T Cell Blast Phase (T-BP) of Chronic Myelogenous Leuikemia (CML).

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 4560-4560
Author(s):  
Alfonso Quintás-Cardama ◽  
Hagop Kantarjian ◽  
Jorge Cortes

Abstract Approximately 65% of patients with CMP BP exhibit a myeloid phenotype, 30% a lymphoid, and 5% of cases have and undifferentiated or mixed phenotype. Most cases of lymphoid BP are of B-cell origin. Only anecdotal cases of T-BP have been reported. To evaluate the incidence and outcome of T-BP, we reviewed 410 patients with CML who underwent transformation to BP at M.D. Anderson Cancer Center between January 1999 and April 2007. Six cases (4 female) were diagnosed as having T-BP (incidence 1.5%). Three patients presented initially with T-BP whereas 3 other cases evolved to T-BP from chronic phase. The median time from diagnosis to transformation was 8 months (range, 0–72). The median age was 50 years (range, 24–66), median white blood cell count at presentation 20.5x109/L (range, 2.6–104), hemoglobin 11.2 g/dL (range, 10.6–13.6), platelet count 139x109/L (range, 20–295), peripheral blood blasts 6% (range, 0–100), and bone marrow blasts 20% (range, 2–88). All but 1 presented with extramedullary disease: 2 with lymphadenopathy, 2 with lymphadenopathy and mediastinal mass (including 1 also with pericardial tamponade), and 1 with splenomegaly, lymphadenopathy, and granulocytic sarcoma of the breast. Four patients had an immunophenotype consistent with byphenotypic T-cell/myeloid leukemia and 2 exhibited an exclusive T-cell phenotype. Three patients expressed a b2a2 BCR-ABL1 transcript (p210), whereas 1 carried b2a2+b3a2 (p210), 1 e1a2 (p190), and 1 expressed an e13b2+e14a2 (p210) transcript at the time of transformation but this switched to e1a2 (p190) during the course of dasatinib therapy. Three patients had failed prior therapies, including interferon-alpha (n=3), high dose imatinib (n=1), and matched-unrelated stem cell transplantation (SCT; n=1) at the time of transformation. Initial therapy for T-BP consisted of chemotherapy: hyper-CVAD in 3 patients (in 1 case with imatinib 600 mg daily), VAD in 1 patient, and a combination of idarubicin and ara-C in 2 cases (1 of them with imatinib 600 mg daily and dexamethasone). Only the 2 patients treated with chemotherapy and imatinib responded, achieving a complete cytogenetic response (CCyR) that lasted 3 and 14 months, respectively. Subsequent therapy in the remainder 4 patients consisted of high-dose imatinib (600–800 mg daily; n=4), which was administered for a median of 27 months (range, 0.5–87), dasatinib (n=1), autologous SCT (n=1), allogeneic SCT (n=1), and other chemotherapeutic regimens (n=4). One of the patients treated with imatinib (600 mg daily) achieved a complete molecular response (CMR) that is ongoing after 87 months of therapy. The patient treated with dasatinib (70 mg twice daily) achieved a CCyR. This patient presented the previously unreported K271R ABL kinase domain mutation prior to the start of dasatinib therapy. At the time of dasatinib failure, DNA expansion of specific clones followed by DNA sequencing detected the dasatinib-resistant mutations V299L and F317L in 80% and 20% of clones, respectively. Currently, 4 patients are dead while 2 are still alive, 1 in CMR receiving imatinib and 1 in CCyR after allogeneic SCT. In conclusion, T-BP is a rare variety of BP CML, which frequently exhibits extramedullary disease and high resistance to conventional chemotherapeutic regimens. Long-term responses can be achieved with ABL kinase inhibitors and/or allogeneic SCT.

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 3769-3769
Author(s):  
Aziz Nazha ◽  
Elias Jabbour ◽  
Jorge E. Cortes ◽  
Susan O'Brien ◽  
Jenny Shan ◽  
...  

Abstract Abstract 3769 Background: Until recently, prognosis of pts with CML treated with TKI was based on scores developed in the chemotherapy and interferon era. Hasford and colleagues [Blood. 2011;(118):686–692] have identified the EUTOS score, using the percentage of basophils and spleen size, as a significant tool to predict the probability of achieving 18-month complete cytogenetic response (CCyR) and progression-free survival in patients treated with imatinib. Aims: To validate the EUTOS score in an independent MDACC cohort of pts, with early CML-CP treated with standard-dose imatinib, high-dose imatinib, dasatinib, and nilotinib, and its ability to predict transformation-free survival (TFS), event-free survival (EFS) and overall survival (OS). Methods: 465 consecutive pts with newly diagnosed CML–CP (0 – 6 months from diagnosis to TKI treatment) were treated with imatinib 400 mg daily (n=71), imatinib 800 mg daily (n=208), and 2nd TKIs (n=186; dasatinib n=88, nilotinib n=98) in sequential phase II trials. Entry criteria were similar for all trials. EUTOS score = (7 x basophils %) + (4 x spleen cm BCM). A EUTOS score of >87 indicates high-risk and ≤87 low-risk. Results: 465 pts with CML-CP were assessed. Median age was 47 years (range, 15–85). Median time from diagnosis to TKI therapy was 1 month (range, 0 to 6). 319 (69%), 112 (24%), and 34 (7%) pts were in low, intermediate, and high-Sokal score category, respectively. Median basophils percentage at baseline was 3 (range, 0 to 19). Median splenomegaly size was 0 cm (range, 0 to 30). 118 pts (25%) received previous cytoreduction therapy. Median follow-up was 117 months (range, 16 to 130) for pts receiving standard-dose imatinib, 88 months (range, 4 to 118) for those receiving high-dose imatinib, and 30 months (range, 3 to 69) for those receiving 2nd TKI. The overall CCyR rates were 87%, 91%, and 95%, respectively. The 4-year EFS, TFS, and OS rates for the whole group were 84%, 94%, and 95%, respectively. Overall, of the 465 pts, 427 (92%) were in low EUTOS score category (Table 1). Pts with low EUTOS score had higher rates of CCyR at anytime compared to pts with high EUTOS score (93% versus 81%, p=0.02). This difference was mainly significant among pts receiving 2nd TKI (p=0.03) while it was not different among pts receiving imatinib (p=0.27). There was no difference in the rates of major molecular response (85% versus 81%, p=0.48) between pts with low and high EUTOS score. There was no difference in TFS, EFS, and OS rates between pts with low and high EUTOS score, overall and among specific therapy (Table 1). Conclusion: Eight percent of pts with CML-CP treated at MDACC are of high EUTOS score. In this population, the EUTOS score was not predictive for overall MMR, TFS, EFS, and OS. Disclosures: Cortes: BMS: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding.


Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 432-432 ◽  
Author(s):  
Alfonso Quintas-Cardama ◽  
Hagop M. Kantarjian ◽  
Dan Jones ◽  
Guillermo Garcia-Manero ◽  
Susan O’Brien ◽  
...  

Abstract Background: Achieving a cytogenetic (CG) response or a molecular response after imatinib therapy has been associated with improved event-free (EFS) and transformation-free survival (TFS). It is unclear whether achieving these responses earlier confers an advantage. A recent update of the IRIS trial suggests that achieving a major CG response (MCyR) at 12, 18 or 24 months (mo) confers a similar TFS advantage. Another report (J Clin Oncol2006;24:454) suggests that achieving a complete CG response (CCyR) at 12 or 24 mo confers equal prognosis to patients. Although some patients may indeed improve their response with continued therapy, a pt not in CCyR faces the competing possibilities of eventually achieving a CCyR vs progressing. Methods: We analyzed the risks of improving the CG response vs progressing for pts not in CCyR at different times to determine whether early responses confer an advantage. 258 pts with CML CP treated with IM were analyzed. Progression was defined as transformation to AP or BP, loss of CHR or major CG response, or a doubling of the white cell count to more than 20x109/L. Results: After 3 mo of IM therapy, 77 (74%) of 104 assessable pts for CG response had a CCyR whereas 17 (16%) progressed. These differences were consistently significant at 6 and 12 mo (p=0.04) (Table 1). We then analyzed the long term risk of progression vs the probability of achieving CCyR according to the molecular response at different time points (Table 2). Patients with Bcr-Abl/Abl transcript levels >1–10 after 3 mo of therapy had a 92% probability of achieving CCyR with continued therapy, similar to the 98% for those with ≤1. However, they have a significantly higher risk of eventual progression that is more similar to that of pts with values of >10. The risk of progression increases to 23% if transcript levels >10 at 6 mo. Conclusion: These results suggest that while the risk of progression may be similar for pts who achieve a CCyR regardless of the time at which that is attained, those who fail to obtain a CCyR within the first 12 mo of IM therapy have higher rates of disease progression. This risk is discernible as early as 3 mo into IM therapy and may provide a rationale for therapies that induce higher rates of early molecular response (e.g. high-dose imatinib, new tyrosine kinase inhibitors). Table 1 Time on imatinib No. not in CCyR No. with eventual outcome with continued therapy (%) CCyR Progression p value 3 months 104 77 (74) 17 (16) 6 months 47 28 (60) 12 (26) 0.04 12 months 26 16 (62) 8 (31) Table 2 % Probability Months %Bcr-Abl/Abl No. CCyR Progression p value ≤ 1 87 98 2 3 > 1–10 76 92 11 0.04 > 10 30 67 13 ≤ 1 140 99 4 6 > 1–10 34 91 9 0.005 > 10 13 62 23


2015 ◽  
Vol 4 (5S) ◽  
pp. 15-18
Author(s):  
Luigia Luciano

Imatinib, a BCR-ABL inhibitor, is the standard of care for the first-line treatment of patients with chronic-phase CML. Despite the optimal results, some patients develop resistance to imatinib. For these patients, the second-generation tyrosine kinase inhibitors represent effective therapeutic options. Here, we describe a report about a young patient with CML developing resistance to imatinib due to BCR-ABL kinase domain mutations. After six months of treatment with imatinib 400 mg daily, the patient had complete cytogenic response, while at 12 months he had a sub-optimal molecular response, that is a “warning” according to the European LeukemiaNet guidelines (both 2006 and 2009). At 29 months, persisting the sub-optimal molecular response, we decided to increase the imatinib dose up to 800 mg daily. Even after the dose escalation, the patient couldn’t achieve a major molecular response. Therefore we subjected him to a mutation screening, that highlighted L248V mutation. After switching to nilotinib the patient has resulted in complete cytogenic response and major molecular response.


Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 3428-3428
Author(s):  
Elias Jabbour ◽  
Hagop M. Kantarjian ◽  
Dan Jones ◽  
Yin Cameron ◽  
Elizabeth Burton ◽  
...  

Abstract Abstract 3428 Point mutations of the BCR-ABL KD are the most frequently identified mechanism of resistance in pts with CML who fail TKI. Experimental models of in vitro drug sensitivity have shown that specific mutations may develop after incubation with second generation TKIs, albeit at a decreased frequency compared with imatinib. Some of the mutations are novel and not previously described after imatinib failure; in some instances they did not confer resistance to imatinib. One of them, V299L was rarely encountered after imatinib therapy but was reported to emerge after dasatinib exposure in induced mutagenesis models causing resistance to dasatinib by impairing its binding. We assessed the incidence and pattern of development of V299L in pts with TKI-resistant CML at our institution, and the response following change of therapy. V299L mutation was detected in 15 pts with CML: 1 occurred among 186 pts assessed for mutations (0.05%) after imatinib failure (1% of all mutation detected), 9 among 69 of the 170 evaluable (i.e., had abl sequencing) pts (13%) who developed mutations on dasatinib, and 5 among 19 of the 72 evaluable pts (26%) who developed mutations on bosutinib (p<0.001); none of the 51 pts who developed mutations on nilotinib (among 125 tested) acquired V299L. Median age for pts with V299L was 56 years (range, 26–82 years). Eight pts were previously treated with interferon-alpha. One pt developed V299L after receiving imatinib for 26 months (mos). The median time to development of V299L was 14 mos (range, 1–30 mos) for those treated with dasatinib (7 received dasatinib after imatinib failure, 1 after imatinib and nilotinib failure; and 1 after failure of imatinib, INNO-406, and bosutinib), and 13 mos (range, 2–48 mos) for those treated with bosutinib (after imatinib failure in 1, and as 3rd TKI after imatinib and dasatinib failure). The best response to TKI immediately preceding V299L (1 imatinib, 9 dasatinib, 5 bosutinib) was complete hematologic response only in 6 (40%, 4 dasatinib, 2 bosutinib), minor cytogenetic response in 2 (13%; 1 imatinib, 1 dasatinib), complete cytogenetic response in 4 (27%; 3 dasatinib, 1 bosutinib); no response in 3 pts (20%; 1 dasatinib, 2 bosutinib). The median duration of response was 17 mos. V299L was associated with primary resistance in 4 pts, and secondary resistance in 9. Two pts on dasatinib therapy remained in CHR and minor cytogenetic response, respectively, 3 months after the mutation detection. At the time the mutation was detected, 5 pts were in chronic (CP), 7 in accelerated (AP), and 3 in blast phase (BP). 3 pts (1 CP, 1 AP, 1 BP) received nilotinib after V299L detection and 1 in CPresponded (major molecular response sustained for 40+ mos). One pt received INNO406 and did not respond. One pt in BP was refractory to allogeneic stem cell transplantation and acquired a T315I mutation. Two pts received homoharringtonine, did not respond, but had an eradication of the mutant clone. After a median follow-up of 23 mos (range, 3–48 mos), from the time V299L was detected, 8 died (4 CP and 4 BP). In conclusion, V299L occurs more frequently after dual Src/Bcr-Abl kinase inhibitors therapy, paralleling the findings of in vitro studies. TKIs showing in vitro activity against this mutation (e.g. nilotinib) may be good treatment options for pts with this mutation if treated in chronic phase, but more data is need to evaluate the long-term benefit of this approach. Disclosures: Jabbour: BMS: Honoraria, Speakers Bureau; Novartis: Honoraria, Speakers Bureau. Cortes:Novartis: Research Funding; BMS: Research Funding; Pfizer: Consultancy, Research Funding.


Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 4562-4562
Author(s):  
Alfonso Quintás-Cardama ◽  
Hagop Kantarjian ◽  
Jorge Cortes

Abstract Approximately 65% of patients with CMP BP exhibit a myeloid phenotype, 30% a lymphoid, and 5% of cases have and undifferentiated or mixed phenotype. Although basophilia is a common feature in both the peripheral blood and the bone marrow during the chronic phase of the disease, transformation to B-BP is a rare occurrence with only anecdotal cases reported. To evaluate the incidence and outcome of B-BP, we reviewed 410 patients with CML who underwent transformation to BP at M.D. Anderson Cancer Center between January 1999 and April 2007. Of them, only 2 (0.5%) cases were diagnosed as having B-BP. CASE 1: a 19-year-old woman with CML in accelerated phase (AP) failed allogeneic stem cell transplantation (allo-SCT), donor lymphocyte infusion (DLI), autologous SCT, IFN-α, splenectomy, and gemtuzumab. In 10/00 started imatinib therapy at 400 mg/d, achieving a complete hematologic response (CHR) that lasted until 04/03 when she transformed to BP. Gentuzumab was again administered and rendered a CHR, after which she immediately received SCT from her haploidentical mother, resulting in a complete molecular response (CMR) that lasted until 6/05. Therapy with imatinib 800 mg/d was unsuccessful. In 7/05 her white cell count (WBC) was 44x109/L with 25% blasts and 28% basophils. A bone marrow (BM) biopsy showed the presence of 36% blasts with coarse basophilic granules (negative for myeloperoxidase [MPO] and butyrate esterase) and 38% basophils. Flow cytometry demonstrated 65% blastic cells with positive markers of myeloid lineage (CD11c, CD13, CD33) without immaturity markers (CD34, HLA-DR, TDT) B or T lymphoid markers (CD10, CD19, CD20, CD22, CD2, CD3, CD5) or other myeloid markers (CD14, CD15, CD65, MPO). Cytogenetics revealed an isochromosome 17, +8, and t(9;22) in 19/20 metaphases. A b3a2 BCR-ABL1 (p210) transcript with no evidence of mutations within the kinase domain was detected. Given her critical condition the patient was received only supportive care and died shortly thereafter. CASE 2: a 39-year-old woman was diagnosed with CML in chronic phase (CP) in 12/04. She failed therapy with IFN-α, allo-SCT, DLI, imatinib 800 mg/d, PR1 vaccine, matched unrelated donor SCT, imatinib 800 mg/d in combination with tipifarnib, and nilotinib 600 mg twice daily, after which she progressed to BP. A CBC revealed a WBC of 21x109/L with 29% blasts and 66% basophils. A hypercellular BM biopsy revealed 44% blasts (MPO negative) with large cytoplasmic granules and 45% basophils. Blast exhibited an immunophenotype characterized by positivity of myeloid markers, negativity of MPO, B and T cell markers, and partial positivity of CD117. Karyotypic analysis showed that 16/19 metaphases carried t(9;22), −16, add(17)(p13). Molecular studies detected a e1a2 p190 BCR-ABL1 transcript. Therapy with dasatinib 70 mg twice daily for 16 weeks was ineffective and the patient was referred to hospice care. In conclusion, B-BP is a rare subtype of BP CML that exhibits high resistance to standard chemotherapy, allo-SCT, and targeted BCR-ABL kinase inhibitors.


2021 ◽  
Vol 42 (06) ◽  
pp. 561-568
Author(s):  
Sivasree Kesana ◽  
Venkatraman Radhakrishnan ◽  
Jayachandran Perumal Kalaiyarasi ◽  
Nikita Mehra ◽  
Gangothri Selvarajan ◽  
...  

Abstract Introduction Chronic myeloid leukemia (CML) is rare in children and constitutes 2% of all leukemia. We present our institute experience in treating pediatric CML for 20 years. Objectives There is a paucity of data on pediatric CML from India, hence we would like to present treatment responses and survival rates in our pediatric population treated with tyrosine kinase inhibitors at our center. Materials and Methods Patients aged less than 18 years, diagnosed with CML from 2000 to 2019, and treated with imatinib were analyzed retrospectively considering demographic features, treatment characteristics, and survival outcomes. Descriptive analysis was done for the baseline characteristics. Event-free survival (EFS) and overall survival (OS) were calculated using the Kaplan-Meier method and the factors were compared using the log-rank test. Results During the study period, 95 patients were diagnosed with CML of which 54 (56.8%) were males. The most common stage at presentation was the chronic phase (CP) with 84 (88.4%) patients followed by accelerated phase (AP) and blast crisis (BC) with 6 (6.3%) and 5 (5.3%) patients respectively. The median duration of follow-up for all patients was 98 months. EFS and OS at 8 years for patients with CML-CP were 43.1% and 80.4% respectively. Complete hematological response, complete cytogenetic response, and major molecular response was documented in 91 (95.7%), 73 (76.8%), and 63 (66.3%) patients respectively. Conclusion Outcomes in pediatric CML are comparable to that of adults. Imatinib is well tolerated in children.


2019 ◽  
Vol 3 (10) ◽  
pp. 1610-1621 ◽  
Author(s):  
Chung H. Kok ◽  
David T. Yeung ◽  
Liu Lu ◽  
Dale B. Watkins ◽  
Tamara M. Leclercq ◽  
...  

Abstract In chronic-phase chronic myeloid leukemia (CP-CML) patients treated with frontline imatinib, failure to achieve early molecular response (EMR; EMR failure: BCR-ABL1 &gt;10% on the international scale at 3 months) is predictive of inferior outcomes. Identifying patients at high-risk of EMR failure at diagnosis provides an opportunity to intensify frontline therapy and potentially avoid EMR failure. We studied blood samples from 96 CP-CML patients at diagnosis and identified 365 genes that were aberrantly expressed in 13 patients who subsequently failed to achieve EMR, with a gene signature significantly enriched for stem cell phenotype (eg, Myc, β-catenin, Hoxa9/Meis1), cell cycle, and reduced immune response pathways. We selected a 17-gene panel to predict EMR failure and validated this signature on an independent patient cohort. Patients classified as high risk with our gene expression signature (HR-GES) exhibited significantly higher rates of EMR failure compared with low-risk (LR-GES) patients (78% vs 5%; P &lt; .0001), with an overall accuracy of 93%. Furthermore, HR-GES patients who received frontline nilotinib had a relatively low rate of EMR failure (10%). However, HR-GES patients still had inferior deep molecular response achievement rate by 24 months compared with LR-GES patients. This novel multigene signature may be useful for selecting patients at high risk of EMR failure on standard therapy who may benefit from trials of more potent kinase inhibitors or other experimental approaches.


Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 2120-2120
Author(s):  
Nikolai Velev ◽  
Jorge Cortes ◽  
Richard Champlin ◽  
Hagop M. Kantarjian ◽  
Gabriela Rondon ◽  
...  

Abstract Background: Resistance to TKI therapy is associated with development of KD mutations in approximately 50–60% of pts. Although many imatinib-resistant mutations respond well to second generation TKI, T315I is insensitive to all currently available TKI (imatinib, dasatinib, nilotinib) in vitro and in the clinic. SCT is frequently recommended for these pts but there is no available data about the efficacy of SCT in such pts. Aims: To investigate the efficacy and safety of SCT for patients with TKI-resistant CML with a T315I mutation. Methods: We reviewed the outcome of all pts with T315I that have received a SCT at MD Anderson Cancer Center. Results: Seven pts received 8 transplants. Their median age was 44 years (yrs) (range, 26 to 64 yrs). The median time from diagnosis to SCT was 42 months (mo) (range, 9–160 mo). All pts had become resistant to with imatinib; 5 received dasatinib and 1 nilotinib after imatinib failure, and 6 pts received other additional therapy prior to SCT. At the time of SCT 2 pts were in chronic phase (CP), both in partial cytogenetic response; 2 in accelerated (AP) with active disease; and 3 in second or greater CP from lymphoid blast phase (BP) (1 in minor cytogenetic response, 2 major molecular response, 1 complete molecular response –CMR-). Six transplants were from matched unrelated donors and 2 from cord blood. Best response after SCT was CCyR in 3 (2 AP, 1 BP), CMR in 4 (2 CP, 2 BP), and 1 unknown (died early). After a median follow-up of 11 months from SCT, 4 pts are alive; the 2 transplanted in CP are alive after 11 and 42 months after SCT and in CMR; 1 pt transplanted in AP has a sustained CCyR 20 mo after SCT with persistent T315I representing 94% of transcripts by pyrosequencing; and 1 in BP has a CMR sustained 6 mo after a second SCT (relapsed 5 months after first SCT) 3 pts have died: 1 AP and 2 BP, all with relapse. Conclusion: SCT appears to be an effective strategy for pts with CML with T315I, although longer follow up is needed. Results are significantly better when pts are transplanted in CP. Thus, SCT should be considered in pts with resistance to TKI once T315I is identified, ideally in CP.


Cancers ◽  
2021 ◽  
Vol 13 (7) ◽  
pp. 1643
Author(s):  
Prahathishree Mohanavelu ◽  
Mira Mutnick ◽  
Nidhi Mehra ◽  
Brandon White ◽  
Sparsh Kudrimoti ◽  
...  

Tyrosine kinase inhibitors (TKIs) are the frontline therapy for BCR-ABL (Ph+) chronic myeloid leukemia (CML). A systematic meta-analysis of 43 peer-reviewed studies with 10,769 CML patients compared the incidence of gastrointestinal adverse events (GI AEs) in a large heterogeneous CML population as a function of TKI type. Incidence and severity of nausea, vomiting, and diarrhea were assessed for imatinib, dasatinib, bosutinib, and nilotinib. Examination of combined TKI average GI AE incidence found diarrhea most prevalent (22.5%), followed by nausea (20.6%), and vomiting (12.9%). Other TKI GI AEs included constipation (9.2%), abdominal pain (7.6%), gastrointestinal hemorrhage (3.5%), and pancreatitis (2.2%). Mean GI AE incidence was significantly different between TKIs (p < 0.001): bosutinib (52.9%), imatinib (24.2%), dasatinib (20.4%), and nilotinib (9.1%). Diarrhea was the most prevalent GI AE with bosutinib (79.2%) and dasatinib (28.1%), whereas nausea was most prevalent with imatinib (33.0%) and nilotinib (13.2%). Incidence of grade 3 or 4 severe GI AEs was ≤3% except severe diarrhea with bosutinib (9.5%). Unsupervised clustering revealed treatment efficacy measured by the complete cytogenetic response, major molecular response, and overall survival is driven most by disease severity, not TKI type. For patients with chronic phase CML without resistance, optimal TKI selection should consider TKI AE profile, comorbidities, and lifestyle.


Blood ◽  
2011 ◽  
Vol 118 (5) ◽  
pp. 1208-1215 ◽  
Author(s):  
Simona Soverini ◽  
Andreas Hochhaus ◽  
Franck E. Nicolini ◽  
Franz Gruber ◽  
Thoralf Lange ◽  
...  

AbstractMutations in the Bcr-Abl kinase domain may cause, or contribute to, resistance to tyrosine kinase inhibitors (TKIs) in chronic myeloid leukemia patients. Recommendations aimed to rationalize the use of BCR-ABL mutation testing in chronic myeloid leukemia have been compiled by a panel of experts appointed by the European LeukemiaNet (ELN) and European Treatment and Outcome Study and are here reported. Based on a critical review of the literature and, whenever necessary, on panelists' experience, key issues were identified and discussed concerning: (1) when to perform mutation analysis, (2) how to perform it, and (3) how to translate results into clinical practice. In chronic phase patients receiving imatinib first-line, mutation analysis is recommended only in case of failure or suboptimal response according to the ELN criteria. In imatinib-resistant patients receiving an alternative TKI, mutation analysis is recommended in case of hematologic or cytogenetic failure as provisionally defined by the ELN. The recommended methodology is direct sequencing, although it may be preceded by screening with other techniques, such as denaturing-high performance liquid chromatography. In all the cases outlined within this abstract, a positive result is an indication for therapeutic change. Some specific mutations weigh on TKI selection.


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