METHODS: A total of 114 autologous stem-cell transplantation patients aged ≥ 18 years old at a national hematology center in Malaysia from April 2019 to December 2020 completed OMDQ-Mal concurrently with physician scores. Internal consistency and reproducibility were determined by Cronbach alpha and intraclass correlation coefficient, respectively. Correlations with physician scores were determined by Spearman correlation. Discriminative validity and construct validity were determined by Mann-Whitney U and CFA, respectively.
RESULTS: OMDQ-Mal demonstrated high internal consistency (α = 0.874). Test-retest reliability between paired days were moderate to excellent (95% CI = 0.676-0.953). Items in OMDQ-Mal had moderate to strong correlations with physician scores (ρ = 0.503-0.721). Discriminative validity indicated that the scores of scales were significantly different between participants with severe and mild conditions. Construct validity results of loading factors 0.708-0.952; composite reliability 0.879-0.974; average variant extracted 0.710-0.841; and heterotrait-monotrait ratio 0.528 established the convergent and divergent validity.
CONCLUSIONS: In conclusion, the OMDQ-Mal, which captured important quality of life responses, demonstrated adequate validity and reliability. This was supported by a two-component model CFA. The strong correlation of OMDQ-Mal with both physician scores indicated its potential as a comprehensive patient-reported outcome measure of mucositis of the entire alimentary tract.
METHODS: The performance of a custom, in-house designed 22-gene NGS panel was technically validated using reference standards across two independent replicate runs. The panel was subsequently used to screen a total of 10 clinical MPN samples (ET n = 3, PV n = 3, PMF n = 4). The resulting NGS data was then analysed via a bioinformatics pipeline.
RESULTS: The custom NGS panel had a detection limit of 1% variant allele frequency (VAF). A total of 20 unique variants with VAFs above 5% (4 of which were putatively novel variants with potential biological significance) and one pathogenic variant with a VAF of between 1 and 5% were identified across all of the clinical MPN samples. All single nucleotide variants with VAFs ≥ 15% were confirmed via Sanger sequencing.
CONCLUSIONS: The high fidelity of the NGS analysis and the identification of known and novel variants in this study cohort support its potential clinical utility in the management of MPNs. However, further optimisation is needed to avoid false negatives in regions with low sequencing coverage, especially for the detection of driver mutations in MPL.
Methods: This was a retrospective epidemiological study involving all cases of TMA from 2012-2016.
Results: We evaluated 243 patients with a median age of 34.2 years; 57.6% were female. Majority of the patients were Malay (62.5%), followed by Chinese (23.5%) and Indian (8.6%). The proportion of patients with thrombotic thrombocytopenic purpura (TTP) was 20.9%, 72.2% of which were acquired while 27.8% were congenital. Patients with ADAMTS-13 activity ≥5% had a four-fold higher odds of mortality compared to those with ADAMTS-13 activity <5% (odds ratio: 4.133, P=0.0425). The mortality rate was 22.6% (N=55). Most cases had secondary etiologies (42.5%), followed by acquired TTP (16.6%), atypical hemolytic uremic syndrome (HUS) or HUS (12.8%) and congenital TTP (6.4%). Patients with secondary TMA had inferior overall survival (P=0.0387). The secondary causes comprised systemic lupus erythematosus (30%), infection (29%), pregnancy (10%), transplant (8%), malignancy (6%), and drugs (3%). Transplant-associated TMA had the worst OS (P=0.0016) among the secondary causes. Plasma exchange, methylprednisolone and intravenous immunoglobulin were recorded as first-line treatments in 162 patients, while rituximab, bortezomib, vincristine, azathioprine, cyclophosphamide, cyclosporine, and tacrolimus were described in 78 patients as second-line treatment.
Conclusion: This study showed that TMA without ADAMTS-13 deficiency yielded inferior outcomes compared to TMA with severeADAMTS-13 deficiency, although this difference was not statistically significant.
Materials and methods: This retrospective national registry of MPN was conducted from year 2009 to 2015 in Malaysia.
Results: A total of 1010 patients were registered over a period of 5 years. The mean age was 54 years with male predominance. The ethnic distribution revealed that Chinese had a relatively high weighted incidence proportion (43.2%), followed by Indian (23.8%), Malay (15.8%) and other ethnic groups (17.2%). The types of MPN reported were 40.4% of ET (n = 408), 38.1% of PV (n = 385), 9.2% of PMF (n = 93), 3.1% of hypereosinophilic syndrome (HES) (n = 31) and 7.9% of unclassifiable MPN (MPN-U) (n = 80). Splenomegaly was only palpable clinically in 32.2% of patients. The positive JAK2 V617F mutation was present in 644 patients with 46.6% in PV, 36.0% in ET, 9.0% in PMF, and 7.4% in MPN-U, and had significantly lower haemoglobin (p
METHODS: This study included participants from the intervention arm of a randomised controlled trial which was conducted to evaluate the effects of pharmacist-led interventions on CML patients treated with TKIs. Participants were recruited and followed up in the haematology clinics of two hospitals in Malaysia from March 2017 to January 2019. A pharmacist identified DRPs and helped to resolve them. Patients were followed-up for six months, and their DRPs were assessed based on the Pharmaceutical Care Network Europe Classification for DRP v7.0. The identified DRPs, the pharmacist's interventions, and the acceptance and outcomes of the interventions were recorded. A Poisson multivariable regression model was used to analyse factors associated with the number of identified DRPs per participant.
RESULTS: A total of 198 DRPs were identified from 65 CML patients. The median number of DRPs per participants was 3 (interquartile range: 2, 4). Most participants (97%) had at least one DRP, which included adverse drug events (45.5%), treatment ineffectiveness (31.5%) and patients' treatment concerns or dissatisfaction (23%). The 228 causes of DRPs identified comprised the following: lack of disease or treatment information, or outcome monitoring (47.8%), inappropriate drug use processes (23.2%), inappropriate patient behaviour (19.9%), suboptimal drug selection (6.1%), suboptimal dose selection (2.6%) and logistic issues in dispensing (0.4%). The number of concomitant medications was significantly associated with the number of DRPs (adjusted Odds Ratio: 1.100; 95% CI: 1.005, 1.205; p = 0.040). Overall, 233 interventions were made. These included providing patient education on disease states or TKI-related side effects (75.1%) and recommending appropriate instructions for taking medications (7.7%). Of the 233 interventions, 94.4% were accepted and 83.7% were implemented by the prescriber or patient. A total of 154 DRPs (77.3%) were resolved.
CONCLUSIONS: The pharmacist-led interventions among CML patients managed to identify various DRPs, were well accepted by both TKI prescribers and patients, and had a high success rate of resolving the DRPs.
METHODS: Through the Association of Southeast Asian Nations Costs in Oncology study, 1,294 newly diagnosed patients with cancer (Ministry of Health [MOH] hospitals [n = 577], a public university hospital [n = 642], private hospitals [n = 75]) were observed in Malaysia. Cost diaries and questionnaires were used to measure incidence of financial toxicity, encompassing financial catastrophe (FC; out-of-pocket costs ≥ 30% of annual household income), medical impoverishment (decrease in household income from above the national poverty line to below that line after subtraction of cancer-related costs), and economic hardship (inability to make necessary household payments). Predictors of financial toxicity were determined using multivariable analyses.
RESULTS: One fifth of patients had private health insurance. Incidence of FC at 1 year was 51% (MOH hospitals, 33%; public university hospital, 65%; private hospitals, 72%). Thirty-three percent of households were impoverished at 1 year. Economic hardship was reported by 47% of families. Risk of FC attributed to conventional medical care alone was 18% (MOH hospitals, 5%; public university hospital, 24%; private hospitals, 67%). Inclusion of expenditures on nonmedical goods and services inflated the risk of financial toxicity in public hospitals. Low-income status, type of hospital, and lack of health insurance were strong predictors of FC.
CONCLUSION: Patients with cancer may not be fully protected against financial hardships, even in settings with universal health coverage. Nonmedical costs also contribute as important drivers of financial toxicity in these settings.