Vivian Soetikno
Department of Pharmacology and Therapeutics, Faculty of Medicine, Universitas Indonesia

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Comparative efficacy, safety, and cost of iron chelation monotherapy vs. combination therapy in pediatric beta-thalassemia major: a single-center retrospective study Dewi Sharon Simorangkir; Nafrialdi Nafrialdi; Pustika Amalia Wahidiyat; Vivian Soetikno
Paediatrica Indonesiana Vol 62 No 2 (2022): March 2022
Publisher : Indonesian Pediatric Society

Show Abstract | Download Original | Original Source | Check in Google Scholar | DOI: 10.14238/pi62.2.2022.91-7

Abstract

Background Iron chelation therapy is used to maintain iron balance in β-thalassemia major patients who undergo repeated blood transfusions. Objective To compare the efficacy, safety, and cost of iron chelation combination regimens [deferiprone (DFP) + deferoxamine (DFO) or DFP + deferasirox (DFX])] vs. high-dose DFP monotherapy (≥ 90 mg/kg/day) in pediatric β-thalassemia major patients. Methods This cross-sectional, retrospective study was done at Cipto Mangunkusumo Hospital, Jakarta, Indonesia. Retrospective data was obtained from electronic medical records of pediatric b-thalassemia major patients with serum ferritin of ≥ 2,500 ng/mL and/or transferrin saturation of ≥ 60%, who received either combination or monotherapy iron chelation agents. Outcome effectiveness was determined by the reduction of serum ferritin level of at least 80%. Safety was analyzed descriptively. A pharmacoeconomic analysis was performed based on clinical outcomes consisting of effectiveness and direct medical costs. Results At the end of the study, serum ferritin was reduced in 34.7% of the combination therapy group and 27.5% of the monotherapy group, however there was no significant difference between the two treatments (P=0.391). Nine (19.5%) patients on combination therapy and 17 (21.2%) patients on monotherapy had adverse drug reaction (ADR), with the most frequently reported ADR was elevated transaminase enzyme levels. Cost minimization analysis revealed that monotherapy for 6 months was IDR 13,556,592.64 less expensive than combination therapy (IDR 44,498,732.07); whereas monotherapy for 12 months was IDR 20,162,836.10 less expensive than combination therapy (IDR 78,877,661.12). Conclusion Combination regimens are as effective as monotherapy regimens in reducing serum ferritin in pediatric β-thalassemia major patients. There is no differences of ADR between combination or monotherapy. The average cost per patient is less expensive with monotherapy compared to combination therapy.
Observational Study of Paclitaxel-Carboplatin versus Pemetrexed-Carboplatin for Advanced Pulmonary Adenocarcinoma at Single-Centre in Indonesia Rianyta Rianyta; Melva Louisa; Elisna Syahruddin; Vivian Soetikno
eJournal Kedokteran Indonesia Vol 9, No. 3 - Desember 2021
Publisher : Faculty of Medicine Universitas Indonesia

Show Abstract | Download Original | Original Source | Check in Google Scholar | Full PDF (224.216 KB) | DOI: 10.23886/ejki.9.16.178-86

Abstract

Platinum-based chemotherapy regimens with two types of drugs, such as paclitaxel-carboplatin and pemetrexedcarboplatin, are first-line therapy for pulmonary adenocarcinoma patients with negative epidermal growth factor receptor (EGFR) mutation. This study aimed to determine the efficacy, toxicity, and cost profile of paclitaxel-carboplatin compared to pemetrexed-carboplatin. This is a cross-sectional study. Pulmonary adenocarcinoma negative EGFR mutation naïve patients treated with paclitaxel-carboplatin or pemetrexed-carboplatin were included. Effectiveness was assessed based on the overall response rate (ORR) according to the response evaluation criteria in solid tumours (RECIST). A pharmacoeconomic analysis is performed based on clinical outcomes consisting of effectiveness and direct medical costs. Medical records from 21 patients with paclitaxel-carboplatin and 21 patients with pemetrexedcarboplatin were successfully evaluated. The effectiveness of the two chemotherapy regimens was not significantly different (OR, 1.25; 95% confidence interval, 0.34 to 4.64; p = 0.739). Frequent haematological toxicities experienced in the two groups were grade 1-2 anaemia, neutropenia, leukopenia. Grade 3 anaemia, leukopenia, and neutropenia were more common in the paclitaxel-carboplatin group. The two groups’ nonhematological toxicities were nausea vomitus and hair loss, with peripheral neuropathy more experienced by the paclitaxel-carboplatin group. Costminimization analysis reveals that the average cost per patient with pulmonary adenocarcinoma negative EGFR mutation with paclitaxel-carboplatin regimen was cheaper IDR 10,986,257.55 or 50.25%, compared to pemetrexedcarboplatin. In conclusion, there was no significant difference in the effectiveness of the two regimens. The most common adverse effects in both regimens were haematological toxicities. The average cost per patient with a paclitaxel-carboplatin regimen was cheaper compared to pemetrexed-carboplatin.