Survival and prognostic factors in a series of adults with medulloblastomas

2009 ◽  
Vol 111 (3) ◽  
pp. 478-487 ◽  
Author(s):  
Laurent Riffaud ◽  
Stephan Saikali ◽  
Emmanuelle Leray ◽  
Abderrahmane Hamlat ◽  
Claire Haegelen ◽  
...  
Keyword(s):  
Overall Survival ◽  
Prognostic Factors ◽  
High Risk ◽  
Survival Time ◽  
Risk Group ◽  
Survival Rates ◽  
Event Free Survival ◽  
Term Survival ◽  
Free Survival ◽  
Standard Risk

Object In this article, the authors report their experience in the management of adult patients with medulloblastoma at their institution to identify prognostic factors important for survival and disease control. Methods Between 1977 and 2005, 27 patients who were ≥ 16 years old and had medulloblastoma were treated consecutively. There were 16 women and 11 men with a median age of 21 years (range 16–54 years). Gross-total resection was performed in 21 patients, subtotal (≥ 90%) in 2, incomplete in 1, and biopsy in 3 patients. Six patients had the desmoplastic variant, and 21 patients presented with classic medulloblastoma. Staging according to the Chang classification showed 4 patients with tumors invading the brainstem (2 with Stage T3b and 2 with Stage T4), 3 patients with metastases (2 with Stage M2 and 1 with Stage M3), and 1 patient in whom the stage was unknown (Stage MX) who died 10 days postoperatively. Twenty patients were assigned to the standard-risk group and 7 to the high-risk group. All patients except the one whose status was classified as Stage MX underwent craniospinal radiotherapy at our institution. Seven patients received chemotherapy before radiotherapy. Results The 5- and 10-year overall survival rates for the present study were 81 and 62%, respectively. The median overall survival time was 17.7 years. The 5- and 10-year event-free survival rates were 72 and 57%, respectively. The median event-free survival time was 17.9 years. Univariate analysis showed that survival was significantly correlated with sex (women had a better prognosis than men) and M stage (patients without metastases had a better outcome). Patient age, duration of symptoms, Karnofsky Performance Scale score at presentation, hydrocephalus, tumor location, brainstem invasion, extent of resection, histological subtype, preradiotherapy chemotherapy, risk group, and period of presentation were not significant variables. Multivariate analysis identified sex and M stage as well as the period of presentation as independent prognostic factors for overall and event-free survival times. Eleven patients suffered tumor recurrence within a median time of 4.2 years. The posterior fossa was not the most common site of recurrence, and delayed recurrence was not rare. All patients in whom the tumor recurred have died despite aggressive treatments. The median survival time after diagnosis of recurrence was 2.5 years. Questionnaires on quality of life and cognition showed high scores in favor of limited negative effects in the perception of mental and physical health after treatment. The authors observed 1 supposed second malignancy (thyroid carcinoma) and no evidence of pituitary dysfunction. Conclusions Long-term survival is possible in adults treated for medulloblastoma. Although rare, metastasis seeding at presentation is a poor prognostic factor. The possibility of delayed recurrence necessitates close follow-up of all patients. Tumor recurrences should be treated with aggressive therapies as some patients may have sustained response. Adjuvant chemotherapy should be given to high-risk patients, but its role in reducing recurrences, particularly distant ones, remains unclear in the standard-risk group.

Prerequisites to improvement of predictive model of childhood Hodgkin’s lymphoma

2015 ◽  
Vol 6 (4) ◽  
pp. 13-18
Author(s):  
Svetlana Aleksandrovna Kulyova ◽  
Andrei Petrovich Karitsky ◽  
Svetlana Viacheslavovna Ivanova
Keyword(s):  
Overall Survival ◽  
Hodgkin’S Lymphoma ◽  
Roc Analysis ◽  
Risk Group ◽  
Survival Rates ◽  
Late Results ◽  
Tumor Burden ◽  
Hodgkin's Lymphoma ◽  
Event Free Survival ◽  
Free Survival

Background. Calculation of relative tumor burden in Hodgkin’s lymphoma patients is the simplest and significant parameter which can be used in daily clinical practice as a risk factor. The aim of study was the assessment of influence of relative tumor burden on the late results of a disease. Material and methods. This research included data on 126 patients with Hodgkin’s lymphoma aged from 0 till 18 years (middle age of 11 years), treated risk-adapted treatment according to the DAL-HD and SPBLH-05. Boys was 70, girls - 56 (a ratio 1,25 : 1). Fifty-eight patients (46 %) are stratified in favorable risk group, 50 (39,7 %) - in intermediate risk group, and 50 (39,7 %) are included in unfavorable risk group. Results. Overall survival at 5 years was 93 % (range 91-95 %), event-free survival - 88 % (85-91 %). The average relative tumor burden was 129,4 cm3/m2 (7-609,7 cm3/m2). When carrying out ROC-analysis value of 122,7 cm3/m2 (р ˂ 0,0001) appeared the critical parameter, which worsen the prognosis of a disease. Overall survival in a patients cohort with this volume was 69,6 %, with the volume less than 122,7 cm3/m2 overall survival was 97,2 % (р = 0,00002). Conclusions. The relative tumor burden is the parameter which is significantly reducing survival rates in children with Hodgkin’s lymphoma. Opinion on interrelation of clinical and laboratory parameters with “sarkoma’s saturation” or tumor volume as end result of immunological frustration, it is represented the most perspective direction of studying of Hodgkin’s lymphoma.

scholarly journals Pediatric-Based Versus Adult Treatment Protocols in Young Adults (18-40 years) with Standard Risk Acute Lymphoblastic Leukemia: The BC Cancer Agency Experience

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 3770-3770 ◽  
Author(s):  
David Simon Kliman ◽  
Michael J Barnett ◽  
Raewyn Broady ◽  
Donna L. Forrest ◽  
Alina S. Gerrie ◽  
...  
Keyword(s):  
British Columbia ◽  
Overall Survival ◽  
Acute Lymphoblastic Leukemia ◽  
Lymphoblastic Leukemia ◽  
Survival Rates ◽  
High Dose ◽  
Event Free Survival ◽  
Free Survival ◽  
Small Cohort ◽  
Standard Risk

Abstract Introduction In recent decades, overall survival rates for children with acute lymphoblastic leukemia (ALL) have improved dramatically. Unfortunately, older patients have not experienced the same benefit. Recent years have seen investigation into the use of pediatric protocols for younger adults with ALL. Increased toxicity has often limited use to patients 40 years or younger. The Leukemia/Bone Marrow Transplant Program of BC is the referral center for adults with ALL in British Columbia. Approximately 20 patients are newly diagnosed with ALL each year. Until 2008, an adult protocol (known as ALL 89-1) was used in patients over 18 years. Since 2008, pediatric-based chemotherapy has been offered to patients 40 years or younger. We analyzed whether this change altered complete remission (CR), relapse and survival rates. We assessed whether the more intense protocol increased toxicity. Methods A retrospective analysis was performed on patients treated for ALL on a pediatric-based protocol. These protocols (ALL 08-01 and ALL 13-01) were modifications of the Donna Farber Cancer Institute 01-175 protocol. The format included induction with high dose methotrexate, followed by central nervous system (CNS) therapy including cranial irradiation. A single cycle of etoposide and high-dose cytarabine was given. Consolidation and continuation cycles included doxorubicin, asparaginase, vincristine and dexamethasone. Patients were included if they were aged 18-40 years and had standard risk, Philadelphia chromosome negative ALL. Between February 2008 and November 2014, 25 eligible patients were identified. These were compared with the 23 consecutive standard risk patients most recently treated with ALL 89-1. They had been diagnosed between February 2003 and July 2008. Exclusion criteria were age greater than 40 and non-standard risk ALL. Demographic and clinical data were collected on all patients from the Leukemia Program databases. Overall survival (OS) was calculated from time of diagnosis until death. Event free survival (EFS) was calculated from diagnosis until death, induction failure or relapse. Estimation of OS and EFS was performed using the Kaplan-Meier method. Patient characteristics were compared using Chi-squared test or Fisher's exact test. Ethics approval was obtained from the University of British Columbia ethics board. Results The median age of the combined patient group was 24.5 years. There were no statistically significant differences pre-treatment between groups. Combined median follow up was 28.7 months All 25 patients receiving a pediatric protocol achieved a CR, compared to 19 of 22 with the adult protocol. Despite the more intense chemotherapy dosing regimen in the pediatric protocol, there was no increase in hospitalizations, invasive fungal infections or deaths from treatment toxicity (Table 1). There was a trend towards increased thrombotic events in the pediatric-treated group, at 32% versus 9%. These included deep vein thrombosis in 4 patients, pulmonary emboli in 2, and cerebral sinus thrombosis in 2. Relapse occurred in 24% of the pediatric-treated patients and 45% of the adult-treated ones (p=0.215). Allogeneic stem cell transplantation was performed in 4 patients in the former group and 7 in the latter. Nine of these were carried out in CR2 or later, with two patients going into transplant with active disease. Overall survival following transplant was 44%. Two year event free survival was significantly improved in the group treated on the pediatric protocol (Figure 1), at 79% versus 36% (p=0.011). There was a trend towards improved overall survival in this small cohort, at 83% versus 49% (Figure 2). Conclusions A pediatric-based ALL treatment protocol was tolerated in patients up to the age of 40 years. In our centre, this is associated with an increase in EFS, and a trend towards increased OS, even considering the small cohort. We await with interest the results of larger studies investigating the ideal upfront therapy for young patients with ALL. Table 1. Results All Patients N=47 Adult N=22 Pediatric N=25 P Number % Number % Number % CR after induction 44 of 47 94 19 of 22 86 25 of 25 100 .095 Severe infection 20 of 47 43 9 of 22 41 11 of 25 44 .831 Thrombosis 10 of 47 21 2 of 22 9 8 of 25 32 .079 Pancreatitis 2 of 47 4 0 of 22 0 2 of 25 8 .491 Toxicity deaths 3 of 47 6 2 of 22 9 1 of 25 4 .593 Relapse 16 of 47 34 10 of 22 45 6 of 25 24 .215 AlloHSCT 11 of 47 23 7 of 22 32 4 of 25 16 .303 Disclosures No relevant conflicts of interest to declare.

Revised Neuroblastoma Risk Classification System: A Report From the Children's Oncology Group

2021 ◽  
pp. JCO.21.00278
Author(s):  
Meredith S. Irwin ◽  
Arlene Naranjo ◽  
Fan F. Zhang ◽  
Susan L. Cohn ◽  
Wendy B. London ◽  
...  
Keyword(s):  
Overall Survival ◽  
High Risk ◽  
Risk Group ◽  
Staging System ◽  
Risk Classification ◽  
Intermediate Risk ◽  
Oncology Group ◽  
Event Free Survival ◽  
Free Survival ◽  
Pathology Classification

PURPOSE Treatment planning for children with neuroblastoma requires accurate assessment of prognosis. The most recent Children's Oncology Group (COG) risk classification system used tumor stage as defined by the International Neuroblastoma Staging System. Here, we validate a revised classifier using the International Neuroblastoma Risk Group Staging System (INRGSS) and incorporate segmental chromosome aberrations (SCA) as an additional genomic biomarker. METHODS Newly diagnosed patients enrolled on the COG neuroblastoma biology study ANBL00B1 between 2007 and 2017 with known age, International Neuroblastoma Staging System, and INRGSS stage were identified (N = 4,832). Tumor MYCN status, ploidy, SCA status (1p and 11q), and International Neuroblastoma Pathology Classification histology were determined centrally. Survival analyses were performed for combinations of prognostic factors used in COG risk classification according to the prior version 1, and to validate a revised algorithm (version 2). RESULTS Most patients with locoregional tumors had excellent outcomes except for those with image-defined risk factors (INRGSS L2) with MYCN amplification (5-year event-free survival and overall survival: 76.3% ± 5.8% and 79.9% ± 5.5%, respectively) or patients age ≥ 18 months with L2 MYCN nonamplified tumors with unfavorable International Neuroblastoma Pathology Classification histology (72.7% ± 5.4% and 82.4% ± 4.6%), which includes the majority of L2 patients with SCA. For patients with stage M (metastatic) and MS (metastatic, special) disease, genomic biomarkers affected risk group assignment for those < 12 months ( MYCN) or 12-18 months ( MYCN, histology, ploidy, and SCA) of age. In a retrospective analysis of patient outcome, the 5-year event-free survival and overall survival using COG version 1 were low-risk: 89.4% ± 1.1% and 97.9% ± 0.5%; intermediate-risk: 86.1% ± 1.3% and 94.9% ± 0.8%; high-risk: 50.8% ± 1.4% and 61.9% ± 1.3%; and using COG version 2 were low-risk: 90.7% ± 1.1% and 97.9% ± 0.5%; intermediate-risk: 85.1% ± 1.4% and 95.8% ± 0.8%; high-risk: 51.2% ± 1.4% and 62.5% ± 1.3%, respectively. CONCLUSION A revised 2021 COG neuroblastoma risk classifier (version 2) that uses the INRGSS and incorporates SCAs has been adopted to prospectively define COG clinical trial eligibility and treatment assignment.

scholarly journals Arsenic Combined With All-Trans Retinoic Acid for Pediatric Acute Promyelocytic Leukemia: Report From the CCLG-APL2016 Protocol Study

2021 ◽  
pp. JCO.20.03096
Author(s):  
Huyong Zheng ◽  
Hui Jiang ◽  
Shaoyan Hu ◽  
Ning Liao ◽  
Diying Shen ◽  
...  
Keyword(s):  
High Risk ◽  
Pediatric Patients ◽  
Risk Group ◽  
High Risk Group ◽  
Event Free Survival ◽  
Free Survival ◽  
Trans Retinoic Acid ◽  
All Trans Retinoic Acid ◽  
Standard Risk

PURPOSE Arsenic combined with all-trans retinoic acid (ATRA) is the standard of care for adult acute promyelocytic leukemia (APL). However, the safety and effectiveness of this treatment in pediatric patients with APL have not been reported on the basis of larger sample sizes. METHODS We conducted a multicenter trial at 38 hospitals in China. Patients with newly diagnosed APL were stratified into two risk groups according to baseline WBC count and FLT3-ITD mutation. ATRA plus arsenic trioxide or oral arsenic without chemotherapy were administered to the standard-risk group, whereas ATRA, arsenic trioxide, or oral arsenic plus reduced-dose anthracycline were administered to the high-risk group. Primary end points were event-free survival and overall survival at 2 years. RESULTS We enrolled 193 patients with APL. After a median follow-up of 28.9 months, the 2-year overall survival rate was 99% (95% CI, 97 to 100) in the standard-risk group and 95% (95% CI, 90 to 100) in the high-risk group ( P = .088). The 2-year event-free survival was 97% (95% CI, 93 to 100) in the standard-risk group and 90% (95% CI, 83 to 96) in the high-risk group ( P = .252). The plasma levels of arsenic were significantly elevated after treatment, with a stable effective level ranging from 42.9 to 63.2 ng/mL during treatment. In addition, plasma, urine, hair, and nail arsenic levels rapidly decreased to normal 6 months after the end of treatment. CONCLUSION Arsenic combined with ATRA is effective and safe in pediatric patients with APL, although long-term follow-up is still needed.

Finding an optimal treatment regimen for endemic Burkitt's lymphoma in a limited resource setting: A historical comparison of an INCTR-03-06 protocol for children treated at a tertiary pediatric oncology unit in Tanzania.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e22507-e22507
Author(s):  
Shakilu Jumanne Kayungo
Keyword(s):  
Overall Survival ◽  
High Risk ◽  
Supportive Care ◽  
Advanced Disease ◽  
Survival Rates ◽  
Phone Call ◽  
Mortality Data ◽  
Event Free Survival ◽  
Free Survival ◽  
Risk Of Death

e22507 Background: Adoption of intensive chemotherapy for endemic Burkitt lymphoma (eBL) in low income settings such as the French LMB-protocol with survival rates over 90% is limited by unavailability of quality supportive care for treatment related toxicity. Late stage at diagnosis and comorbidities such as HIV, Malnutrition and other socioeconomic challenges complicate this quagmire. Review the nonclinical trial setting performance of a three-drug regimen previously reported by the INCTR-03-06 study among children treated for eBL from 2012 to 2017 at a National Hospital in Dar es Salaam Tanzania. Methods: Medical record review of children treated for eBL to document demographic, clinical, chemotherapy regimen and outcome was conducted followed by phone call structured interviews for current survival status. Overall survival (OS) and Event Free Survival (EFS) were determined by Kaplan-Meier method and survival differences compared using the Log-rank test. Cox regression analysis was used to relate variables with mortality. Data analysis was done by SPSS version 20 and p-value of < 0.05 considered statistically significant. Results: Out of the 123 children enrolled, male to female ratio was 1.6:1, median age 7 years (IQR 4 -10). Abdominal disease was the commonest presentation, 39% (48/123) and 25.2% (31/123) jaw mass. On phone call interviews 39.84% (49/123) of the children were in remission, 33.33%(41/123) died while 26.83% (33/123) were lost to follow up. Complete tumor response was achieved in 55.9% (62/111) of children treated on 1st line chemotherapy. Overall Survival rates (OS) at 12 and 18 months were 63.4% and 54% respectively while Event-Free Survival (EFS) were 38.8% and 36.4% respectively. HIV infected children (aHR 5.12 95% CI 1.39- 19.0; log-rank P< 0.01). and those with advanced disease at diagnosis( aHR2.34 95% CI 1.09-5.05 P < 0.03) were at high risk of death. Conclusions: Contrary to prior understanding of eBL as a predominantly jaw disease, abdominal site is increasingly reported likely due to widespread use of sensitive imaging modalities. In line with other studies in low- and middle-income countries, few children with eBL achieve complete remission using low intensity chemotherapy regimens calling for more studies for safe and feasible regimens to be used as efforts to improve supportive care continue. HIV infected children with eBL and those with advanced disease at diagnosis should be considered at high risk of mortality.

Long-Term Survival Results of a Randomized Trial Comparing Gemcitabine Plus Cisplatin, With Methotrexate, Vinblastine, Doxorubicin, Plus Cisplatin in Patients With Bladder Cancer

2005 ◽  
Vol 23 (21) ◽  
pp. 4602-4608 ◽  
Author(s):  
Hans von der Maase ◽  
Lisa Sengelov ◽  
James T. Roberts ◽  
Sergio Ricci ◽  
Luigi Dogliotti ◽  
...  
Keyword(s):  
Overall Survival ◽  
Prognostic Factors ◽  
Locally Advanced ◽  
Survival Rates ◽  
Progression Free Survival ◽  
Term Survival ◽  
Free Survival ◽  
Visceral Metastases ◽  
Overall Survival Rates

Purpose To compare long-term survival in patients with locally advanced or metastatic transitional cell carcinoma (TCC) of the urothelium treated with gemcitabine/cisplatin (GC) or methotrexate/vinblastine/doxorubicin/cisplatin (MVAC). Patients and Methods Efficacy data from a large randomized phase III study of GC versus MVAC were updated. Time-to-event analyses were performed on the observed distributions of overall and progression-free survival. Results A total of 405 patients were randomly assigned: 203 to the GC arm and 202 to the MVAC arm. At the time of analysis, 347 patients had died (GC arm, 176 patients; MVAC arm, 171 patients). Overall survival was similar in both arms (hazard ratio [HR], 1.09; 95% CI, 0.88 to 1.34; P = .66) with a median survival of 14.0 months for GC and 15.2 months for MVAC. The 5-year overall survival rates were 13.0% and 15.3%, respectively (P = .53). The median progression-free survival was 7.7 months for GC and 8.3 months for MVAC, with an HR of 1.09. The 5-year progression-free survival rates were 9.8% and 11.3%, respectively (P = .63). Significant prognostic factors favoring overall survival included performance score (> 70), TNM staging (M0 v M1), low/normal alkaline phosphatase level, number of disease sites (≤ three), and the absence of visceral metastases. By adjusting for these prognostic factors, the HR was 0.99 for overall survival and 1.01 for progression-free survival. The 5-year overall survival rates for patients with and without visceral metastases were 6.8% and 20.9%, respectively. Conclusion Long-term overall and progression-free survival after treatment with GC or MVAC are similar. These results strengthen the role of GC as a standard of care in patients with locally advanced or metastatic TCC.

scholarly journals Escalating intravenous methotrexate improves event-free survival in children with standard-risk acute lymphoblastic leukemia: a report from the Children's Oncology Group

Blood ◽  
2011 ◽  
Vol 118 (2) ◽  
pp. 243-251 ◽  
Author(s):  
Yousif Matloub ◽  
Bruce C. Bostrom ◽  
Stephen P. Hunger ◽  
Linda C. Stork ◽  
Anne Angiolillo ◽  
...  
Keyword(s):  
Overall Survival ◽  
Acute Lymphoblastic Leukemia ◽  
High Risk ◽  
Lymphoblastic Leukemia ◽  
Oncology Group ◽  
Event Free Survival ◽  
Free Survival ◽  
Cancer Group ◽  
Leucovorin Rescue ◽  
Standard Risk

Abstract Children's Cancer Group-1991 selected 2 components from the Children's Cancer Group studies shown to be effective in high-risk acute lymphoblastic leukemia and examined them in children with National Cancer Institute standard-risk acute B-precursor lymphoblastic leukemia. These were (1) vincristine and escalating IV methotrexate (MTX) without leucovorin rescue during the interim maintenance (IM) phases and (2) addition of a second delayed intensification (DI) phase. Eligible patients (n = 2078) were randomly assigned to regimens containing either oral (PO) MTX, PO mercaptopurine, dexamethasone, and vincristine or IV MTX during IM phases, and regimens with either single DI or double DI. Five-year event-free survival (EFS) and overall survival for patients on the PO MTX arms were 88.7% ± 1.4% and 96% ± 0.9% versus 92.6% ± 1.2% and 96.5% ± 0.8% for those on the IV MTX arms (P = .009, P = .66). Five-year EFS and overall survival for patients who received single DI were 90.9% ± 1.3% and 97.1% ± 0.8% versus 90.5% ± 1.3% and 95.4% ± 3.8% for those who received double DI (P = .71, P = .12). No advantage was found for a second DI; however, replacement of PO MTX, PO mercaptopurine, vincristine, and dexamethasone during IM with vincristine and escalating IV MTX improved EFS.

Genome-Wide Genotype-Based Risk Model for Survival in Core Binding Factor Acute Myeloid Leukemia Patients

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 1623-1623
Author(s):  
Silvia Park ◽  
Choi Hangseok ◽  
Hee-Je Kim ◽  
Jae-Sook Ahn ◽  
Hyeoung Joon Kim ◽  
...  
Keyword(s):  
Overall Survival ◽  
High Risk ◽  
Risk Model ◽  
Risk Group ◽  
Snp Array ◽  
High Risk Group ◽  
Event Free Survival ◽  
Free Survival ◽  
Genome Wide ◽  
Binding Factor

Abstract Introduction: The present study attempted to build a single nucleotide polymorphism (SNP)-based risk model for predicting overall survival (OS) and event free survival (EFS) in patients with core binding factor acute myeloid leukemia (CBF-AML). Methods: Adopting genome-wide SNP array using Affymetrix SNP array 6.0, we analyzed 868,157 SNPs with respect to OS and EFS in 104 patients with CBF-AML. Significant SNPs were identified from single SNP analysis. The risk model was constructed with incorporation of six SNPs and three clinical factors (age, c-kit exon 17 mutation, and LDH) for OS, and six SNPs and three clinical factors (age, WBC, and LDH) for EFS. The model was further defined into low and high risk groups based on risk scores. Results: The median age was 39 years, and the subgroup of t(8;21) and inv(16) or t(16;16) was assessed in 68 (65.4%) and 36 patients (34.6%). Finally 6 SNPs per each OS (rs4353685, rs4908185, rs7709207, rs12034, rs1554844, and rs17241868) and EFS (rs13385610, rs11210617, rs11169282, rs7709207, rs4438401 and rs16894846) were incorporated into the risk model. OS was significantly different in favor of the low risk group (80.4%) compared to the high risk group (22.0% at 3 years; p= 8.75 x 10-13; HR 8.67). For EFS, there was also a significant difference between the low (75.0%) versus high risk group (17.1% at 3 years; p=5.95 x 10-13; HR 7.67). Conclusion: A genome-wide SNP based risk model can stratify CBF-AML patients according to their OS and EFS in 104 patients. Figure 1 Overall survival and event free survival by risk model composed of SNPs and clinical risk factors Figure 1. Overall survival and event free survival by risk model composed of SNPs and clinical risk factors Figure 2 Figure 2. Disclosures No relevant conflicts of interest to declare.

Chronic Myeloid Leukemia Patients In Tunisia: Epidemiology and Outcome In The Imatinib Era (A Multicentric Experience)

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 4037-4037
Author(s):  
Raihane Ben Lakhal ◽  
Hela Ghedira ◽  
Hatem Bellaaj ◽  
Yosra Ben Youssef ◽  
Samia Menif ◽  
...  
Keyword(s):  
Overall Survival ◽  
Developing Countries ◽  
High Risk ◽  
Myeloid Leukemia ◽  
Risk Group ◽  
Progression Free Survival ◽  
Molecular Response ◽  
Event Free Survival ◽  
Free Survival ◽  
Optimal Response

Abstract Background The introduction of the first targeted tyrosine kinase inhibitor (TKI), imatinib mesylate (IM) revolutionized the therapeutic paradigm and dramatically improved outcomes of chronic myeloid leukemia(CML). Data are limited in developing countries regarding the clinicopathologic features and response to therapy in the era of IM. Aims To report on the clinicoepidemiologic features of CML in Tunisia, to evaluate the long-term outcome of patients in chronic (CP) or accelerated phase (AP) treated with IM 400mg daily as frontline therapy and to determine imatinib’s efficacy and safety. Methods From October 2002 to December 2011, two hundred and ninety two CML patients were treated with IM in 6 Tunisian departments of hematology. Monitoring response was defined as the ELN provided guidelines. Response (Hematologic, cytogenetic and molecular responses), adverse events and outcome (overall survival, event free survival and progression free survival) were evaluated. The factors associated with outcome of IM therapy were also analyzed. Results Two hundred ninety-two patients enrolled with a median follow-up duration of 56 (8 -290) months: The median age was 44 years (3-78 years). One hundred and fifty (51.3%) patients were male, 134 (49%) were asymptomatic at diagnosis. Splenomegaly was present in 237 of 292 (81%). Additional cytogenetic abnormalities were encountred in 24 (8.3%) patients. At diagnosis, 271 (92.8%) patients were in CP, 21 (7.2%) were in AP. The Sokal risk was low in 64 (23%), intermediate in 94 (33.5%), and high in 122 patients (43.5%). The Eutos risk was low in 179 (75%), and high in 60 (25%) patients.The rate of cumulative complete hematologic response (CHR), complete cytogenetic response (CCyR), major molecular response (MMR) and molecular response 4/5 log (CMR) in CP/AP CML patients were 93.8%, 73%, 65% and 33.9%, respectively. According to the 2009 ELN criteria, optimal, suboptimal response and failure were noted in 132 (47%), 68 (24%) and 82 (29%) patients, respectively. Five year event free survival (EFS), progression free survival (PFS) and overall survival (OS) were 78%, 89% and 91%, respectively. By multivariate analyzis, AP, high Eutos risk and baseline WBC ≥ 150G/l remained independent predictive factors of non optimal response to IM. AP was an adverse independent prognostic factor for EFS, PFS and OS. Patients obtained CCyR at 12 months after the initiation of IM treatment were associated with longer PFS (P< 0.0001) and OS (P< 0.0001). ELN response was also significantly associated with EFS. The adverse events (AE) of IM were moderate and tolerable. Only 3 patients discontinued IM for intolerance. IM-related hematologic AE included neutropenia in 6.2%, anemia in 8.9%, and thrombocytopenia in 17.2%. Nonhematologic AE (21%), including mainly edema in 7.1%, digestive disorders in 5.5%, weight gain and skin rash in 3.1%. Conclusion We found that substantial number of patients in our series were in intermediate or high risk group. With the caveats that the monitoring of the disease was not optimal, response rates were similar to those reported in previous studies. It is clear to us that improvements should be made in treatment of AP-CML and high Sokal risk group of CP-CML. The front-line use of 2nd TKI are expected to improve the results of the first line treatment of these high risk Tunisian patients, but cost and accessibility of this therapy remain the problems in developing countries. Disclosures: No relevant conflicts of interest to declare.

Treatment of Pulmonary Metastases in Children With Stage IV Nephroblastoma With Risk-Based Use of Pulmonary Radiotherapy

2012 ◽  
Vol 30 (28) ◽  
pp. 3533-3539 ◽  
Author(s):  
Arnauld Verschuur ◽  
Harm Van Tinteren ◽  
Norbert Graf ◽  
Christophe Bergeron ◽  
Bengt Sandstedt ◽  
...  
Keyword(s):  
Overall Survival ◽  
Survival Rate ◽  
High Risk ◽  
Pulmonary Metastases ◽  
Stage Iv ◽  
Line Treatment ◽  
Event Free Survival ◽  
First Line ◽  
Free Survival ◽  
First Line Treatment

Purpose The purpose of this study was to determine the outcome of children with nephroblastoma and pulmonary metastases (PM) treated according to International Society of Pediatric Oncology (SIOP) 93-01 recommendations using pulmonary radiotherapy (RT) in selected patients. Patients and Methods Patients (6 months to 18 years) were treated with preoperative chemotherapy consisting of 6 weeks of vincristine, dactinomycin, and epirubicin or doxorubicin. If pulmonary complete remission (CR) was not obtained, metastasectomy was considered. Patients in CR received three-drug postoperative chemotherapy, whereas patients not in CR were switched to a high-risk (HR) regimen with an assessment at week 11. If CR was not obtained, pulmonary RT was mandatory. Results Two hundred thirty-four of 1,770 patients had PM. Patients with PM were older (P < .001) and had larger tumor volumes compared with nonmetastatic patients (P < .001). Eighty-four percent of patients were in CR postoperatively, with 17% requiring metastasectomy. Thirty-five patients (16%) had multiple inoperable PM and required the HR protocol. Only 14% of patients received pulmonary RT during first-line treatment. For patients with PM, 5-year event-free survival rate was 73% (95% CI, 68% to 79%), and 5-year overall survival (OS) rate was 82% (95% CI, 77% to 88%). Five-year OS was similar for patients with local stage I and II disease (92% and 90%, respectively) but lower for patients with local stage III disease (68%; P < .001). Patients in CR after chemotherapy only and patients in CR after chemotherapy and metastasectomy had a better outcome than patients with multiple unresectable PM (5-year OS, 88%, 92%, and 48%, respectively; P < .001). Conclusion Following the SIOP protocol, pulmonary RT can be omitted for a majority of patients with PM and results in a relatively good outcome.

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