Akshara Richhariya

Systematic Review of Low-Density Lipoprotein Cholesterol Apheresis for the Treatment of Familial Hypercholesterolemia.

Background Apheresis is an important treatment for reducing low‐density lipoprotein cholesterol (LDL‐C) in patients with familial hypercholesterolemia (FH). We systematically reviewed the current literature surrounding LDL‐C apheresis for FH. Methods and Results Electronic databases were searched for publications of LDL‐C apheresis in patients with FH. Inclusion criteria include articles in English published in 2000–2013 that provide descriptions of practice patterns, efficacy/effectiveness, and costs related to LDL‐C apheresis in patients with FH. Data were stratified by country and FH genotype where possible. Thirty‐eight studies met the inclusion criteria: 8 open‐label clinical trials, 11 observational studies, 17 reviews/guidelines, and 2 health technology assessments. The prevalence of FH was not well characterized by country, and underdiagnosis was a barrier to FH treatment. Treatment guidelines varied by country, with some guidelines recommending LDL‐C apheresis as first‐line treatment in patients with homozygous FH and after drug therapy failure in patients with heterozygous FH. Additionally, guidelines typically recommended weekly or biweekly LDL‐C apheresis treatments conducted at apheresis centers that may last 2 to >3 hours per session. Studies reported a range for mean LDL‐C reduction after apheresis: 57–75% for patients with homozygous FH and 58–63% for patients with heterozygous FH. Calculated annual costs (in US

Quality of life results from a phase 3 study of brentuximab vedotin consolidation following autologous haematopoietic stem cell transplant for persons with Hodgkin lymphoma

2015) may reach US

Brentuximab vedotin consolidation post-autologous stem cell transplant in Hodgkin lymphoma patients at risk of residual disease: number needed to treat

66 374 to US

Real-World Economic Burden Associated with Transplantation-Related Complications

228 956 per patient for weekly treatment. Conclusions LDL‐C apheresis treatment may be necessary for patients with FH when drug therapy is inadequate in reducing LDL‐C to target levels. While apheresis reduces LDL‐C, high per‐session costs and the frequency of guideline‐recommended treatment result in substantial annual costs, which are barriers to the optimal treatment of FH.

Brentuximab vedotin in relapsed/refractory Hodgkin lymphoma following autologous stem cell transplant: A cost-effectiveness analysis updated with 5-year follow-up data from the pivotal trial.

Brentuximab vedotin (BV) significantly improved progression‐free survival in a phase 3 study in patients with relapsed or refractory Hodgkin lymphoma (RR‐HL) post‐autologous‐haematopoietic stem cell transplant (auto‐HSCT); we report the impact of BV on quality of life (QOL) from this trial. The European Quality of Life five dimensions questionnaire was administered at the beginning of each cycle, end of treatment, and every 3 months during follow‐up; index value scores were calculated using the time trade‐off (TTO) method for UK‐weighted value sets. Questionnaire adherence during the trial was 87·5% (N = 329). In an intent‐to‐treat analysis, compared with placebo, TTO scores in the BV arm did not exceed the minimally important difference (MID) of 0·08 except at month 15 (−0·084; 95% confidence interval, −0·143 to −0·025). On‐treatment index scores were similar between arms and did not reach the MID at any time point; mixed‐effect modelling showed that BV treatment effect was not significant (P = 0·2127). BV‐associated peripheral neuropathy did not meaningfully impact QOL. Utility scores for patients who progressed declined compared with those who did not; TTO scores between these patients exceeded the MID beginning at month 15. In conclusion, QOL decreased modestly with BV consolidation treatment in patients with RR‐HL at high risk of relapse after auto‐HSCT.

Brentuximab vedotin (BV) consolidation post-autologous stem cell transplant (ASCT) in patients (pts) with Hodgkin lymphoma (HL) at risk of residual disease: Number needed to treat (NNT) analysis.

Abstract The number needed to treat (NNT) with brentuximab vedotin consolidation therapy post-autologous stem cell transplant (ASCT) versus placebo in the phase 3 AETHERA trial to avoid one additional event of disease progression/death was evaluated. AETHERA included 329 Hodgkin lymphoma patients at increased risk of progression post-ASCT who received brentuximab vedotin 1.8 mg/kg (n = 165) or placebo (n = 164) on day 1 of each 21-d cycle (up to 16 cycles). Over 60 months, the NNT with brentuximab vedotin ranged from 4.08 to 7.79 for the intent-to-treat population, 3.18–6.07 for patients with ≥2 risk factors, and 2.98–5.65 for patients with ≥3 risk factors. At various time points, and dependent on the risk group, 3–8 patients would need to be treated with brentuximab vedotin consolidation therapy to prevent a disease progression/death, compared with placebo. Patients with increased risk of relapse may benefit most from brentuximab vedotin.

Cost-effectiveness of brentuximab vedotin in relapsed or refractory systemic anaplastic large cell lymphoma.

Approximately 20,000 hematopoietic cell transplantation (HCT) procedures are performed annually in the United States. Real-world data on the costs associated with post-transplantation complications are limited. Patients with hematologic malignancies aged ≥18 years undergoing autologous HCT (auto-HCT) or allogeneic HCT (allo-HCT) between January 1, 2011, and June 30, 2014, were identified in the Truven Health MarketScan Research Databases. Patients were required to have 12 months of continuous medical and pharmacy enrollment before and after HCT; patients who experience inpatient death within 12 months post-HCT were also included. Patients with previous HCT were excluded. Potential HCT-related complications were identified if they had a medical claim with a diagnosis code for relapse; infection; cardiovascular, renal, neurologic, pulmonary, hepatic, or gastrointestinal disease; secondary malignancy; thrombotic microangiopathy; or posterior reversible encephalopathy syndrome within 1 year post-HCT. Healthcare costs attributable to these complications were evaluated by comparing total costs in HCT recipients with complications and those without complications. The MarketScan Research Databases were further linked to the Social Security Administrations Master Death File to obtain patient death events in a subset of patients. A total of 2672 HCT recipients were included in the analysis. The mean ± SD age of recipients was 54.5 ± 11.6 years, and the majority of recipients (63.6%) underwent auto-HCT. Complications were identified in 81% of auto-HCT recipients and in 95.5% of allo-HCT recipients. Most complications occurred within 180 days post-HCT. Compared with Auto-HCT recipients without complications, those with complications incurred

Trends in use of intravenous bisphosphonates in patients with prostate cancer and newly diagnosed metastases to bone in two large U.S. integrated health systems.

51,475 higher adjusted total costs (P < .01). Compared with allo-HCT recipients without complications, those with complications incurred

Risk of skeletal-related events (SREs) in patients with breast cancer (BC) and newly diagnosed metastases to bone.

181,473 higher adjusted total costs (P < .01). Among the patients with mortality data, auto-HCT recipients with complications had a higher mortality rate (13.4% vs 5.7%, P < .01) and a lower probability of survival (P < .01) compared with those without complications. In allo-HCT recipients, however, the mortality rate and probability of survival were not significantly different between those with complications and those without complications. HCT recipients with complications were associated with considerable economic burden in terms of direct healthcare costs in a commercially insured population, and in the case of auto-HCT, a higher mortality rate was observed in those with complications.

Pulmonary events in patients with Hodgkin lymphoma after first line chemotherapy in the US Department of Defense (DOD) healthcare system.

19 Background: In 2015, the Scottish Medicines Consortium (SMC) made a positive recommandation for brentuximab vedotin (BV) in patients with relapsed or refractory (R/R) Hodgkin lymphoma (HL) who have received autologous stem cell transplantation (ASCT) based on 3-year follow-up data from the pivotal phase 2 single-arm trial (SG035-0003; NCT00848926). At 3-years, the incremental cost-effectiveness ratio (ICER) for brentuximab vedotin compared with chemotherapy +/- radiotherapy (C/R) was £43,731 per quality-adjusted life year (QALY). This study re-evaluated the cost-effectiveness analysis with 5-year follow-up data from the pivotal trial. METHODS A partitioned survival model was developed using a Scottish health system perspective over a lifetime time-horizon. Three health states were evaluated: progression-free survival (PFS), post-progression survival, and death. The relevant comparators were C/R, or C/R with intent to allogeneic stem cell transplantation. Clinical outcomes (PFS and overall survival [OS]) for BV were estimated based on data from the pivotal trial in 102 patients. A naïve comparison with the specified comparators was conducted using published survival data. ICERs were calculated with measures of the clinical outcomes, direct costs and QALYs. Deterministic and probabilistic sensitivity analyses were conducted to evaluate the robustness of the model. RESULTS The 5-year follow-up data reduced the base case ICER for BV from £43,731 to £38,769 per QALY versus C/R and increased the probability of cost-effectiveness. The variation in ICER for BV generated by the deterministic sensitivity analyses was also reduced resulting from reduced uncertainty in the estimation of long term clinical outcomes. CONCLUSIONS This update has strengthened the cost-effectiveness evidence for BV in patients with R/R HL post-ASCT. The 5-year follow-up has reduced the uncertainty in the long term outcomes and reduced the ICER, which is low in comparison to other treatments for orphan diseases approved by UK agencies. BV may therefore represent a cost-effective treatment option for this patient group.