SETTING: Tertiary level teaching institution in Malaysia.
PARTICIPANTS: The validation process involved 211 adult patients (English language n=101, Malay language n=110) with chronic liver disease. Characteristics of the study subjects were as follows: mean (SD) age was 56 (12.8) years, 58.3% were male and 41.7% female. The inclusion criteria were patients 18 years or older with chronic hepatitis and/or liver cirrhosis of any aetiology. The exclusion criteria were as follows: presence of hepatic encephalopathy, ongoing treatment with interferon and presence of other chronic conditions that have an impact on health-related quality of life (HRQOL).
METHODS: A cross-sectional study was conducted. Cultural adaptation of the English version of the CLDQ was performed, and a Malay version was developed following standard forward-backward translation by independent native speakers. Psychometric properties of both versions were determined by assessing their internal consistency, test-retest reliability and discriminant and convergent validity.
RESULTS: Cronbach's alpha for internal consistency across the various domains of the CLDQ was 0.95 for the English version and 0.92 for the Malay version. Test-retest analysis showed excellent reliability with an intraclass correlation coefficient of 0.89 for the English version and 0.93 for the Malay version. The average scores of both the English and Malay versions of the CLDQ demonstrated adequate discriminant validity by differentiating between non-cirrhosis (English 6.3, Malay 6.1), compensated cirrhosis (English 5.6, Malay 6.0) and decompensated cirrhosis (English 5.1, Malay 4.9) (p<0.001). Convergent validity showed that correlation was fair between the English (ρ=0.59) and Malay (p=0.47) CLDQ versions with the EQ-5D, a generic HRQOL instrument.
CONCLUSION: The English and Malay versions of the CLDQ are reliable and valid disease-specific instruments for assessing HRQOL in Malaysian patients with chronic liver disease.
PURPOSE: To evaluate the effect of IE on the marrow fat content and fat unsaturation levels in the proximal femur using 1 H-MRS.
STUDY TYPE: Prospective.
SUBJECTS: Twenty-three subjects were included in this study, seven control and 16 β-thalassemia subjects.
FIELD STRENGTH/SEQUENCE: 3.0T; stimulated echo acquisition Mode (STEAM); magnetic resonance spectroscopy (MRS) sequence.
ASSESSMENT: Multiecho MRS scans were performed in four regions of the proximal left femur of each subject, that is, diaphysis, femoral neck, femoral head, and greater trochanter. The examined regions were grouped into red (diaphysis and femoral neck) and yellow marrow regions (femoral head and greater trochanter).
STATISTICAL TESTS: The Jonckheere-Terpstra test was used to evaluate the impact of increasing disease severity on bone marrow fat fraction (BMFF), marrow conversion index, and fat unsaturation index (UI). Pairwise comparison analysis was performed when a significant trend (P
PURPOSE: To investigate the relationship between marrow fat and cortical bone thickness in β-thalassemia and to identify key determinants influencing these variables.
STUDY TYPE: Prospective.
SUBJECTS: Thirty-five subjects in four subject groups of increasing disease severity: 6 healthy control (25.0 ± 5.3 years, 2 male), 4 β-thalassemia minor, 13 intermedia, and 12 major (29.1 ± 6.4 years, 15 male).
FIELD STRENGTH/SEQUENCE: 3.0 T, 3D fast low angle shot sequence and T1-weighted turbo spin echo.
ASSESSMENT: Analyses on proton density fat fraction (PDFF) and R2* values in femur subregions (femoral head, greater trochanter, intertrochanteric, diaphysis, distal) and cortical thickness (CBI) of the subjects' left femur. Clinical data such as age, sex, body mass index (BMI), and disease severity were also included.
STATISTICAL TESTS: One-way analysis of variance (ANOVA), mixed ANOVA, Pearson correlation and multiple regression. P-values <0.05 were considered significant.
RESULTS: Bone marrow PDFF significantly varied between the femur subregions, F(2.89,89.63) = 44.185 and disease severity, F(1,3) = 12.357. A significant interaction between subject groups and femur subregions on bone marrow PDFF was observed, F(8.67,89.63) = 3.723. Notably, a moderate positive correlation was observed between PDFF and CBI (r = 0.33-0.45). Multiple regression models for both PDFF (R2 = 0.476, F(13,151) = 10.547) and CBI (R2 = 0.477, F(13,151) = 10.580) were significant. Significant predictors for PDFF were disease severity (βTMi = 0.36, βTMa = 0.17), CBI (β = 0.24), R2* (β = -0.32), and height (β = -0.29) while for CBI, the significant determinants were sex (β = -0.27), BMI (β = 0.55), disease severity (βTMi = 2.15), and PDFF (β = 0.25).
DATA CONCLUSION: This study revealed a positive correlation between bone marrow fat fraction and cortical bone thickness in β-thalassemia with varying disease severity, potentially indicating a complex interplay between bone health and marrow composition.
EVIDENCE LEVEL: 2 TECHNICAL EFFICACY: Stage 3.
METHODS: This is a retrospective study on NDMM patients diagnosed between 1 January 2008 and 31 December 2022 in a single academic center. Patients' demographic and treatment details were included for analysis of progression free survival (PFS) and overall survival (OS).
RESULTS: One hundred and thirty-six NDMM patients with a median age of 64.0 years (ranged from 38 to 87 years old) were included. Bortezomib-containing regimens were the most commonly used induction agent, followed by thalidomide. Almost half of the patients (47.1%) achieved very good partial response (VGPR) or complete remission (CR), while 31.6% achieved partial response (PR). Bortezomib containing regimen was associated with significantly deeper and more rapid response, (p=0.001 and p=0.017, respectively) when compared to other agents. Only 22.8% of these patients proceeded to upfront autologous haematopoietic stem cell transplantation. The median OS and PFS were 60.0 months and 25.0 months, respectively. Best initial response and upfront autologous stem cell transplantation (ASCT) were significantly associated with better PFS.
CONCLUSION: Achieving at least a VGPR significantly associated with better outcome in NDMM patients. In a resource constrain country, we recommend incorporating bortezomib in the induction therapy followed with an upfront ASCT.