Splenectomy in Immune Thrombocytopenia: A Retrospective Analysis of 25-Year Follow-up Data from a Tertiary Health Clinic.

Immune thrombocytopenia (ITP) is a rare autoimmune disorder presenting with isolated thrombocytopenia. Splenectomy is still one of the treatment alternatives for these patients. Here we aim to analyze long term follow-up data of splenectomy in immune thrombocytopenia. This retrospectively designed study was conducted in a tertiary health clinic. Patients with ITP who were splenectomized between 1990 and 2015 were included. Response to treatment was interpreted as 'complete response', 'response' or 'no response'. The incidence of response loss was evaluated. Perioperative and long term complications and overall survival rates were determined. Out of 51 patients, who underwent splenectomy after 12 months of diagnosis, 47 achieved a response (92.2%). Of 47 patients who had a platelet count at least 30.000/µL, 41 (87.2%) had CR. Incidence of loss of response was 10.5% (95% confidence interval (CI): 4%-26.1%) at 30 months. Two patients died, and overall survival rate was 97.4% (95% CI: 82.8%-99.6%) at 30 months of follow up. Considering the complications: two patients had venous thromboembolism, 11 had minor bleeding episodes and 15 suffered from perioperative infections. Our study suggests that splenectomy promises a high level of response with acceptable complication rates. Although less preferred recently, splenectomy should still be taken into consideration when remission is not achieved especially after 12 months of disease.

Effects of Deeper Molecular Responses on Outcomes in Chronic Myeloid Leukemia Patients in Chronic Phase Treated With Imatinib Mesylate.

BACKGROUND: The prognostic significance of complete cytogenetic response (CCyR) is well defined in patients with chronic phase chronic myeloid leukemia treated with imatinib as first-line therapy. However, the effect on outcomes of obtaining molecular response itself and the depth of it is not clear. In this study we aimed to determine the frequency of complete molecular response (CMR) during long-term follow-up and the clinical significance of CMR on patient outcomes and survival. PATIENTS AND METHODS: We retrospectively evaluated the files of 178 chronic phase chronic myeloid leukemia patients using imatinib therapy. Forty-seven patients with missing data were excluded from the study and the assessment was done in 131 patients. CMR was defined as undetectable BCR-ABL transcripts using real-time quantitative polymerase chain reaction with a sensitivity level of ≥ 104 in 2 consecutive analyses at least 3 months apart. Cytogenetic and molecular monitoring during treatment was performed according to the European LeukemiaNet recommendations criteria. Our primary objective was to analyze the association of deeper molecular response with differences in progression-free survival (PFS). RESULTS: Eighty-eight patients (67%) achieved CMR at any time in a median of 65 months of follow-up. The rate of CMR was higher in patients who achieved CCyR at 12 months and major molecular response (MMR) at 18 months. Fewer events occurred in the CMR group than the MMR group (26.1% vs. 50.0%). Overall survival was not different in both groups. CMR was associated with longer PFS with borderline significance. CONCLUSION: Prolonged imatinib therapy helps to achieve a deeper molecular response in the long-term. Achieving deeper molecular response at any time positively affects maintaining the cytogenetic and molecular responses, and decreases the transformation to accelerated and/or blastic phase. The slight prolongation in PFS did not reach statistical significance.

Efficacy and safety of eltrombopag in treatment-refractory primary immune thrombocytopenia: a retrospective study.

Eltrombopag was used in patients with chronic primary immune thrombocytopenia (ITP) who did not tolerate or were refractory to two or more previous treatments. The primary aims of the study were to determine the efficacy and safety of long-term eltrombopag treatment. Data were extracted from medical chart records retrospectively. Platelet count of at least 50 000/µl at any time point during the treatment was defined as the 'response'. Median duration of eltrombopag treatment was 29 weeks (11-74). The number of patients who had a platelet count of at least 50 000/µl at any time point was 26 (83.9%). The response was achieved by the second week in most of the patients. Concomitant ITP medications were withdrawn in nine out of the 11 patients. Eltrombopag was discontinued in one patient due to sustained response despite discontinuation of the treatment. Age, sex, concomitant ITP treatments, and previous ITP treatment failures had no impact on the treatment response. The treatment was discontinued due to thrombosis in only four patients. Four patients experienced a minor bleeding event. Hepatotoxicity and all other adverse events were mild and manageable. Eltrombopag is effective, safe, and well tolerated in the long-term treatment of chronic ITP patients.