Background Sinusoidal obstruction syndrome (SOS) is an important non-haematological toxicity in patients who have received chemotherapy for haematopoietic stem cell transplantation (HSCT). In March 2013, European Medicines Agency refused marketing authorisation for the orphan drug defibrotide to prevent and treat SOS. In July 2013, after re-examination, defibrotide had a new positive opinion for marketing authorisation but only for treatment. Purpose To investigate the effectiveness, safety and cost of defibrotide for SOS. Materials and methods Retrospective observational study (Period: January 2009–September 2013). We analysed patients with SOS treated with defibrotide. Response criteria were defined as total serum bilirubin <2 mg/dl and resolution of multiple organ failure (MOF), including renal (recovery of normal creatinine value and lack of dialysis dependence), pulmonary (no oxygen dependence) and central nervous system (absence of encephalopathy) function. Evolution of the Baltimore criteria, including hepatomegaly, ascites and weight gain was measured. Secondary endpoint was survival by 100 days post-HSCT. Adverse events related to defibrotide administration were also retrieved. Results Eleven patients (five adults (mean age: 55.7(SD = 4.3) years) and four children (3.1(SD = 1.3)) received defibrotide at a dose of 6.25 mg/kg every 6 h (two patients received 10 mg/kg/6 h). Median duration of treatment was 9 days (range: 5–25). Overall complete response was achieved in seven patients (63.6%), all four children and only three adults. 45.5% of patients had completely recovered from hepatomegaly at the end of defibrotide administration. Six of the eight patients with ascites and one patient with encephalopathy recovered their normal status. All patients reduced their weight gain after defibrotide treatment. 100 day post-HSCT survival was 72.7%. Three patients presented haemorrhagic episodes during defibrotide treatment (two gastrointestinal episodes and one nasal bleeding). Median cost per patient was 19,180 € (range 5,480–41,100 €). Conclusions According to our limited results, defibrotide is an effective option for SOS treatment in children, although cost per patient is high. Cost-effectiveness studies comparing treatment for SOS with and without defibrotide are needed. No conflict of interest.
optimise trt management, safety and outcomes. This innovative cancer care model could improve drug awareness of drug consumption and patient education to promptly recognise and manage AEs.
BackgroundProgressive multifocal leukoencephalopathy(PML) is caused by JC virus (JCV) and has severe consequences for the central nervous system. PML is an opportunistic infection, affecting HIV positive patients. There is no curative treatment, and the current approach is focused on immune reconstitution and antiviral therapy.1 PurposeThis is a case report of two patients with PML associated with HIV infection treated with aldesleukin/interleukin-2 (IL-2).Material and methodsWe report two cases, both men, aged 48 years (patient A) and 57 years (patient B), HIV diagnosed in 1992 and 1993, who developed LMP opportunistic JCV infection. Antiretrovirals were the only therapy previously employed. Patient A developed progressive neurological impairment, slow psychic reactions and right upper limb strength loss. Symptoms in patient B were progressive cognitive impairment, memory loss, lack of coordination, dysarthria, strength loss and right facial paralysis. Both patients showed hypodensity of white matter in the semi-oval centre on cranial CT.ResultsIL-2 was administered intravenously to both patients at 0.5 MU/m²/day for 4 weeks. Patient-A: IL-2 treatment (from 8 February 2010 to 7 March 2010) was well tolerated, although a self-limited fever and eosinophilia were reported without clinical correlations. The patient showed improvements in image tests (disappearance of signal hyperintensity and cytotoxic oedema on CT and MRI) and clinical outcomes (improvement of neurological symptoms), although progressive spasticity and dysarthria were maintained. JCV load was undetectable in February 2011. Patient A was treated for 1 year with mefloquine 250 mg/24 hours with good tolerance and stabilisation of PML, but without resolution. The patient died in December 2012 following intraparenchymal cerebral haemorrhage. Patient-B: therapy with IL-2 and mirtazapine (from 8 September 2016 to 5 October 2016) was well tolerated, but the infusion was discontinued once by a fever episode. Cranial CT showed no significant changes. The patient´s neurological impairment, hemiparesis and dysarthria persisted, but a slight improvement was observed. JCV load declined from >100 million to 12 million copies/mL, HIV from 645 to 147 copies/mL and CD4 T lymphocytes increased.ConclusionIL-2 could be an effective and well tolerated therapy in LMP, without severe adverse events. Patient A showed improvements in neurological and image tests, and undetectable viral load, whereas the response in patient B was only partial, with a decline in JCV and HIV, but maintaining neurological deterioration.References and/or acknowledgements1. Pavlovic D, et al. Ther Adv Neurol Disord2015;8:255–73.No conflict of interest
Background Minimization of chemotherapy costs has become a rational goal in today’s economic environment. Purpose To assess the cost savings achieved by optimising vial residues during chemotherapy preparation. Materials and MethodsA longitudinal prospective study was conducted in the Intravenous Chemotherapy Unit of the Pharmacy Service between 15 January and 31 March of 2012. We selected the six drugs with more potential cost saving (bevacizumab, bortezomib, liposomal doxorubicin, panitumumab, rituximab and trastuzumab). Data were collected with the Oncofarm software: number of patients, number of preparations, theoretical and actual number of vials used. For economic estimates the retail price (RRP) was used. Results During the study period, 365 preparations were administered to 190 patients; these required the potential use of 716 vials, but actually 545 vials were used, saving 219,538€ (33% of the cost without recycling excess vials). Data analysis showed that 81% of the total savings were achieved with only 2 drugs: bevacizumab (50%, 80 vials, €110,556) and rituximab (31%, 50 vials, €67,752). Their high frequency of use (66% of preparations and 68% of patients), high cost and greater variability of prescribed doses, justifies these results. Theoretical average costs of the preparations analysed without managing the residues of partially-used containers were 1,790 (SD:476) €/preparation and 3,568 (SD:642) €/patient. After savings were made the averages were 499 (SD:253) €/preparation and 965 (SD:389) €/patient. Rituximab (€836/preparation, €1,063/patient), bevacizumab (€700/preparation, €1,602/patient) and panitumumab (€625/preparation, €1,111/patient) were the drugs with greater savings. We estimated the difference between potential savings if the adjustment had been perfect and the actual saving obtained (€21,135), possibly caused by the preparation process or expiry of some reconstituted vials Conclusions Residues management is a common practise to improve the efficiency of the preparation process. Optimizing this process of updating medicines’ stabilities, recording the opening date on the vial, checking expiries and storage conditions, achieved significant savings in the cost of treatments. No conflict of interest.
BackgroundPaclitaxel is commonly associated with infusion reactions (IR) with no clear influence of different paclitaxel formulations.PurposeTo analyse the number and severity of IR related to administration of different generic formulations of paclitaxel registered by means of an adverse drug reactions reporting programme (ADRRP).Material and methodsObservational, retrospective study from January 2010 to March 2015. Identification of IR was carried out by an active collaboration of day hospital nursing staff based on voluntary reporting of adverse drug reactions (ADRs) documented centrally at the pharmacy department (chemotherapy unit) using the application Farmis-Oncofarm within the framework of ADRRP. Variables collected: sex, age, generic brand name, cycle, IR severity (CTCAE v4.03), ADRs medication management and re-administration tolerance.5 different generic formulations (A-E) were used during the study period, with no significant differences in type and concentration of the excipients. All patients received premedication with corticosteroids, antihistamines and H2 antagonists, as recommended by the summary of product characteristics.Relative frequencies and severity were calculated, and χ2 and Fisher exact tests were used for statistical comparison (SPSS v.19).ResultsDuring the study period, 648 patients (401 women (61.9%)), median age 59.5 years (range 23–86) received a total of 4845 paclitaxel intravenous infusions: 61.3% (paclitaxel A), 28.4% (B), 6.7% (C), 3.3% (D) and 0.4% (E).61 IR were recorded. Paclitaxel A: 36 (1.21%), B: 14 (1.02%), C: 6 (1.86%), D: 1 (0.62%) and E: 4 (23.53%). No statistically significant differences (SSD) were observed in IR number or severity except with E paclitaxel (p < 0.001). 41% of IR occurred during the first administration. 46/61 grade 2; 14/61 grade 3; and 1 grade 4 (ICU admission after the second cycle). All IR were managed by temporarily stopping the current infusion and symptomatic treatment with corticosteroid+antihistamine±paracetamol as per protocol. 18/61 did not tolerate re-administration.ConclusionSSD were only observed with E paclitaxel without finding out the cause. Sample imbalance among formulations was due to the regional health department centralised purchasing system through public tenders and several shortage supplies over the study period. The ADRRP based on the active voluntary collaboration of nurses was effective in detecting drug related problems and implementing interventions accordingly (notification to national surveillance programme, laboratory involved and changing the available presentation at the hospital) to enhance drug safety.No conflict of interest.
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