu-Ju Yen Hsiu-Hao Chang Yin-Hsiu Chien Meng-Yao Lu Shiann-Tarng Jou Kai-Hsin Lin Wuh-Liang Hwu Dong-Tsamn Lin Long-term outcome for Down syndrome patients with hematopoietic disorders Journal of the Formosan Medical Association acute myeloid leukemia Down syndrome GATA1 myeloproliferative disorders transcription factor |
| title | Long-term outcome for Down syndrome patients with hematopoietic disorders |
| title_full | Long-term outcome for Down syndrome patients with hematopoietic disorders |
| title_fullStr | Long-term outcome for Down syndrome patients with hematopoietic disorders |
| title_full_unstemmed | Long-term outcome for Down syndrome patients with hematopoietic disorders |
| title_short | Long-term outcome for Down syndrome patients with hematopoietic disorders |
| title_sort | long term outcome for down syndrome patients with hematopoietic disorders |
| topic | acute myeloid leukemia Down syndrome GATA1 myeloproliferative disorders transcription factor |
| url | http://www.sciencedirect.com/science/article/pii/S0929664615000601 |
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