scholarly journals Relapsed or Refractory Burkitt Lymphoma in Children and Adolescents after BFM-Type First-Line Therapy - a BFM Group Report

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 1738-1738 ◽  
Author(s):  
Wilhelm Woessmann ◽  
Martin Zimmermann ◽  
Birgit Burkhardt ◽  
Andrea Meinhardt ◽  
Christine Rosenbusch ◽  
...  
Keyword(s):  
Risk Factor ◽  
Children And Adolescents ◽  
Continuous Infusion ◽  
Burkitt Lymphoma ◽  
Significant Risk ◽  
Front Line ◽  
First Line ◽  
Hematopoietic Stem ◽  
First Line Therapy ◽  
Line Therapy

Abstract Introduction: Current effective front-line therapies for Burkitt lymphoma leave few patients with relapsed or refractory disease. The objective of this analysis was to evaluate outcome, re-induction and SCT -approaches as well as risk factors in children and adolescents with progression or relapse of a Burkitt lymphoma/leukemia after current BFM-type first-line therapy. Patients and Methods: We analyzed a population-based cohort of 145 children with relapsed Burkitt lymphoma/leukemia after BFM-type first-line therapy between 1986 and 2013. Recommended salvage until 2000 was BFM-type re-induction chemotherapy followed by autologous hematopoietic stem cell transplantation (SCT). From 2001 on different re-inductions were used, Rituximab was applied in relapse and both autologous and allogeneic SCT were performed. Results:With a median follow-up of 8.2 (2.7-9.6) years, the 4-year survival after first relapse was 15±3%. Survival significantly improved for patients with relapse/progression after 2000 (until 2000, n=82: 11±3%, 2001 and later, n=63: 21±5%, p=.004) so that further risk factor analyses were performed on these 63 patients: Almost all patients with progression during front-line therapy (21/22), progression during re-induction (26/27), those not reaching CR before SCT (14/15) or not receiving rituximab during re-induction (9/9) died. Survival of 12 patients relapsing after initial therapy for low risk disease (R1/R2) was 42±14% compared to 16±5% for 51 patients relapsing after R3/R4-therapy (p=.02). CNS involvement could not be confirmed as statistically significant risk factor. Re-induction by CC (25 patients), ICE (8 patients), ICI (6 patients) or other regimen (11 patients) was followed by SCT in 31 patients (autologous, 18 patients; allogeneic, 13 patients). These approaches were associated with comparable survival of 16±5%. Two centers used a strategy of two to three courses of intensive continuous-infusion re-induction based on Vincristin (or Paclitaxel), Ifosfamide, Carboplatin, Idarubicine, Rituximab and intrathecal triple therapy without complete hematological regeneration between courses followed by allogeneic SCT. Survival of 8 patients treated this way was 63±17% (p=.034 as compared to the survival with other regimen). A center-effect may confound. Conclusion: Patients with relapsed Burkitt lymphoma/leukemia still have a poor chance to survive after current effective front-line therapy. Time-condensed continuous-infusion re-induction with CD20-antibodies followed by SCT might form the basis for trials testing new drugs. Disclosures Off Label Use: Most chemotherapy drugs used for relapsed Burkitt lymphoma as well as Rituximab are not licenced for children and adolescents..

Greek Registry Of Essential Thrombocythemia: Baseline Characteristics and Therapeutic Strategy

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 4084-4084
Author(s):  
Damianos Sotiropoulos ◽  
Argiris Symeonidis ◽  
Vassilios K Papadopoulos ◽  
Panagiotis Tsirigotis ◽  
Maria Pagoni ◽  
...  
Keyword(s):  
Risk Factor ◽  
Significant Risk ◽  
Jak2 V617f ◽  
Significant Risk Factor ◽  
First Line ◽  
Platelet Counts ◽  
First Line Therapy ◽  
Line Therapy ◽  
History Of

Abstract The Greek Registry of Essential Thrombocythemia (ET) is implemented under the auspices of the Acute Leukemias and Myeloproliferative Neoplasms Study Group of the Hellenic Society of Haematology. Hereby, we present results after four years of retrospective data collection. The total number of patients included is 1078, from 14 Greek sites; ET was diagnosed between 1982 and 2012. The male to female ratio is 1:1.19. Median age at diagnosis is 63 years, median platelet counts (PLT) 826x109/L, hemoglobin (Hb) 13.6 g/dL, white blood cell counts (WBC) 9.4x109/L. The presenting symptoms were a thrombotic event in 6.8%, a hemorrhagic event in 1.5% of patients. In 79.8% of the patients the diagnosis was made after incidental finding of elevated platelet counts on routine laboratory investigation. Molecular studies were performed in 677 patients and 42.8% of them were positive for the JAK2-V617F mutation. The presence of JAK2-V617F mutation (mutant vs wild type allele) was associated with baseline platelet counts (757.5 vs 882 x109/L) and hemoglobin levels (14.4 vs 13.4 g/dL), p<0.001 (Mann-Whitney U-test). A history of thrombosis or hemorrhage was present in 18.6% and 6.6% of patients respectively. Chi-square test was performed to assess whether platelet counts at diagnosis (<600, 600-800, >800 x109/L), Hb<13.8g/dL, WBC>9.5x109/L, or splenomegaly are associated with thrombotic or hemorrhagic events in the past medical history or during the follow-up of ET patients. The only statistically significant difference was observed in the occurrence of thrombosis during the follow up: 10.1% of those with PLT between 600-800 x109/L, 4.5% in PLT<600 and 5.6% in PLT>800 x109/L. To assess for possible confounders the multivariable logistic regression model was used, with independent variables the PLT at diagnosis, age >60 years, history of thrombosis and first line therapy. The history of thrombosis was the only statistically significant risk factor with odds ratio (OR) 3.9 (p=0.0005), while PLT was not a statistically significant risk factor (OR=2.5, p=0.074). Antiplatelet therapy was offered in 80% of patients (aspirin in 59.1%, clopidogrel in 4.7%, and combination therapy in 6.5%); anticoagulants (low molecular weight heparin or warfarin) were given in 2.3%, while the remaining 17.8% of patients did not receive any antithrombotic therapy. During the first six months post diagnosis, 31.6% of patients did not need any cytoreductive therapy. The rest 68.4% of the patients received first line therapy (hydroxyurea 80.6%, anagrelide 11.4% and interferon 5.4%). The response rates were 89.9%, 82.1% and 85.7%, respectively. Second-line therapy was received by 25.8% of the patients (hydroxyurea 23%, anagrelide 44.6%, interferon 9.5%), while the off-label combination of hydroxyurea and anagrelide was administered to 21.2% of the patients. Of the 852 patients treated with hydroxyurea as first line therapy, 12.1% switched to anagrelide and 1.2% to interferon. Of those initially treated with anagrelide, 27.6% switched to hydroxyurea and 8.2% to interferon. During the follow up phase, secondary solid tumor occurred in 4% and hematological malignancy in 2.7% of the patients. The aim of the registry and the subsequent data analysis is to convey the practice of managing the disease. Moreover, useful conclusions can be reached regarding to the patients’ responsiveness to therapy and the minimization of thrombotic and hemorrhagic adverse events. Disclosures: Spanoudakis: Genesis Hellas: Honoraria. Kotsianidis:Genesis Hellas: Honoraria, Research Funding.

Relapsed or Refractory Anaplastic Large-Cell Lymphoma in Children and Adolescents After Berlin-Frankfurt-Muenster (BFM)–Type First-Line Therapy: A BFM-Group Study

2011 ◽  
Vol 29 (22) ◽  
pp. 3065-3071 ◽  
Author(s):  
Willi Woessmann ◽  
Martin Zimmermann ◽  
Meike Lenhard ◽  
Birgit Burkhardt ◽  
Claudia Rossig ◽  
...  
Keyword(s):  
Children And Adolescents ◽  
Anaplastic Large Cell Lymphoma ◽  
Cell Lymphoma ◽  
Large Cell ◽  
First Line ◽  
Hematopoietic Stem ◽  
First Line Therapy ◽  
Line Therapy ◽  
First Relapse ◽  
Large Cell Lymphoma

Purpose To evaluate risk factors for outcome in children and adolescents with relapse of anaplastic large-cell lymphoma (ALCL) after comparable first-line therapy. Patients and Methods We analyzed a population-based cohort of 74 children with relapsed ALCL after Berlin-Frankfurt-Muenster–type first-line therapy between April 1990 and December 2003. The recommended salvage strategy was reinduction chemotherapy followed by autologous hematopoietic stem-cell transplantation (SCT). Results With a median follow-up time of 8.4 years (range, 4.5 to 16.4 years), the 5-year overall survival (OS) rate after first relapse was 57% ± 6%. Survival correlated with time of relapse and clinically advanced dissemination. Five-year OS of 16 patients who experienced progression during first-line therapy was 25% ± 11% compared with 66% ± 6% for 58 patients with a later relapse (P = .002). Five-year OS of 11 patients with bone marrow or CNS involvement was 27% ± 13% compared with 62% ± 6% for 63 patients without involvement (P = .001). Five-year event-free survival (EFS) and OS of 39 children who received the recommended autologous SCT were 59% ± 8% and 77% ± 7%, respectively. EFS after autologous SCT was significantly associated with time to relapse (progression: n = 3; EFS, 0; later relapse: n = 36; EFS, 64% ± 8%; P = .014) and CD3 expression (CD3 negative: n = 25; EFS, 72% ± 9%; CD3 positive: n = 11; EFS, 18% ± 12%; P < .001), but not with site of relapse, conditioning regimen, or graft manipulation. No relapses occurred among 10 patients with relapsed CD3-positive ALCL treated with allogeneic SCT. Conclusion Reinduction chemotherapy followed by autologous SCT proved feasible and efficacious for patients with a first relapse of CD3-negative ALCL after first-line therapy. Patients with progression during first-line therapy or relapsed CD3-positive ALCL may benefit from allogeneic SCT.

scholarly journals Progressive or Relapsed Burkitt Lymphoma or Leukemia in Children and Adolescents after BFM-type First-line Therapy

Blood ◽  
2020 ◽  
Author(s):  
Wilhelm Woessmann ◽  
Martin Zimmermann ◽  
Andrea Meinhardt ◽  
Stephanie Mueller ◽  
Holger Hauch ◽  
...  
Keyword(s):  
Risk Factors ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Burkitt Lymphoma ◽  
Initial Therapy ◽  
Front Line ◽  
First Line ◽  
First Line Therapy ◽  
Line Therapy

Children with refractory or relapsed Burkitt lymphoma have a poor chance to survive. We describe characteristics, outcome, re-induction and transplantation-approaches and evaluate risk factors among children with progression of a Burkitt lymphoma/leukemia included in NHL-BFM studies between 1986 and 2016. Treatment recommendation was re-induction including rituximab from the early 2000s followed by blood stem cell transplantation. The 3-year survival of the 157 children was 18.5{plus minus}3%. Survival significantly improved from 11{plus minus}3% before to 27{plus minus}5% after 2000 (p&lt;.001) allowing for risk factor analyses among the latter 75 patients, of whom 28 had disease progressive during initial therapy. Survival of 14 patients with relapse after initial therapy for low risk disease (R1/R2) was 50{plus minus}13% compared to 21{plus minus}5% for 61 patients progressing after R3/R4-therapy (p&lt;.02). 25 of 28 patients with progression during first-line therapy, 31 of 32 with progression during re-induction, 15 of 16 not reaching a complete remission before transplantation, 9 of 10 treated with rituximab front-line and all 13 patients not receiving rituximab during re-induction died. 46 patients received stem cell transplantation (20 autologous, 26 allogeneic). Survival after a regimen combining Rituximab with continuous-infusion chemotherapy followed by allogeneic transplantation was 67{plus minus}12% compared to 18{plus minus}5% for all other regimen and transplantations (p=.003). Patients with relapsed Burkitt lymphoma/leukemia have a poor chance to survive after current effective front-line therapies. Progression during initial or re-induction chemotherapy and initial high-risk disease are risk-factors in relapse. Time-condensed continuous-infusion re-induction followed by stem cell transplantation forms the basis for testing new drugs.

scholarly journals Use of crizotinib for refractory ALK-positive lymphomas

2017 ◽  
Vol 89 (7) ◽  
pp. 51-56 ◽  
Author(s):  
L N Shelikhova ◽  
V V Fominykh ◽  
D S Abramov ◽  
N V Myakova ◽  
M A Maschan ◽  
...  
Keyword(s):  
Pediatric Patients ◽  
Cell Lymphoma ◽  
Large Cell ◽  
First Line ◽  
Hematopoietic Stem ◽  
First Line Therapy ◽  
Line Therapy ◽  
Positron Emission ◽  
Before And After ◽  
Alk Positive

Aim. To evaluate the safety and efficacy of crizotinib used in pediatric patients with relapsed or refractory ALK-positive anaplastic large-cell lymphoma (ALCL). Subjects and methods. The paper describes the experience with crizotinib used in 8 patients with refractory ALK-ALCL before and after allogeneic hematopoietic stem cell transplantation (HSCT). Results. All the 8 (100%) patients treated with crizotinib were recorded to have complete responses, including complete metabolic ones (tumor disappearance as evidenced by positron emission tomography (PET)/computed tomography. Conclusion. Low and manageable toxicity of crizotinib and complete PET-negative responses in patients with resistant ALK lymphomas favor the need to test the drug as first-line therapy, by possibly decreasing the intensification of chemotherapy.

Continuous intravenous infusion Rh-endostatin in combination with dacarbazine and cisplatin as the first-line therapy for metastatic melanoma.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e21007-e21007
Author(s):  
Chuanliang Cui ◽  
BIN LIAN ◽  
Xuan Wang ◽  
Zhihong Chi ◽  
Lu Si ◽  
...  
Keyword(s):  
Continuous Infusion ◽  
Metastatic Melanoma ◽  
Intravenous Infusion ◽  
Objective Response ◽  
Stage Iv ◽  
Advanced Melanoma ◽  
Drug Discontinuation ◽  
First Line ◽  
First Line Therapy ◽  
Line Therapy

e21007 Background: Immunotherapy and targeted therapy have dramatically improved the survival of advanced melanoma patients (pts), but they show relative lower efficacy in Asian pts especially in acral and mucosal subtypes. Rh-Endostatin (endostar) is a potent endogenous inhibitor of angiogenesis. Previous studies have indicated that endostar combined with dacarbazine (DTIC) was effective in the treatment of metastatic melanoma. To further improve the effectiveness, this study was designed to observe the efficacy and safety of continuous infusion (CIV) of endostar combined with DTIC and cisplatin as the first line therapy for advanced melanoma pts. Methods: Pts with treatment naive, ECOG 0/1, and unresectable stage IIIC or IV melanoma were enrolled. DTIC (250 mg/m2, intravenous infusion, day 1-5), cisplatin (75 mg/m2, intravenous infusion, separated in 3 days) and endostar (15 mg/m2, CIV, day 1-14) were administered in a 28-day cycle until disease progression or intolerable toxicity. The primary endpoint was progression free survival (PFS). Secondary endpoints included disease control rate (DCR) and safety. Results: From January 2016 to May 2018, 64 pts were enrolled and 50 pts were evaluable. 26 pts were female. The median age was 50 years (range 28-71 years) old. 10/64 (15.6%) were primary mucosal and 21/64 (32.8%) were primary acral, 40 pts were at stage IV and 32% pts got BRAF mutation. At the last follow up of Dec 2018, 5 pts achieved partial response and 27 pts got stable disease. The objective response rate was 10%. The DCR was 64%. The median PFS was 6.0 months (95% CI 1.7-10.3 months). The median overall survival was not reached. The most common adverse events were nausea (56.25%), vomiting (31.25%) and leucopenia (29.7%). Grade 3-4 toxicity was few, one got intermittent palpitation and one got atrial fibrillation, which caused drug discontinuation and recovered to normal. Conclusions: Continuous infusion of endostar plus DTIC and cisplatin improved median PFS as the first line therapy for advanced melanoma. This combination therapy was well tolerated and might be recommended as the first line therapy for advanced melanoma. Clinical trial information: NCT03095079.

scholarly journals Outcomes and Treatment Patterns of Patients with CML in an Inner-City, Underserved, Multi-Ethnic Patient Cohort Reveals Good Overall Survival with Both First and Second Generation TKIs As Initial Therapy

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 5522-5522
Author(s):  
Amer Assal ◽  
Ramadevi Medavarapu ◽  
Ellen W Friedman ◽  
Yiting Yu ◽  
Amit Verma ◽  
...  
Keyword(s):  
Overall Survival ◽  
Inner City ◽  
Second Generation ◽  
Chronic Phase ◽  
Treatment Patterns ◽  
Front Line ◽  
First Line ◽  
2Nd Generation ◽  
First Line Therapy ◽  
Line Therapy

Abstract Introduction: Recent approval of second generation tyrosine kinase inhibitors (TKI) in chronic myelogenous leukemia (CML) has raised issues about their efficacy and pharmaco-economic utility in front line therapy of CML. We aimed to study these issues as well as reasons for switching TKIs in a real world setting of inner-city, multi-ethnic, underserved patient populations. Methods: We conducted an analysis of CML treatment and outcomes in an inner city cohort in the Bronx, NY. We identified 149 confirmed cases of CML that were treated over the last 17 years. All chart review was conducted by one of the study authors and discrepancies were reviewed by at least two of the authors. Data were analyzed using Chi-squared, t-testing for unpaired samples, and analysis of variance (ANOVA) to determine significance. Mortality was analyzed using Kaplan-Meir curves. Results: Demographics and Presentation:The mean age at time of presentation was 50 years (range 10 - 89 years, n=124). Average follow-up time was 5.1 (± 3.9) years. The cohort was minority rich and included 38.9% Hispanic, 32.9% African American, 18.8% Caucasian, 6.7% Asian and 2.7% multi-ethnic. The majority of the patients had private insurance or Medicare (62.4%), followed by Medicaid (30.9%) and emergency Medicaid (6.7%). The majority of evaluable patients presented in chronic phase (96.1%). Treatment Patterns: Front-line therapy was a 1st generation TKI (imatinib) in the majority of evaluable patients (83.9%) followed by a 2nd generation TKI (dasatinib or nilotinib) (11%). Rates of imatinib use as first line therapy were similar for both Private/Medicare (82.1%) and Medicare (88.1%) patients. Interestingly, a higher percentage of patients with Emergency Medicaid were started on a 2nd generation TKI (22.2%) as opposed to Private/Medicare and Medicaid (9.0% and 11.9% respectively). This was due to the availability of patient assistance programs to pay for TKIs. After the approval of dasatinib and nilotinib in 2008, there was increased use of the 2nd generation TKIs as first line though a majority of patients were still treated with imatinib as first line (72.2%, 80.0%, and 66.7% for Private/Medicare, Medicaid and Emergency Medicaid respectively). Outcomes:At the time of conclusion of the study, a total of 31 patients had expired. Twenty patients were evaluable for therapy at the time of death, percentages of patients on 1st, 2nd and 3rd line therapy were 10%, 50%, and 25% respectively. Allogeneic stem cell transplant was performed in 15% of expired patients. Of evaluable live patients (n=109), 45.9% were receiving the original front line therapy, whereas 37.6% and 16.5% were receiving second and third line therapies respectively. Regarding reasons for switch from a one TKI to another, we noted 34 patients who had been switched from a 1st to a 2nd generation TKI. Progression of disease was the cause in 53.0% of cases, followed by lack of response (17.6%), adverse events (14.7%), cytopenias (8.8%) and intolerance (5.9%). Two patients were switched from a 2nd generation TKI to a 1st generation, and both cases were due to intolerance. Overall survival of the cohort at 2 and 5 years was 94% and 82% respectively. No differences in survival were detected between different ethnicities, gender and age groups. Emergency Medicaid patients had a poorer overall survival compared to other insurance types though this did not reach statistical significance (p=0.0928). Interestingly, overall survival was similar for patients treated with 1st of 2nd generation TKIs as first line therapy (Median survival not reached, Log rank P value =0.4). Discussion: We analyzed CML outcomes and treatment patterns in a large inner city underserved multiethnic cohort in the Bronx. The CML population reflected the ethnic composition of the borough which is predominantly Hispanic and African American. The majority of patients presented in chronic phase CML which is in agreement with other studies. Majority of patients were started on imatinib as first line therapy and had similar overall survival to those that were started on Dasatinib or Nilotinib. The majority of patients who were switched from a first generation TKI to a second generation TKI were due to relapse or lack of adequate response. Our results suggest that TKIs are successfully used in real world populations and are leading to high overall survival even with use of imatinib as first line therapy. Disclosures No relevant conflicts of interest to declare.

scholarly journals A Multicenter, Prospective Study for Pediatric Chronic Myeloid Leukemia in Chronic Phase: The JPLSG CML-08 Study

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3605-3605
Author(s):  
Hiroyuki Shimada ◽  
Akihiro Watanabe ◽  
Masaki Ito ◽  
Chikako Tono ◽  
Haruko Shima ◽  
...  
Keyword(s):  
Children And Adolescents ◽  
Myeloid Leukemia ◽  
Second Generation ◽  
Chronic Phase ◽  
Line Treatment ◽  
First Line ◽  
First Line Therapy ◽  
Line Therapy ◽  
First Line Treatment

Abstract Background: Tyrosine kinase inhibitor (TKI) has been used in pediatric chronic myeloid leukemia (CML) for more than 10 years, but only a few prospective clinical studies have been conducted in pediatric patients with CML due to their rarity. We conducted the JPLSG CML-08 study to determine the efficacy and tolerability of TKIs in children and adolescents with newly diagnosed CML in chronic phase (CML-CP). Methods: The JPLSG CML-08 study was a prospective multicenter observational study (UMIN000002581). Patients under 18 years of age with untreated BCR-ABL1-positive CML-CP were eligible and treated according to the modified ELN-2009 recommendation, and the efficacy and safety of TKIs were evaluated. Results: From October 2009 until September 2014, 79 patients were enrolled in 46 hospitals in Japan. A total of 78 patients (49 males and 29 females) were eligible for inclusion. Median age at diagnosis was 11 years (range, 1-17). Median observational period for survivors was 82 months (range, 48-118). Median WBC, Hb and platelet counts were 275x10 9/L (range, 8-765), 9.6g/dL (range, 5.8-14.6) and 560x10 9/L (range, 110-2875), respectively. Splenomegaly was found in 76%. High risk of Sokal, Hasford, EUTOS, and ELTS scores were observed in 21, 13, 27, and 9%, respectively. Clonal chromosome abnormalities in Ph-positive cells occurred in 1 patient at diagnosis. Imatinib, dasatinib, and, nilotinib were used as a first-line treatment in 69 (88%), 7 (9%), and 2 (3%) patients, respectively. The median initial dose of imatinib, dasatinib, and nilotinib was 276, 63, and 262mg/m2, respectively. 5y-PFS and OS was 96.2% (95%CI, 88.6 to 98.7%) and 97.4% (95%CI, 90.1 to 99.4%), respectively. Deaths were observed in 2 patients due to transplant complications. Hematopoietic cell transplantation was conducted in 14 patients (18%). Nine patients (12%) discontinued TKI with the aim of treatment-free remission (TFR), and five of them achieved TFR. In 69 patients with first-line imatinib, complete hematologic response was achieved in 95.7% at 3 months, complete cytogenetic response in 75.4% at 12 months, major molecular response (MMR) in 40.1% at 18 months, and MR4.0 in 52.8% at 60 months; If a transplant was performed, the follow-up period was censored at the date of transplant. Of the 69 patients, 52% changed treatment from imatinib to another TKI or transplant due to poor response, and 20% did due to intolerance. The most common cause of intolerance to imatinib was musculoskeletal events. BCR-ABL1 (IS) &lt;10% at 3 months strongly correlated with higher achievement of MMR, MR4.0, and MR4.5. The EUTOS score was significantly associated with achievement of IS &lt;10% at 3 months. Patients with a first-line second-generation TKI had a higher cumulative incidence of MR4.5 (P = 0.0191) than patients with a first-line imatinib. Second-generation TKI was used as first-line therapy only in patients older than 9 years, but other clinical characteristics, including risk scores, did not differ significantly between the two groups. The incidence of grade 3/4 adverse events (≥ 10%) included neutropenia (47%), anemia (39%), leukopenia (13%), arthralgia (13%), and myalgia (11%) for imatinib, neutropenia (21%), anemia (13%), and thrombocytopenia (11%) for dasatinib, and neutropenia (14%), elevated ALT (14%), hyperbilirubinemia (14%), skin rash (14%), and high CPK (14%) for nilotinib. Gastrointestinal bleeding was an adverse event specific to dasatinib (11% in all grades). Conclusion: This clinical study extends and confirms previous data showing that first-line treatment with imatinib is effective in children and adolescents, with response rates similar to those seen in adults. Although longer follow-up is needed to fully assess the long-term toxic effects, adverse events with imatinib, dasatinib, and nilotinib have been acceptable. As reported in adults, there was an advantage in selecting second-generation TKI over imatinib as first-line therapy to achieve deep molecular remission (DMR). Since discontinuation of TKI after achieving DMR is the preferred strategy, second-generation TKI is expected to become the standard therapy for children and adolescents. Disclosures No relevant conflicts of interest to declare.

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