Effectiveness of Three Prognostic Scoring Systems in Predicting the Response and Outcome in Pediatric Chronic Myeloid Leukemia Chronic Phase on Frontline Imatinib.

INTRODUCTION: The Sokal and Hasford (Euro) scores were developed in the chemotherapy and interferon eras and are widely used as prognostic indicators in patients with chronic myeloid leukemia (CML). Recently, European Treatment and Outcome Study (EUTOS) scoring system was introduced. Data on risk stratification in pediatric CML population was lacking due to its rarity (<3%). OBJECTIVE: To study the effectiveness in predicting the response and outcome with three prognostic scores in pediatric CML-chronic phase patients on front line Imatinib. Materials and Methods: We retrospectively analyzed the hospital records of newly diagnosed CML CP patients (aged ≤18 years) from 2006 to 2010 for their risk score, cytogenetic response at 18 months and event free survival (EFS) at the end of 4 years. Events include loss of hematological response, loss of cytological response, progression to accelerated/blast phase (AP/BC). All received free Imatinib under Gleevac international patient assistance program. RESULTS: Data of 106 children was analyzed with median age of 13.5 (ranged 5-18 years) and male preponderance (M:F = 1.14:1). The distribution of children was 63%, 32% and 5% in Sokal low, intermediate and high risk respectively, 50%, 43% and 5% in Hasford/Euro low, intermediate and high risk respectively, 71% and 29% in EUTOS low and high risk respectively. The overall cumulative complete hematological response at the end of 3 month was 94%, and complete cytogenetic response at 12 months was 75%. The CCyR at 18 month was seen in 90%,74% and 83% among Sokal low, intermediate and high risk groups respectively, 83%, 86% and 83% among Hasford/Euro low, intermediate and high risk groups respectively, 84% and 86% EUTOS low and high risk groups respectively. The EFS at the end of 48 months was seen in 87%,79% and 83% among Sokal low, intermediate and high risk groups respectively, 83%, 86% and 83% among Hasford/Euro low, intermediate and high risk groups respectively, 86% and 80% EUTOS low and high risk groups respectively. CONCLUSION: None of the scoring systems predicted the response and outcome effectively in children with CML CP on front line Imatinib.

Impact of treatment protocol on outcome of localized Ewing's sarcoma.

BACKGROUND: The outcome of localized Ewing's sarcoma has improved with multi-disciplinary approach. Survivals of Ewing's sarcoma from the Asian countries differed between centers. METHODS: We retrospectively analyzed the records of newly diagnosed localized Ewing's sarcoma patients from 2002 to 2012. The patients were analyzed in three groups; Group 1(2002-2004) who received non-ifosfomide based regimens, Group 2(2005-2008) who received VDC/IE for 12 cycles, and Group 3(2009-2012), who received VDC/IE for 17 cycles. The groups were compared for their baseline characteristics, treatment protocol and outcome. RESULTS: Seventy three patients were included in the study. The median age of presentation was 15 years, with slight male predominance. Axial primary was seen in 62%. The median RFS of the three groups was 26.4, 31.4 and 36.8 months respectively (P = 0.0018). The median OS was 27.9, 35 and 43 months respectively (P = 0.0007). At a median follow-up of 35 months, the 3 year RFS and OS for the three treatment groups were 17%, 31%, 60% and 35%, 45% and 70% respectively. Larger tumor size, axial primary, high LDH were associated with poorer survival. Radiotherapy was associated with inferior local control and survival. CONCLUSIONS: We found that the survival of our ESFT patients improved over time with intensified multiagent chemotherapy and with lesser time to local therapy. But the results were still inferior to those reported in literature. We had majority of patients presenting in axial site and radiotherapy as the predominant mode of local control. The outcome may further improve with surgery as local control procedure.

Trends in management of acute lymphoblastic leukemia: Influence of insurance based healthcare and treatment compliance on the outcome of adolescents and adults with acute lymphoblastic leukemia.

AIM: In this study, we attempted to analyze the impact of insurance based health care system and treatment compliance on the outcome of adolescent and adults with acute lymphoblastic leukemia (ALL). MATERIALS AND METHODS: Patients who underwent treatment for ALL during the period 2003-2011 were enrolled into this retrospective study. Patients on supportive or palliative care only and patients with age <10 years were excluded. The hospital records and tumor registry records were studied. Patients were stratified into two groups, Group A (prior to the introduction of state health insurance [SHI], 2003-2007) and Group B (after the introduction of SHI, 2008-2011). Overall survival (OS) was calculated using Kaplan-Meier method. RESULTS: A total of 420 patients with suspected or confirmed ALL visited our center during the study period and 179 patients (87 in Group A and 92 in Group B) were considered for inclusion. The median age in years (range) was 18 (10-57) and 18 (10-58) respectively in Groups A and B with males more than females. Median OS (95% CI) was 9 (6.7-11.2) and 12 (7.3-16.7) months in the Groups A and B respectively (P = 0.265). Poor treatment compliance in both groups was high (36% in Group A and 41% in Group B, [P = 0.107]) with lower default rates in Group B (P = 0.019). Patients with good compliance in the total study population and the individual study groups had significantly better OS. CONCLUSIONS: Insurance based health care has improved outcomes in the present study but not compliance to treatment. Significantly better OS was observed in patients with good compliance.

Impact of Imatinib Adherence on the Cytogenetic Response in Pediatric Chronic Myeloid Leukemia - Chronic Phase.

The authors aimed to study the impact of adherence to imatinib during initial 6 mo on the cytogenetic response in pediatric chronic myeloid leukemia - chronic phase (CML CP). The hospital records of pediatric CML patients (age ≤18 y) from 2009 through 2012, were analyzed retrospectively for the drug adherence and cytogenetic response (CyR) at 6 mo. Forty eight children were analyzed, with the median age of 13 y (range 5-18) and slight male preponderance (M:F- 1.18:1). Sokal scores were low, intermediate and high in 14 (29.3 %), 26 (54.1 %), 8 (16.6 %) children respectively. Only a little more than half of the children were adherent (58 %). At the end of 6 mo, complete cytogenetic response (CCyR) was achieved by 78.5 % of adherent children as compared to 5 % of non-adherent children. Majority (80 %) of the non-adherent children had only a partial cytogenetic response (PCyR). Therefore, it is concluded that most of the adherent children had optimal cytogenetic response at the end of 6 mo and majority of those in the non-adherent group did not attain it.

Response to imatinib mesylate in childhood chronic myeloid leukemia in chronic phase.

INTRODUCTION: Childhood chronic myeloid leukemia (CML) accounts for less than 3% of all childhood leukemias, hence, data on imatinib (IM) in adult CML patients has been largely extrapolated to children. We have analyzed our data to add to the existing literature. AIMS: Primary objective is to assess the progression-free survival (PFS). Secondary objective are cytogenetic response, overall survival (OS), and toxicities. SETTINGS AND DESIGN: This is a retrospective analysis from the case records from a single institution. MATERIALS AND METHODS: Institutional ethics committee approval was obtained. All the children diagnosed CML in chronic phase (CML-CP) aged less than 18 years registered between 2000 and 2009 were enrolled. All the patients were started on IM at 260 mg/m(2). STATISTICAL ANALYSIS: Kaplan-Meier curves were used to calculate the PFS and OS. RESULTS: There were 64 children with median age of 13 years (range, 1-18) with male predominance (male:female (M: F) - 1.85:1). Sixty-one patients (95.4%) achieved complete hematological response (CHR) at median of 8 weeks. Thirty-seven (57.8%) patients had evaluation of cytogenetic response and were subjects for outcome analysis. The median time to best cytogenetic response evaluation was 13 months (range, 4-52). Twenty-nine patients (78.3%) achieved complete cytogenetic response (CCyR). At a median follow-up of 36 months (range 5-75), 21 (56.8%) remained progression free and 35 (94.5%) are alive. Adverse events were tolerable. CONCLUSIONS: PFS at a median follow-up of 36 months is 56.8% and OS 94.5%.