Трудности диагностики и терапии Ph-подобных острых лимфобластных лейкозов: описание 3 клинических случаев
Материалы доступны только для специалистов сферы здравоохранения. Авторизуйтесь или зарегистрируйтесь.
Ключевые слова: Ph-подобный острый лимфобластный лейкоз, рефрактерность, ингибиторы тирозинкиназ, блинатумомаб, полностью транс-ретиноевая кислота.
________________________________________________
B-cell acute lymphoblastic leukemia (B-ALL) is a diverse group of malignant blood disorders both with regard to the biological properties of the tumor and to therapeutic approaches. Immunophenotyping, molecular genetic techniques,
whole-genome sequencing characterize B-ALL as a very diverse group for sensitivity to chemotherapy and prognosis.
We present three clinical cases of patients with B-ALL and expected good response to standard therapy, in whom standard protocol treatment failured: refractoriness, persistence of minimal residual disease (MRD), and progression (MRD increase). The remission in these patients was achieved after chemotherapy change to immunological targeted therapy.
Nowadays a unified therapeutic approach to all primary patients of the B-ALL is considered generally outdated. Great efforts are carrying out to develop molecular genetic classifications. The molecular dissection of subtypes of B-ALL goes on, and new protocols for selective treatment with targeting are clearly outlined for each subtype of B-ALL.
Keywords: Ph-like acute lymphoblastic leukemia, refractory, tyrosine kinase inhibitors, blinatumomab, all-trans-retinoic acid.
2. Moorman AV. The clinical relevance of chromosomal and genomic abnormalities in B-cell precursor acute lymphoblastic leukaemia. Blood Reviews. 2012;26(3):123-135. https://doi.org/10.1016/j.blre.2012. 01.001
3. Den Boer ML, van Slegtenhorst M, De Menezes RX, Cheok MH, Buijs-Gladdines JG, Peters ST, Van Zutven LJ, Beverloo HB, Van der Spek PJ, Escherich G, Horstmann MA, Janka-Schaub GE, Kamps WA, Evans WE, Pieters R. A subtype of childhood acute lymphoblastic leukaemia with poor treatment outcome: a genome-wide classification study. The Lancet Oncology. 2009;10(2):125-134. https://doi.org/10. 1016/S1470-2045(08)70339-5
4. Mullighan CG, Su X, Zhang J, Radtke I, Phillips LA, Miller CB, Ma J, Liu W, Cheng C, Schulman BA, Harvey RC, Chen IM, Clifford RJ, Carroll WL, Reaman G, Bowman WP, Devidas M, Gerhard DS, Yang W, Relling MV, Shurtleff SA, Campana D, Borowitz MJ, Pui CH, Smith M, Hunger SP, Willman CL, Downing JR; Children's Oncology Group. Deletion ofIKZF1and Prognosis in Acute Lymphoblastic Leukemia. New England Journal of Medicine. 2009;360(5):470-480. https://doi.org/10.1056/nejmoa0808253
5. Roberts KG, Li Y, Payne-Turner D, Harvey RC, Yang YL, Pei D, McCastlain K, Ding L, Lu C, Song G, Ma J, Becksfort J, Rusch M, Chen SC, Easton J, Cheng J, Boggs K, Santiago-Morales N, Iacobucci I, Fulton RS, Wen J, Valentine M, Cheng C, Paugh SW, Devidas M, Chen IM, Reshmi S, Smith A, Hedlund E, Gupta P, Nagahawatte P, Wu G, Chen X, Yergeau D, Vadodaria B, Mulder H, Winick NJ, Larsen EC, Carroll WL, Heerema NA, Carroll AJ, Grayson G, Tasian SK, Moore AS, Keller F, Frei-Jones M, Whitlock JA, Raetz EA, White DL, Hughes TP, Guidry Auvil JM, Smith MA, Marcucci G, Bloomfield CD, Mrózek K, Kohlschmidt J, Stock W, Kornblau SM, Konopleva M, Paietta E, Pui CH, Jeha S, Relling MV, Evans WE, Gerhard DS, Gastier-Foster JM, Mardis E, Wilson RK, Loh ML, Downing JR, Hunger SP, Willman CL, Zhang J, Mullighan CG. Targetable Kinase-Activating Lesions in Ph-like Acute Lymphoblastic Leukemia. New England Journal of Medicine. 2014;371(11):1005-1015. https://doi.org/10.1056/nejmoa1403088
6. Herold T, Baldus C, Gökbuget N. Ph-like Acute Lymphoblastic Leukemia in Older Adults. New England Journal of Medicine. 2014;371(23):2235-2235. https://doi.org/10.1056/nejmc1412123
7. Herold T, Schneider S, Metzeler KH, Neumann M, Hartmann L, Roberts KG, Konstandin NP, Greif PA, Bräundl K, Ksienzyk B, Huk N4, Schneider I, Zellmeier E, Jurinovic V, Mansmann U, Hiddemann W, Mullighan CG, Bohlander SK, Spiekermann K, Hoelzer D, Brüggemann M, Baldus CD, Dreyling M, Gökbuget N. Adults with Philadelphia chromosome–like acute lymphoblastic leukemia frequently have IGH-CRLF2 and JAK2 mutations, persistence of minimal residual disease and poor prognosis. Haematologica. 2016;102(1):130-138. https://doi.org/10.3324/haematol.2015.136366
8. Jain N, Roberts KG, Jabbour E, Patel K, Eterovic AK, Chen K, Zweidler-McKay P, Lu X, Fawcett G, Wang SA, Konoplev S, Harvey RC, Chen IM, Payne-Turner D, Valentine M, Thomas D, Garcia-Manero G, Ravandi F, Cortes J, Kornblau S, O'Brien S, Pierce S, Jorgensen J, Shaw KR, Willman CL, Mullighan CG, Kantarjian H, Konopleva M. Ph-like acute lymphoblastic leukemia: a high-risk subtype in adults. Blood. 2016;129(5):572-581. https://doi.org/10.1182/blood-2016-07-726588
9. Roberts KG, Gu Z, Payne-Turner D, McCastlain K, Harvey RC, Chen IM, Pei D, Iacobucci I, Valentine M, Pounds SB, Shi L, Li Y, Zhang J, Cheng C, Rambaldi A, Tosi M, Spinelli O, Radich JP, Minden MD, Rowe JM, Luger S, Litzow MR, Tallman MS, Wiernik PH, Bhatia R, Aldoss I, Kohlschmidt J, Mrózek K, Marcucci G, Bloomfield CD, Stock W, Kornblau S, Kantarjian HM, Konopleva M, Paietta E, Willman CL, Mullighan CG. Roberts K, Gu Z, Payne-Turner D et al. High Frequency and Poor Outcome of Philadelphia Chromosome–Like Acute Lymphoblastic Leukemia in Adults. Journal of Clinical Oncology. 2017;35(4):394-401. https://doi.org/10.1200/jco.2016.69.0073
10. Boer JM, Koenders JE, van der Holt B, Exalto C, Sanders MA, Cornelissen JJ, Valk PJ, den Boer ML, Rijneveld AW. Expression profiling of adult acute lymphoblastic leukemia identifies a BCR-ABL1-like subgroup characterized by high non-response and relapse rates. Haematologica. 2015;100(7):e261-e264. https://doi.org/10.3324/ haematol.2014.117424
11. Imamura T, Kiyokawa N, Kato M, Imai C, Okamoto Y, Yano M, Ohki K, Yamashita Y, Kodama Y, Saito A, Mori M, Ishimaru S, Deguchi T, Hashii Y, Shimomura Y, Hori T, Kato K, Goto H, Ogawa C, Koh K, Taki T, Manabe A, Sato A, Kikuta A, Adachi S, Horibe K, Ohara A, Watanabe A, Kawano Y, Ishii E, Shimada H. Characterization of pediatric Philadelphia-negative B-cell precursor acute lymphoblastic leukemia with kinase fusions in Japan. Blood Cancer J. 2016;6(5): e419-e419. https://doi.org/10.1038/bcj.2016.28
12. van der Veer A, Waanders E, Pieters R, Willemse ME, Van Reijmersdal SV, Russell LJ, Harrison CJ, Evans WE, van der Velden VH, Hoogerbrugge PM, Van Leeuwen F, Escherich G, Horstmann MA, Mohammadi Khankahdani L, Rizopoulos D, De Groot-Kruseman HA, Sonneveld E, Kuiper RP, Den Boer ML. Independent prognostic value of BCR-ABL1-like signature and IKZF1 deletion, but not high CRLF2 expression, in children with B-cell precursor ALL. Blood. 2013;122(15):2622-2629. https://doi.org/10.1182/blood-2012-10-462 358
13. Loh ML, Zhang J, Harvey RC, Roberts K, Payne-Turner D, Kang H, Wu G, Chen X, Becksfort J, Edmonson M, Buetow KH, Carroll WL, Chen IM, Wood B, Borowitz MJ, Devidas M, Gerhard DS, Bowman P, Larsen E, Winick N, Raetz E, Smith M, Downing JR, Willman CL, Mullighan CG, Hunger SP. Tyrosine kinome sequencing of pediatric acute lymphoblastic leukemia: a report from the Children's Oncology Group TARGET Project. Blood. 2012;121(3):485-488. https://doi.org/ 10.1182/blood-2012-04-422691
14. Harvey RC, Mullighan CG, Chen IM, Wharton W, Mikhail FM, Carroll AJ, Kang H, Liu W, Dobbin KK, Smith MA, Carroll WL, Devidas M, Bowman WP, Camitta BM, Reaman GH, Hunger SP, Downing JR, Willman CL. Rearrangement of CRLF2 is associated with mutation of JAK kinases, alteration of IKZF1, Hispanic/Latino ethnicity, and a poor outcome in pediatric B-progenitor acute lymphoblastic leukemia. Blood. 2010;115(26):5312-5321. https://doi.org/10.1182/blood-2009-09-245944
15. Boer JM, Marchante JR, Evans WE, Horstmann MA, Escherich G, Pieters R, Den Boer ML. CR-ABL1-like cases in pediatric acute lymphoblastic leukemia: a comparison between DCOG/Erasmus MC and COG/St. Jude signatures. Haematologica. 2015;100(9):e354-e357. https://doi.org/10.3324/haematol.2015.124941
16. Russell LJ, Capasso M, Vater I, Akasaka T, Bernard OA, Calasanz MJ, Chandrasekaran T, Chapiro E, Gesk S, Griffiths M, Guttery DS, Haferlach C, Harder L, Heidenreich O, Irving J, Kearney L, Nguyen-Khac F, Machado L, Minto L, Majid A, Moorman AV, Morrison H, Rand V, Strefford JC, Schwab C, Tönnies H, Dyer MJ, Siebert R, Harrison CJ. Deregulated expression of cytokine receptor gene, CRLF2, is involved in lymphoid transformation in B-cell precursor acute lymphoblastic leukemia. Blood. 2009;114(13):2688-2698. https://doi.org/10.1182/ blood-2009-03-208397
17. Mullighan CG, Collins-Underwood JR, Phillips LA, Loudin MG,
Liu W, Zhang J, Ma J, Coustan-Smith E, Harvey RC, Willman CL, Mikhail FM, Meyer J, Carroll AJ, Williams RT, Cheng J, Heerema NA, Basso G, Pession A, Pui CH, Raimondi SC, Hunger SP, Downing JR, Carroll WL, Rabin KR. Rearrangement of CRLF2 in B-progenitor– and Down syndrome–associated acute lymphoblastic leukemia. Nat Genet. 2009;41(11):1243-1246. https://doi.org/10.1038/ng.469
18. Mullighan CG, Zhang J, Harvey RC, Collins-Underwood JR, Schulman BA, Phillips LA, Tasian SK, Loh ML, Su X, Liu W, Devidas M, Atlas SR, Chen IM, Clifford RJ, Gerhard DS, Carroll WL, Reaman GH, Smith M, Downing JR, Hunger SP, Willman CL. JAK mutations in high-risk childhood acute lymphoblastic leukemia. Proceedings of the National Academy of Sciences. 2009;106(23):9414-9418. https://doi.org 10.1073/pnas.0811761106
19. Weston BW, Hayden MA, Roberts KG, Bowyer S, Hsu J, Fedoriw G, Rao KW, Mullighan CG. Tyrosine Kinase Inhibitor Therapy Induces Remission in a Patient With Refractory EBF1-PDGFRB–Positive Acute Lymphoblastic Leukemia. Journal of Clinical Oncology. 2013;31(25):e413-e416. https://doi.org 10.1200/jco.2012.47.6770
20. Lengline E, Beldjord K, Dombret H, Soulier J, Boissel N, Clappier E. Successful tyrosine kinase inhibitor therapy in a refractory B-cell precursor acute lymphoblastic leukemia with EBF1-PDGFRB fusion. Haematologica. 2013;98(11):e146-e148. https://doi.org 10.3324/ haematol.2013.095372
21. Roberts KG, Morin RD, Zhang J, Hirst M, Zhao Y, Su X, Chen SC, Payne-Turner D, Churchman ML, Harvey RC, Chen X, Kasap C, Yan C, Becksfort J, Finney RP, Teachey DT, Maude SL, Tse K, Moore R, Jones S, Mungall K, Birol I, Edmonson MN, Hu Y, Buetow KE, Chen IM, Carroll WL, Wei L, Ma J, Kleppe M, Levine RL, Garcia-Manero G, Larsen E, Shah NP, Devidas M, Reaman G, Smith M, Paugh SW, Evans WE, Grupp SA, Jeha S, Pui CH, Gerhard DS, Downing JR, Willman CL, Loh M, Hunger SP, Marra MA, Mullighan CG. Genetic Alterations Activating Kinase and Cytokine Receptor Signaling in High-Risk Acute Lymphoblastic Leukemia. Cancer Cell. 2012;22(2):153-166. https://doi.org 10.1016/j.ccr.2012.06.005
22. Maude SL, Dolai S, Delgado-Martin C, Vincent T, Robbins A, Selvanathan A, Ryan T, Hall J, Wood AC, Tasian SK, Hunger SP, Loh ML, Mullighan CG, Wood BL, Hermiston ML, Grupp SA, Lock RB, Teachey DT.Efficacy of JAK/STAT pathway inhibition in murine xenograft models of early T-cell precursor (ETP) acute lymphoblastic leukemia. Blood. 2015;125(11):1759-1767. https://doi.org 10.1182/ blood-2014-06-580480
23. Wilhelm SM, Carter C, Tang L, Wilkie D, McNabola A, Rong H, Chen C, Zhang X, Vincent P, McHugh M, Cao Y, Shujath J, Gawlak S, Eveleigh D, Rowley B, Liu L, Adnane L, Lynch M, Auclair D, Taylor I, Gedrich R, Voznesensky A, Riedl B, Post LE, Bollag G, Trail PA. BAY 43-9006 Exhibits Broad Spectrum Oral Antitumor Activity and Targets the RAF/MEK/ERK Pathway and Receptor Tyrosine Kinases Involved in Tumor Progression and Angiogenesis. Cancer Res. 2004; 64(19):7099-7109. https://doi.org 10.1158/0008-5472.can-04-1443
24. Roberts K, Mullighan C. Genomics in acute lymphoblastic leukaemia: insights and treatment implications. Nature Reviews Clinical Oncology. 2015;12(6):344-357. https://doi.org 10.1038/nrclinonc.2015.38
25. Pui C, Roberts K, Yang J, Mullighan C. Philadelphia Chromosome–like Acute Lymphoblastic Leukemia. Clinical Lymphoma Myeloma and Leukemia. 2017;17(8):464-470. https://doi.org 10.1016/j.clml. 2017.03.299
26. Churchman ML, Evans K, Richmond J, Robbins A, Jones L, Shapiro IM, Pachter JA, Weaver DT, Houghton PJ, Smith MA, Lock RB, Mullighan CG. Synergism of FAK and tyrosine kinase inhibition in Ph+ B-ALL. JCI Insight. 2016;1(4). https://doi.org 10.1172/jci.insight.86082
27. Churchman ML, Low J, Qu C, Paietta EM, Kasper LH, Chang Y, Payne-Turner D, Althoff MJ, Song G, Chen SC, Ma J, Rusch M, McGoldrick D, Edmonson M, Gupta P, Wang YD, Caufield W, Freeman B, Li L, Panetta JC, Baker S, Yang YL, Roberts KG, McCastlain K, Iacobucci I, Peters JL, Centonze VE, Notta F, Dobson SM, Zandi S, Dick JE, Janke L, Peng J, Kodali K, Pagala V, Min J, Mayasundari A, Williams RT, Willman CL, Rowe J, Luger S, Dickins RA, Guy RK, Chen T, Mullighan CG. Efficacy of Retinoids in IKZF1-Mutated BCR-ABL1 Acute Lymphoblastic Leukemia. Cancer Cell. 2015;28(3):343-356. https://doi.org 10.1016/j.ccell.2015.07.016
28. Kantarjian H, Stein A, Gökbuget N, Fielding AK, Schuh AC, Ribera JM, Wei A, Dombret H, Foà R, Bassan R, Arslan Ö, Sanz MA, Bergeron J, Demirkan F, Lech-Maranda E, Rambaldi A, Thomas X, Horst HA, Brüggemann M, Klapper W, Wood BL, Fleishman A, Nagorsen D, Holland C, Zimmerman Z, Topp MS. Blinatumomab versus Chemotherapy for Advanced Acute Lymphoblastic Leukemia. New England Journal of Medicine. 2017;376(9):836-847. https://doi.org 10.1056/nejmoa1609783
29. Sokolov AN, Parovichnikova EN, Troitskaya VV, Kuzmina LA, Galtseva IV, Kulikov SM, Bondarenko SN, Davidova JO, Kapranov NM, Lukyanova IA, Lobanova TI, Usikova EI, Zarubina KI, Savchenko VG. Blinatumomab + Tyrosine Kinase Inhibitors with No Chemotherapy in BCR-ABL-Positive or IKZF1-Deleted or FLT3-ITD-Positive Relapsed/Refractory Acute Lymphoblastic Leukemia Patients: High Molecular Remission Rate and Toxicity Profile. Blood. 2017;130:3884.
30. Sokolov A, Parovichnikova E, Troitskaya V, Galtseva I, Kuzmina L, Davidova J, Kapranov N, Lukyanova I, Lobanova T, Zarubina K, Usikova E, Savchenko V. Targetable blinatumomab + tyrosine kinase inhibitors treatment in relapsed/refractory acute lymphoblastic leukemia patients: clinical effectiveness and peripheral lymphocytes subpopulations kinetics. Haematologica. 2017; 102(s2):354-355.
________________________________________________
1. Thomas D, O'Brien S, Faderl S, Garcia-Manero G, Ferrajoli A, Wierda W, Ravandi F, Verstovsek S, Jorgensen J, Bueso-Ramos C, Andreeff M, Pierce S, Garris R, Keating M, Cortes J and Kantarjian H. Chemoimmunotherapy With a Modified Hyper-CVAD and Rituximab Regimen Improves Outcome in De Novo Philadelphia Chromosome–Negative Precursor B-Lineage Acute Lymphoblastic Leukemia. Journal of Clinical Oncology. 2010; 28(24):3880-3889. https://doi.org/10.1200/ JCO.2009.26.9456
2. Moorman AV. The clinical relevance of chromosomal and genomic abnormalities in B-cell precursor acute lymphoblastic leukaemia. Blood Reviews. 2012;26(3):123-135. https://doi.org/10.1016/j.blre.2012. 01.001
3. Den Boer ML, van Slegtenhorst M, De Menezes RX, Cheok MH, Buijs-Gladdines JG, Peters ST, Van Zutven LJ, Beverloo HB, Van der Spek PJ, Escherich G, Horstmann MA, Janka-Schaub GE, Kamps WA, Evans WE, Pieters R. A subtype of childhood acute lymphoblastic leukaemia with poor treatment outcome: a genome-wide classification study. The Lancet Oncology. 2009;10(2):125-134. https://doi.org/10. 1016/S1470-2045(08)70339-5
4. Mullighan CG, Su X, Zhang J, Radtke I, Phillips LA, Miller CB, Ma J, Liu W, Cheng C, Schulman BA, Harvey RC, Chen IM, Clifford RJ, Carroll WL, Reaman G, Bowman WP, Devidas M, Gerhard DS, Yang W, Relling MV, Shurtleff SA, Campana D, Borowitz MJ, Pui CH, Smith M, Hunger SP, Willman CL, Downing JR; Children's Oncology Group. Deletion ofIKZF1and Prognosis in Acute Lymphoblastic Leukemia. New England Journal of Medicine. 2009;360(5):470-480. https://doi.org/10.1056/nejmoa0808253
5. Roberts KG, Li Y, Payne-Turner D, Harvey RC, Yang YL, Pei D, McCastlain K, Ding L, Lu C, Song G, Ma J, Becksfort J, Rusch M, Chen SC, Easton J, Cheng J, Boggs K, Santiago-Morales N, Iacobucci I, Fulton RS, Wen J, Valentine M, Cheng C, Paugh SW, Devidas M, Chen IM, Reshmi S, Smith A, Hedlund E, Gupta P, Nagahawatte P, Wu G, Chen X, Yergeau D, Vadodaria B, Mulder H, Winick NJ, Larsen EC, Carroll WL, Heerema NA, Carroll AJ, Grayson G, Tasian SK, Moore AS, Keller F, Frei-Jones M, Whitlock JA, Raetz EA, White DL, Hughes TP, Guidry Auvil JM, Smith MA, Marcucci G, Bloomfield CD, Mrózek K, Kohlschmidt J, Stock W, Kornblau SM, Konopleva M, Paietta E, Pui CH, Jeha S, Relling MV, Evans WE, Gerhard DS, Gastier-Foster JM, Mardis E, Wilson RK, Loh ML, Downing JR, Hunger SP, Willman CL, Zhang J, Mullighan CG. Targetable Kinase-Activating Lesions in Ph-like Acute Lymphoblastic Leukemia. New England Journal of Medicine. 2014;371(11):1005-1015. https://doi.org/10.1056/nejmoa1403088
6. Herold T, Baldus C, Gökbuget N. Ph-like Acute Lymphoblastic Leukemia in Older Adults. New England Journal of Medicine. 2014;371(23):2235-2235. https://doi.org/10.1056/nejmc1412123
7. Herold T, Schneider S, Metzeler KH, Neumann M, Hartmann L, Roberts KG, Konstandin NP, Greif PA, Bräundl K, Ksienzyk B, Huk N4, Schneider I, Zellmeier E, Jurinovic V, Mansmann U, Hiddemann W, Mullighan CG, Bohlander SK, Spiekermann K, Hoelzer D, Brüggemann M, Baldus CD, Dreyling M, Gökbuget N. Adults with Philadelphia chromosome–like acute lymphoblastic leukemia frequently have IGH-CRLF2 and JAK2 mutations, persistence of minimal residual disease and poor prognosis. Haematologica. 2016;102(1):130-138. https://doi.org/10.3324/haematol.2015.136366
8. Jain N, Roberts KG, Jabbour E, Patel K, Eterovic AK, Chen K, Zweidler-McKay P, Lu X, Fawcett G, Wang SA, Konoplev S, Harvey RC, Chen IM, Payne-Turner D, Valentine M, Thomas D, Garcia-Manero G, Ravandi F, Cortes J, Kornblau S, O'Brien S, Pierce S, Jorgensen J, Shaw KR, Willman CL, Mullighan CG, Kantarjian H, Konopleva M. Ph-like acute lymphoblastic leukemia: a high-risk subtype in adults. Blood. 2016;129(5):572-581. https://doi.org/10.1182/blood-2016-07-726588
9. Roberts KG, Gu Z, Payne-Turner D, McCastlain K, Harvey RC, Chen IM, Pei D, Iacobucci I, Valentine M, Pounds SB, Shi L, Li Y, Zhang J, Cheng C, Rambaldi A, Tosi M, Spinelli O, Radich JP, Minden MD, Rowe JM, Luger S, Litzow MR, Tallman MS, Wiernik PH, Bhatia R, Aldoss I, Kohlschmidt J, Mrózek K, Marcucci G, Bloomfield CD, Stock W, Kornblau S, Kantarjian HM, Konopleva M, Paietta E, Willman CL, Mullighan CG. Roberts K, Gu Z, Payne-Turner D et al. High Frequency and Poor Outcome of Philadelphia Chromosome–Like Acute Lymphoblastic Leukemia in Adults. Journal of Clinical Oncology. 2017;35(4):394-401. https://doi.org/10.1200/jco.2016.69.0073
10. Boer JM, Koenders JE, van der Holt B, Exalto C, Sanders MA, Cornelissen JJ, Valk PJ, den Boer ML, Rijneveld AW. Expression profiling of adult acute lymphoblastic leukemia identifies a BCR-ABL1-like subgroup characterized by high non-response and relapse rates. Haematologica. 2015;100(7):e261-e264. https://doi.org/10.3324/ haematol.2014.117424
11. Imamura T, Kiyokawa N, Kato M, Imai C, Okamoto Y, Yano M, Ohki K, Yamashita Y, Kodama Y, Saito A, Mori M, Ishimaru S, Deguchi T, Hashii Y, Shimomura Y, Hori T, Kato K, Goto H, Ogawa C, Koh K, Taki T, Manabe A, Sato A, Kikuta A, Adachi S, Horibe K, Ohara A, Watanabe A, Kawano Y, Ishii E, Shimada H. Characterization of pediatric Philadelphia-negative B-cell precursor acute lymphoblastic leukemia with kinase fusions in Japan. Blood Cancer J. 2016;6(5): e419-e419. https://doi.org/10.1038/bcj.2016.28
12. van der Veer A, Waanders E, Pieters R, Willemse ME, Van Reijmersdal SV, Russell LJ, Harrison CJ, Evans WE, van der Velden VH, Hoogerbrugge PM, Van Leeuwen F, Escherich G, Horstmann MA, Mohammadi Khankahdani L, Rizopoulos D, De Groot-Kruseman HA, Sonneveld E, Kuiper RP, Den Boer ML. Independent prognostic value of BCR-ABL1-like signature and IKZF1 deletion, but not high CRLF2 expression, in children with B-cell precursor ALL. Blood. 2013;122(15):2622-2629. https://doi.org/10.1182/blood-2012-10-462 358
13. Loh ML, Zhang J, Harvey RC, Roberts K, Payne-Turner D, Kang H, Wu G, Chen X, Becksfort J, Edmonson M, Buetow KH, Carroll WL, Chen IM, Wood B, Borowitz MJ, Devidas M, Gerhard DS, Bowman P, Larsen E, Winick N, Raetz E, Smith M, Downing JR, Willman CL, Mullighan CG, Hunger SP. Tyrosine kinome sequencing of pediatric acute lymphoblastic leukemia: a report from the Children's Oncology Group TARGET Project. Blood. 2012;121(3):485-488. https://doi.org/ 10.1182/blood-2012-04-422691
14. Harvey RC, Mullighan CG, Chen IM, Wharton W, Mikhail FM, Carroll AJ, Kang H, Liu W, Dobbin KK, Smith MA, Carroll WL, Devidas M, Bowman WP, Camitta BM, Reaman GH, Hunger SP, Downing JR, Willman CL. Rearrangement of CRLF2 is associated with mutation of JAK kinases, alteration of IKZF1, Hispanic/Latino ethnicity, and a poor outcome in pediatric B-progenitor acute lymphoblastic leukemia. Blood. 2010;115(26):5312-5321. https://doi.org/10.1182/blood-2009-09-245944
15. Boer JM, Marchante JR, Evans WE, Horstmann MA, Escherich G, Pieters R, Den Boer ML. CR-ABL1-like cases in pediatric acute lymphoblastic leukemia: a comparison between DCOG/Erasmus MC and COG/St. Jude signatures. Haematologica. 2015;100(9):e354-e357. https://doi.org/10.3324/haematol.2015.124941
16. Russell LJ, Capasso M, Vater I, Akasaka T, Bernard OA, Calasanz MJ, Chandrasekaran T, Chapiro E, Gesk S, Griffiths M, Guttery DS, Haferlach C, Harder L, Heidenreich O, Irving J, Kearney L, Nguyen-Khac F, Machado L, Minto L, Majid A, Moorman AV, Morrison H, Rand V, Strefford JC, Schwab C, Tönnies H, Dyer MJ, Siebert R, Harrison CJ. Deregulated expression of cytokine receptor gene, CRLF2, is involved in lymphoid transformation in B-cell precursor acute lymphoblastic leukemia. Blood. 2009;114(13):2688-2698. https://doi.org/10.1182/ blood-2009-03-208397
17. Mullighan CG, Collins-Underwood JR, Phillips LA, Loudin MG,
Liu W, Zhang J, Ma J, Coustan-Smith E, Harvey RC, Willman CL, Mikhail FM, Meyer J, Carroll AJ, Williams RT, Cheng J, Heerema NA, Basso G, Pession A, Pui CH, Raimondi SC, Hunger SP, Downing JR, Carroll WL, Rabin KR. Rearrangement of CRLF2 in B-progenitor– and Down syndrome–associated acute lymphoblastic leukemia. Nat Genet. 2009;41(11):1243-1246. https://doi.org/10.1038/ng.469
18. Mullighan CG, Zhang J, Harvey RC, Collins-Underwood JR, Schulman BA, Phillips LA, Tasian SK, Loh ML, Su X, Liu W, Devidas M, Atlas SR, Chen IM, Clifford RJ, Gerhard DS, Carroll WL, Reaman GH, Smith M, Downing JR, Hunger SP, Willman CL. JAK mutations in high-risk childhood acute lymphoblastic leukemia. Proceedings of the National Academy of Sciences. 2009;106(23):9414-9418. https://doi.org 10.1073/pnas.0811761106
19. Weston BW, Hayden MA, Roberts KG, Bowyer S, Hsu J, Fedoriw G, Rao KW, Mullighan CG. Tyrosine Kinase Inhibitor Therapy Induces Remission in a Patient With Refractory EBF1-PDGFRB–Positive Acute Lymphoblastic Leukemia. Journal of Clinical Oncology. 2013;31(25):e413-e416. https://doi.org 10.1200/jco.2012.47.6770
20. Lengline E, Beldjord K, Dombret H, Soulier J, Boissel N, Clappier E. Successful tyrosine kinase inhibitor therapy in a refractory B-cell precursor acute lymphoblastic leukemia with EBF1-PDGFRB fusion. Haematologica. 2013;98(11):e146-e148. https://doi.org 10.3324/ haematol.2013.095372
21. Roberts KG, Morin RD, Zhang J, Hirst M, Zhao Y, Su X, Chen SC, Payne-Turner D, Churchman ML, Harvey RC, Chen X, Kasap C, Yan C, Becksfort J, Finney RP, Teachey DT, Maude SL, Tse K, Moore R, Jones S, Mungall K, Birol I, Edmonson MN, Hu Y, Buetow KE, Chen IM, Carroll WL, Wei L, Ma J, Kleppe M, Levine RL, Garcia-Manero G, Larsen E, Shah NP, Devidas M, Reaman G, Smith M, Paugh SW, Evans WE, Grupp SA, Jeha S, Pui CH, Gerhard DS, Downing JR, Willman CL, Loh M, Hunger SP, Marra MA, Mullighan CG. Genetic Alterations Activating Kinase and Cytokine Receptor Signaling in High-Risk Acute Lymphoblastic Leukemia. Cancer Cell. 2012;22(2):153-166. https://doi.org 10.1016/j.ccr.2012.06.005
22. Maude SL, Dolai S, Delgado-Martin C, Vincent T, Robbins A, Selvanathan A, Ryan T, Hall J, Wood AC, Tasian SK, Hunger SP, Loh ML, Mullighan CG, Wood BL, Hermiston ML, Grupp SA, Lock RB, Teachey DT.Efficacy of JAK/STAT pathway inhibition in murine xenograft models of early T-cell precursor (ETP) acute lymphoblastic leukemia. Blood. 2015;125(11):1759-1767. https://doi.org 10.1182/ blood-2014-06-580480
23. Wilhelm SM, Carter C, Tang L, Wilkie D, McNabola A, Rong H, Chen C, Zhang X, Vincent P, McHugh M, Cao Y, Shujath J, Gawlak S, Eveleigh D, Rowley B, Liu L, Adnane L, Lynch M, Auclair D, Taylor I, Gedrich R, Voznesensky A, Riedl B, Post LE, Bollag G, Trail PA. BAY 43-9006 Exhibits Broad Spectrum Oral Antitumor Activity and Targets the RAF/MEK/ERK Pathway and Receptor Tyrosine Kinases Involved in Tumor Progression and Angiogenesis. Cancer Res. 2004; 64(19):7099-7109. https://doi.org 10.1158/0008-5472.can-04-1443
24. Roberts K, Mullighan C. Genomics in acute lymphoblastic leukaemia: insights and treatment implications. Nature Reviews Clinical Oncology. 2015;12(6):344-357. https://doi.org 10.1038/nrclinonc.2015.38
25. Pui C, Roberts K, Yang J, Mullighan C. Philadelphia Chromosome–like Acute Lymphoblastic Leukemia. Clinical Lymphoma Myeloma and Leukemia. 2017;17(8):464-470. https://doi.org 10.1016/j.clml. 2017.03.299
26. Churchman ML, Evans K, Richmond J, Robbins A, Jones L, Shapiro IM, Pachter JA, Weaver DT, Houghton PJ, Smith MA, Lock RB, Mullighan CG. Synergism of FAK and tyrosine kinase inhibition in Ph+ B-ALL. JCI Insight. 2016;1(4). https://doi.org 10.1172/jci.insight.86082
27. Churchman ML, Low J, Qu C, Paietta EM, Kasper LH, Chang Y, Payne-Turner D, Althoff MJ, Song G, Chen SC, Ma J, Rusch M, McGoldrick D, Edmonson M, Gupta P, Wang YD, Caufield W, Freeman B, Li L, Panetta JC, Baker S, Yang YL, Roberts KG, McCastlain K, Iacobucci I, Peters JL, Centonze VE, Notta F, Dobson SM, Zandi S, Dick JE, Janke L, Peng J, Kodali K, Pagala V, Min J, Mayasundari A, Williams RT, Willman CL, Rowe J, Luger S, Dickins RA, Guy RK, Chen T, Mullighan CG. Efficacy of Retinoids in IKZF1-Mutated BCR-ABL1 Acute Lymphoblastic Leukemia. Cancer Cell. 2015;28(3):343-356. https://doi.org 10.1016/j.ccell.2015.07.016
28. Kantarjian H, Stein A, Gökbuget N, Fielding AK, Schuh AC, Ribera JM, Wei A, Dombret H, Foà R, Bassan R, Arslan Ö, Sanz MA, Bergeron J, Demirkan F, Lech-Maranda E, Rambaldi A, Thomas X, Horst HA, Brüggemann M, Klapper W, Wood BL, Fleishman A, Nagorsen D, Holland C, Zimmerman Z, Topp MS. Blinatumomab versus Chemotherapy for Advanced Acute Lymphoblastic Leukemia. New England Journal of Medicine. 2017;376(9):836-847. https://doi.org 10.1056/nejmoa1609783
29. Sokolov AN, Parovichnikova EN, Troitskaya VV, Kuzmina LA, Galtseva IV, Kulikov SM, Bondarenko SN, Davidova JO, Kapranov NM, Lukyanova IA, Lobanova TI, Usikova EI, Zarubina KI, Savchenko VG. Blinatumomab + Tyrosine Kinase Inhibitors with No Chemotherapy in BCR-ABL-Positive or IKZF1-Deleted or FLT3-ITD-Positive Relapsed/Refractory Acute Lymphoblastic Leukemia Patients: High Molecular Remission Rate and Toxicity Profile. Blood. 2017;130:3884.
30. Sokolov A, Parovichnikova E, Troitskaya V, Galtseva I, Kuzmina L, Davidova J, Kapranov N, Lukyanova I, Lobanova T, Zarubina K, Usikova E, Savchenko V. Targetable blinatumomab + tyrosine kinase inhibitors treatment in relapsed/refractory acute lymphoblastic leukemia patients: clinical effectiveness and peripheral lymphocytes subpopulations kinetics. Haematologica. 2017; 102(s2):354-355.
1 ФГБУ «Национальный медицинский исследовательский центр гематологии» Минздрава России, Москва, Россия;
2 НИИ детской онкологии, гематологии и трансплантологии им. Р.М. Горбачевой, Первый Санкт-Петербургский государственный медицинский университет им. акад. И.П. Павлова, Санкт-Петербург, Россия
________________________________________________
K.I. ZARUBINA 1, E.N. PAROVICHNIKOVA 1, G.A. BASKHAEVA 1, A.E. KRASILNIKOVA 1, O.A. GAVRILINA 1, B.V. BIDERMAN 1, A.B. SUDARIKOV 1, S.N. BONDARENKO 2, Y.O. DAVYDOVA 1, I.V. GALTSEVA 1, A.N. SOKOLOV 1, V.V. TROITSKAYA1, V.G. SAVCHENKO 1
1 National Research Center for Hematology, Moscow, Russia;
2 Pavlov First Saint Petersburg State Medical University, Saint Petersburg, Russia