| 1. |
- Astermark, Jan, et al.
(author)
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Efficacy of rfixfc versus rix-fp for the treatment of patients with hemophilia b : Matching-adjusted indirect comparison of b-long and prolong-9fp trials
- 2021
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In: Journal of Blood Medicine. - 1179-2736. ; 12, s. 613-621
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Journal article (peer-reviewed)abstract
- Purpose: In patients with hemophilia B, treatment with extended half-life (EHL) recombi-nant factor IX allows for longer dosing intervals while providing equal or superior bleeding protection compared with standard half-life products. This enables flexible, individualized treatment schedules, which reduce the burden of prophylaxis and improve patient outcomes. This analysis compared the efficacy of recombinant factor IX Fc fusion protein (rFIXFc) and recombinant factor IX albumin fusion protein (rIX-FP), two EHL therapies approved for prophylaxis and treatment of bleeding in hemophilia B. Patients and Methods: Matching-adjusted indirect treatment comparison (MAIC) was used to adjust the between-treatment differences in baseline characteristics. Individual patient data for rFIXFc (B-LONG) were matched to aggregated data for rIX-FP (PROLONG-9FP) followed by statistical comparison for estimated annualized bleeding rate (ABR) using a Poisson regression model with adjustment for over dispersion. Data were analyzed according to treatment regimen prior to study entry: prior prophylaxis (rFIXFc, n=48; rIX-FP, n=40) or prior episodic treatment (n=43 and n=19, respectively). Relative treatment effects are presented as incidence rate ratios (IRR) with 95% confidence intervals (CI). Results: After adjustment for baseline characteristics, estimated ABR observed for rFIXFc and rIX-FP was not significantly different in patients on prior prophylaxis (1.87 versus 1.58; IRR 1.18, 95% CI 0.67–2.10) or prior episodic (2.25 versus 2.22; IRR 1.01 95% CI 0.40–2.57) regimens. Conclusion: This MAIC analysis shows that the estimated ABR for rFIXFc-treated patients from B-LONG was similar to that of rIX-FP-treated patients from PROLONG-9FP and, therefore, indicates that the two EHL therapies provide similar efficacy when used as prophylaxis for patients with hemophilia B. Trough levels differ between the two products (1–3% [targeted] versus 20% [observed], respectively), suggesting that trough level is not a surrogate indicator when ABR is used as a criterion for clinical efficacy when comparing these FIX products in hemophilia B.
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| 2. |
- Kozlowski, Piotr, 1969-, et al.
(author)
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The risk of renal disease is increased in lambda myeloma with bone marrow amyloid deposits
- 2017
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In: Journal of Blood Medicine. - : DOVE MEDICAL PRESS LTD. - 1179-2736. ; 8, s. 29-34
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Journal article (peer-reviewed)abstract
- Background: Light chain amyloidosis (AL) is a rare deposition disease and is present in 10-15% of patients with myeloma (MM). In contrast to symptomatic AL in MM, presence of bone marrow (BM) amyloid deposits (AD) in MM is not connected to kidney damage. Renal AD but not BM-AD occur mostly in MM with lambda paraprotein (lambda MM). Methods: We investigated amyloid presence in BM clots taken at diagnosis in 84 patients with symptomatic MM and compared disease characteristics in MM with kappa paraprotein (kappa MM)/lambda MM with and without BM-AD. Results: Lambda MM with BM-AD was compared with kappa MM without BM-AD, kappa MM with BM-AD, and lambda MM without BM-AD: lambda MM with BM-AD patients had a significantly higher mean creatinine level (4.23 mg/dL vs 1.69, 1.14, and 1.28 mg/dL, respectively) and a higher proportion presented with severe kidney failure (6/11 [55%] vs 6/32 [19%], 1/22 [5%], and 3/19 [16%], respectively). Proteinuria was more common in lambda MM with BM-AD patients compared with kappa MM without BM-AD patients (8/11 [73%] vs 5/32 [16%], respectively). Conclusion: Kidney damage was more common in lambda MM with BM-AD indicating presence of renal AD.
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| 3. |
- Masika, Lyidia V., et al.
(author)
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Forgetting to Take Medication, Treatment Adherence and Their Relationship with Viral Load Suppression Among People Living with HIV in the Kilimanjaro Region, Tanzania
- 2024
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In: HIV/AIDS - Research and Palliative Care. - 1179-1373. ; 16, s. 245-257
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Journal article (peer-reviewed)abstract
- Background: Antiretroviral therapy (ART) adherence is crucial for virological suppression and positive treatment outcomes among people living with HIV (PLHIV), but remains a challenge in ensuring patients achieve and sustain viral load suppression. Despite the recommended use of digital tools medications uptake reminders, the contribution of forgetting to take medication is unknown. This study investigated the contribution of forgetting to take medication on the total missed medication and its effects on detectable viral load (VL). Methods: This mixed-method research was conducted among children, adolescents, pregnant, and breastfeeding women living with HIV on ART in northern Tanzania. Forgetting to take medication constituted reporting to have missed medication due to forgetfulness. A multivariable logistic regression model was used to estimate the adjusted odds ratio (AOR) with a 95% confidence interval (CI) to determine the contribution of forgetting medication intakes on total missed medication and other factors associated with having a detectable VL. Results: Of 427 respondents, 33.3% were children, 33.4% adolescents, and 33.3% pregnant and breastfeeding women, whose median age (interquartile range) was 9 (7–12), 18 (16–18), and 31 (27–36) years, respectively. Ninety-two (22.3%) reported missing medication over the past month, of which 72 (17.9%) was due to forgetting. Forgetting to take medication (AOR: 1.75 95% CI: 1.01–3.06) and being on second-line regimen (AOR: 2.89 95% CI: 1.50–5.55) increased the chances of a detectable VL, while females had lower chances of detectable VL (AOR: 0.62 95% CI: 0.41–0.98). The themes on the reasons for forgetting to take medication from qualitative results included being busy with work and the importance of reminders. Conclusion: Forgetting to take medication is common among PLHIV and an important predictor of a detectable VL. This calls for the use of automated short message services (SMS) reminders or Digital Adherence Tools with reminders to improve and promote good ART adherence among PLHIV.
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