Acute megakaryoblastic leukemia natural history, complications and prognosis
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief:
Overview
Natural History
Due to the rarity of this subtype of hematological malignancy, limited data is available on the natural course or prognosis.
- Clonal proliferation of early megakaryoblasts in the bone marrow results in acute megakaryoblastic leukemia (AMKL).[1] It has a bimodal onset of presentation—occurs both in the pediatric age group (<4 years) and adults.[2]
- In childhood, it is more prevalent in patients with Down syndrome. While it is rare in adults, approximately 0.6% (24/3603) reported in the GIMEMA trial.[3]
- Cases of AMKL secondary to chronic myelogenous leukemia and essential thrombocytosis have been reported in the medical literature.[4][5]
Complications
- Periorbital swelling
- Periosteal elevation
- Osteolytic lesions, especially in long bones, e.g., femur[6]
- Osteoporosis with pathologic fractures[7][8]
- Sweet syndrome (SS)[9]
- Leukemia cutis[10]
- Hypercalcemia[11]
Prognosis
Apart from AMKL in Down syndrome patients, the prognosis of AMKL is poor. The efficacy profile of AMKL in Down syndrome patients is favorable, but it comes with a lot of treatment-related toxicity.
- According to the Children’s Oncology Group (COG) AML0431 trial results, the 5-year event-free survival and overall survival rates were 90% and 93% in 204 eligible Down syndrome with AMKL patients.[12]
- Similarly, the reported 3-year overall survival rate was 100% among 3 AMKL with Down syndrome patients while (47±12%) in non-Down syndrome patients.[13]
- The 5-year overall survival rate in AMKL was 10.6% versus 17.5% in non-M7 Acute myeloid leukemia subtypes.[14] Currently, chemotherapy and allogenic bone marrow transplant (Allo-BMT) are main therapy. Treatment-related toxicity is a big challenge. To address this issue, elaborated large future clinical studies are required.
References
- ↑ Tallman MS, Neuberg D, Bennett JM, Francois CJ, Paietta E, Wiernik PH; et al. (2000). "Acute megakaryocytic leukemia: the Eastern Cooperative Oncology Group experience". Blood. 96 (7): 2405–11. PMID 11001891.
- ↑ Gassmann, Winfried; Löffler, Helmut (2009). "Acute Megakaryoblastic Leukemia". Leukemia & Lymphoma. 18 (sup1): 69–73. doi:10.3109/10428199509075307. ISSN 1042-8194.
- ↑ Pagano, L; Pulsoni, A; Vignetti, M; Mele, L; Fianchi, L; Petti, MC; Mirto, S; Falcucci, P; Fazi, P; Broccia, G; Specchia, G; Di Raimondo, F; Pacilli, L; Leoni, P; Ladogana, S; Gallo, E; Venditti, A; Avanzi, G; Camera, A; Liso, V; Leone, G; Mandelli, F (2002). "Acute megakaryoblastic leukemia: experience of GIMEMA trials". Leukemia. 16 (9): 1622–1626. doi:10.1038/sj.leu.2402618. ISSN 0887-6924.
- ↑ ecancermedicalscience. doi:10.3332/ecancer.2013.375. ISSN 1754-6605. Missing or empty
|title=(help) - ↑ Radaelli, Franca; Mazza, Rita; Curioni, Elisabetta; Ciani, Alberto; Pomati, Mauro; Maiolo, Anna-Teresa (2002). "Acute megakaryocytic leukemia in essential thrombocythemia: an unusual evolution?". European Journal of Haematology. 69 (2): 108–111. doi:10.1034/j.1600-0609.2002.02734.x. ISSN 0902-4441.
- ↑ Athale, Uma H.; Kaste, Sue C.; Razzouk, Bassem I.; Rubnitz, Jeffrey E.; Ribeiro, Raul C. (2002). "Skeletal Manifestations of Pediatric Acute Megakaryoblastic Leukemia". Journal of Pediatric Hematology/Oncology. 24 (7): 561–565. doi:10.1097/00043426-200210000-00014. ISSN 1077-4114.
- ↑ Athale, Uma H.; Kaste, Sue C.; Razzouk, Bassem I.; Rubnitz, Jeffrey E.; Ribeiro, Raul C. (2002). "Skeletal Manifestations of Pediatric Acute Megakaryoblastic Leukemia". Journal of Pediatric Hematology/Oncology. 24 (7): 561–565. doi:10.1097/00043426-200210000-00014. ISSN 1077-4114.
- ↑ Kushner, David C.; Weinstein, Howard J.; Kirkpatrick, John A. (1980). "The radiologic diagnosis of leukemia and lymphoma in children". Seminars in Roentgenology. 15 (4): 316–334. doi:10.1016/0037-198X(80)90027-9. ISSN 0037-198X.
- ↑ Yokoyama K, Kojima M, Komatsumoto S, Nara M, Ohyashiki K, Ikeda Y; et al. (1993). "[Acute megakaryoblastic leukemia associated with Sweet's syndrome, including review of the literature]". Rinsho Ketsueki. 34 (3): 341–7. PMID 8479086.
- ↑ Tsurumi H, Takahashi T, Koshino Y, Oyama M, Matsutomo K, Yasuda M; et al. (1991). "[Acute megakaryoblastic leukemia with leukemia cutis, meningeal leukemia, and myelofibrosis]". Rinsho Ketsueki. 32 (11): 1475–80. PMID 1758056.
- ↑ Qayed M, Ahmed I, Valentini RP, Cushing B, Rajpurkar M (2009). "Hypercalcemia in pediatric acute megakaryocytic leukemia: case report and review of the literature". J Pediatr Hematol Oncol. 31 (5): 373–6. doi:10.1097/MPH.0b013e31819a5d29. PMID 19415024.
- ↑ Taub, Jeffrey W.; Berman, Jason N.; Hitzler, Johann K.; Sorrell, April D.; Lacayo, Norman J.; Mast, Kelley; Head, David; Raimondi, Susana; Hirsch, Betsy; Ge, Yubin; Gerbing, Robert B.; Wang, Yi-Cheng; Alonzo, Todd A.; Campana, Dario; Coustan-Smith, Elaine; Mathew, Prasad; Gamis, Alan S. (2017). "Improved outcomes for myeloid leukemia of Down syndrome: a report from the Children's Oncology Group AAML0431 trial". Blood. 129 (25): 3304–3313. doi:10.1182/blood-2017-01-764324. ISSN 0006-4971.
- ↑ Qi, Haixiao; Mao, Yan; Cao, Qian; Sun, Xingzhen; Kuai, Wenxia; Song, Junhong; Ma, Li; Hong, Ze; Hu, Jian; Zhou, Guoping (2020). "Clinical Characteristics and Prognosis of 27 Patients with Childhood Acute Megakaryoblastic Leukemia". Medical Science Monitor. 26. doi:10.12659/MSM.922662. ISSN 1643-3750.
- ↑ Giri, Smith; Pathak, Ranjan; Prouet, Philippe; Li, Bojia; Martin, Mike G. (2014). "Acute megakaryocytic leukemia is associated with worse outcomes than other types of acute myeloid leukemia". Blood. 124 (25): 3833–3834. doi:10.1182/blood-2014-09-603415. ISSN 0006-4971.