Initiating immunoglobulin replacement therapy helps reduce severe infections and shifts healthcare resource utilization to outpatient services among US patients with inborn errors of immunity.

AIMS: Patients with inborn errors of immunity (IEI) are predisposed to severe recurrent/chronic infections, and often require hospitalization, resulting in substantial burden to patients/healthcare systems. While immunoglobulin replacement therapies (IgRTs) are the standard first-line treatment for most forms of IEI, limited real-world data exist regarding clinical characteristics and treatment costs for patients with IEI initiating such treatment. This retrospective analysis examined infection and treatment characteristics in US patients with IEI initiating IgRT with immune globulin infusion (human), 10% (IG10%). Healthcare resource utilization (HCRU) and associated costs before and after treatment initiation were compared. Additionally, the impact of COVID-19 on infection diagnoses was evaluated. METHODS: Patients with IEI initiating IG10% between July 2012 and August 2019 were selected from Merative MarketScan Databases using diagnosis/prescription codes. Patients were followed 6 months before and after first IG10% claim date. Demographic and clinical characteristics were described. Treatment characteristics and HCRU before and after IG10% initiation were compared. Infection diagnoses during 2020 and 2019 (March-December) were compared. RESULTS: The study included 1,497 patients with IEI diagnoses (mean age = 43.4 years) initiating IG10%, with frequently reported comorbidities like asthma (32.1%). Following IG10% initiation, fewer severe infection diagnoses (11.6% vs 19.9%), fewer infection-related inpatient (10.8% vs 19.5%) and outpatient services (71.6% vs 79.9%), and lower infection-related total healthcare costs ($7,849 vs $13,995; p < 0.001)-driven by lower inpatient costs ($2,746 vs $9,900)-were observed than before. Fewer patients had infection diagnoses during COVID-19 (22.8%) than the prior year (31.2%). CONCLUSION: Patients with IEI are susceptible to severe infections leading to high disease burden and treatment costs. Following IG10% initiation, we observed fewer infections, lower infection-related treatment costs, and shift in care (inpatient to outpatient) leading to significant cost savings. Among patients with IEI, 27% fewer infection diagnoses were observed during the early COVID-19 lockdown period than the prior year.

Some people are born with inborn errors of immunity, or IEI. This study included 1,497 people with IEI who recently started taking a drug called immunoglobulin therapy. Before taking this drug, the participants got infections easily, were hospitalized often, and had to take other costly medicines. After starting this drug, they had fewer infections and could be treated at the doctor's office. They had fewer infections during the COVID-19 pandemic than before the pandemic.

A systematic review of the cost and cost-effectiveness of immunoglobulin treatment in patients with hematological malignancies.

OBJECTIVES: Patients with hematological malignancies are likely to develop hypogammaglobulinemia. Immunoglobulin (Ig) is commonly given to prevent infections, but its overall costs and cost-effectiveness are unknown. METHODS: A systematic review was conducted following the PRISMA guidelines to assess the evidence on the costs and cost-effectiveness of Ig, administered intravenously (IVIg) or subcutaneously (SCIg), in adults with hematological malignancies. RESULTS: Six studies met the inclusion criteria, and only two economic evaluations were identified; one cost-utility analysis (CUA) of IVIg versus no Ig, and another comparing IVIg with SCIg. The quality of the evidence was low. Compared to no treatment, Ig reduced hospitalization rates. One study reported no significant change in hospitalizations following a program to reduce IVIg use, and an observational study comparing IVIg with SCIg suggested that there were more hospitalizations with SCIg but lower overall costs per patient. The CUA comparing IVIg versus no Ig suggested that IVIg treatment was not cost-effective, and the other CUA comparing IVIg to SCIg found that home-based SCIg was more cost-effective than IVIg, but both studies had serious limitations. CONCLUSIONS: Our review highlighted key gaps in the literature: the cost-effectiveness of Ig in patients with hematological malignancies is very uncertain. Despite increasing Ig use worldwide, there are limited data regarding the total direct and indirect costs of treatment, and the optimal use of Ig and downstream implications for healthcare resource use and costs remain unclear. Given the paucity of evidence on the costs and cost-effectiveness of Ig treatment in this population, further health economic research is warranted.

Economic evaluation: immunoglobulin vs prophylactic antibiotics in hypogammaglobulinemia and hematological malignancies.

ABSTRACT: Patients with hematological malignancies are at high risk of developing hypogammaglobulinemia (HGG) and infections. Immunoglobulin (Ig) is one recommended option to prevent these infections, but it is expensive, and its cost-effectiveness compared with other prevention strategies remains unknown. We conducted a trial-based economic evaluation from the Australian health care system perspective to estimate the 12-month cost-effectiveness of prophylactic Ig vs prophylactic antibiotics in 63 adults with HGG and hematological malignancies participating in the RATIONAL feasibility trial. Two analyses were conducted: (1) cost-utility analysis to assess the incremental cost per quality-adjusted life year (QALY) gained; and (2) cost-effectiveness analysis to assess the incremental cost per serious infection prevented (grade ≥3) and per any infection (any grade) prevented. Over 12 months, the total cost per patient was significantly higher in the Ig group than in the antibiotic group (mean difference, AU$29 140; P < .001). Most patients received IVIg, which was the main cost driver; only 2 patients in the intervention arm received subcutaneous Ig. There were nonsignificant differences in health outcomes. Results showed Ig was more costly than antibiotics and associated with fewer QALYs. The incremental cost-effectiveness ratio of Ig vs antibiotics was AU$111 262 per serious infection prevented, but Ig was more costly and associated with more infections when all infections were included. On average and for this patient population, Ig prophylaxis may not be cost-effective compared with prophylactic antibiotics. Further research is needed to confirm these findings in a larger population and considering longer-term outcomes. The trial was registered at the Australian and New Zealand Clinical Trials Registry as #ACTRN12616001723471.

Cost Utility of Lifelong Immunoglobulin Replacement Therapy vs Hematopoietic Stem Cell Transplant to Treat Agammaglobulinemia.

Importance: Lifelong immunoglobulin replacement therapy (IRT) is standard-of-care treatment for congenital agammaglobulinemia but accrues high annual costs ($30 000-$90 000 per year) and decrements to quality of life over patients' life spans. Hematopoietic stem cell transplant (HSCT) offers an alternative 1-time therapy, but has high morbidity and mortality. Objective: To evaluate the cost utility of IRT vs matched sibling donor (MSD) and matched unrelated donor (MUD) HSCT to treat patients with agammaglobulinemia in the US. Design, Setting, and Participants: This economic evaluation used Markov analysis to model the base-case scenario of a patient aged 12 months with congenital agammaglobulinemia receiving lifelong IRT vs MSD or MUD HSCT. Costs, probabilities, and quality-of-life measures were derived from the literature. Microsimulations estimated premature deaths for each strategy in a virtual cohort. One-way sensitivity and probabilistic sensitivity analyses evaluated uncertainty around parameter estimates performed from a societal perspective over a 100-year time horizon. The threshold for cost-effective care was set at $100 000 per quality-adjusted life-year (QALY). This study was conducted from 2020 across a 100-year time horizon. Exposures: Immunoglobulin replacement therapy vs MSD or MUD HSCT for treatment of congenital agammaglobulinemia. Main Outcomes and Measures: The primary outcomes were incremental cost-effectiveness ratio (ICER) expressed in 2020 US dollars per QALY gained and premature deaths associated with each strategy. Results: In this economic evaluation of patients with congenital agammaglobulinemia, lifelong IRT cost more than HSCT ($1 512 946 compared with $563 776 [MSD] and $637 036 [MUD]) and generated similar QALYs (20.61 vs 17.25 [MSD] and 17.18 [MUD]). Choosing IRT over MSD or MUD HSCT yielded ICERs of $282 166 per QALY gained over MSD and $255 633 per QALY gained over MUD HSCT, exceeding the US willingness-to-pay threshold of $100 000/QALY. However, IRT prevented at least 2488 premature deaths per 10 000 microsimulations compared with HSCT. When annual IRT price was reduced from $60 145 to below $29 469, IRT became the cost-effective strategy. Findings remained robust in sensitivity and probabilistic sensitivity analyses. Conclusions and Relevance: In the US, IRT is more expensive than HSCT for agammaglobulinemia treatment. The findings of this study suggest that IRT prevents more premature deaths but does not substantially increase quality of life relative to HSCT. Reducing US IRT cost by 51% to a value similar to IRT prices in countries implementing value-based pricing may render it the more cost-effective strategy.

Mitigating the risk of COVID-19 exposure by transitioning from clinic-based to home-based immune globulin infusion.

PURPOSE: Intravenous immune globulin (IVIG) therapy is used in patients with hypogammaglobulinemia to lower the risk of infections. IVIG and subcutaneous IVIG (SCIG) therapy have been to shown to be safe and effective when administered as clinic-based infusions. Concern from both patients and providers for increased transmission of the coronavirus disease 2019 (COVID-19) virus to immunosuppressed patients with scheduled medical visits and procedures made it necessary for us to reassess our process of how we manage patient care in general and chronic clinic infusions in particular. Here we describe our experience of transitioning patients from clinic-based to home based IVIG and/or SCIG infusions to decrease the risk of COVID-19 exposure. METHODS: Criteria were developed to identify high-risk immunosuppressed patients who would be appropriate candidates for potential conversion to home based IVIG infusions. Data were collected via chart review, and cost analysis was performed using Medicare Part B reimbursement data. A patient outcome questionnaire was developed for administration through follow-up phone calls. RESULTS: From March to May 2020, 45 patients met criteria for home-based infusion, with 27 patients (60%) agreeing to home-based infusion. Posttransition patient outcomes assessment, conducted in 26 patients (96%), demonstrated good patient understanding of the home-based infusion process. No infusion-related complications were reported, and 24 patients (92%) had no concerns about receiving future IVIG and/or SCIG doses at home. No patient tested positive for COVID-19 during the study period. Clinic infusion visits decreased by 26.6 visits per month, resulting in a total of 106 hours of additional available infusion chair time per month and associated cost savings of $12,877. CONCLUSION: Transition of clinic based to home based IVIG/SCIG infusion can be successfully done to decrease potential exposure during a pandemic in a high-risk immunosuppressed population, with no impact on patient satisfaction, adherence, or efficacy. The home-based infusion initiative was associated with a reduction in costs to patients and an increase in available chair time in the infusion clinic.

Characterization of antibodies in human immunoglobulin products from different regions worldwide.

AIM: The antibody levels against a broad spectrum of pathogens were assessed in commercial intravenous immunoglobulin (IVIG) manufactured from pooled plasma obtained from different global regions. METHODS: Twenty-four IVIG commercial lots from eight manufacturers corresponding to 12 brands were analyzed. The plasma was collected in 10 countries/regions. Depending on each pathogen, antibody levels were measured using specific commercial IgG-specific enzyme immunoassay kits or by cell culture neutralization test and guinea pig skin neutralization test. A principal component analysis was performed. RESULTS: For polio and diphtheria (reference markers of the US authorities), all IVIGs had relevant titers in accordance with reference levels. IVIGs from Canada, Australia, and the USA were positive for titers against globally distributed pathogens or those under vaccination programs in the developed world (parainfluenza, Epstein-Barr, varicella-zoster, influenza B, parvovirus B19, and measles viruses). IVIG from Taiwan and Hong Kong showed low antibody titers for these pathogens but high titers for Pseudomonas aeruginosa. IVIG from India had high titers for pathogens frequently found in developing countries (West Nile, dengue, chikungunya, and hepatitis E viruses and Streptococcus pneumoniae). IVIGs from Argentina, Spain, Israel, and Czechia showed intermediate antibody concentrations. CONCLUSION: The antibody profile in IVIG was greatly influenced by regional characteristics including climate, vaccination programs, and the prevalence of pathogens in the different countries and regions.

A Cost Comparison of Infliximab Versus Intravenous Immunoglobulin for Refractory Kawasaki Disease Treatment.

BACKGROUND AND OBJECTIVES: In 10% to 20% of cases, Kawasaki disease is refractory to intravenous immunoglobulin (IVIg), an expensive medication under a national shortage. Data suggest that infliximab is a viable alternative to a second dose of IVIg, with similar efficacy and safety. We compared the cost of a second IVIg dose to that of infliximab in the treatment of refractory Kawasaki disease (rKD). METHODS: A decision analysis model was used to compare rKD treatments: a second dose of IVIg at 2 g/kg versus infliximab at 10 mg/kg. Infliximab monitoring times were 24, 36, and 48 hours. Direct hospital costs beginning at rKD diagnosis were estimated by using 2016-2017 Truven MarketScan data. Redbook was used for drug costs. Calculations were applied to 3 hypothetical cohorts of 100 patients aged 2 (12.5 kg), 4 (16 kg), and 8 years (25.5 kg). Indirect costs included parental missed workdays. RESULTS: The total direct cost for children receiving IVIg was $1 677 801, $1 791 652, and $2 100 675 for the 2-, 4-, and 8-year-old cohorts. The direct cost of infliximab with 24 hours of monitoring was $853 042, $899 096, and $1 024 101, respectively. A 20% bidirectional sensitivity analysis revealed stability of our model, with overall cost savings with use of infliximab. With monitoring 48 hours after infliximab treatment, 20% changes in length of stay (LOS) tipped the balance for the 2- and 4-year-old cohorts. Overall, IVIg and infliximab LOS had the most influence on our model. CONCLUSIONS: Infliximab has potential to yield shorter LOS and significant cost savings in the treatment of rKD. Infliximab treatment, followed by 24 hours of monitoring, nearly halved hospital costs, regardless of age.

Home-based subcutaneous immunoglobulin for chronic inflammatory demyelinating polyneuropathy patients: A Swiss cost-minimization analysis.

PURPOSE: To compare the cost of two patient management strategies with similar efficacies for chronic inflammatory demyelinating polyneuropathy (CIDP) patients in the chronic phase: hospital-based IV immunoglobulin G (IVIg) and home-based subcutaneous immunoglobulin G (SCIg) associated with an interprofessional drug therapy management programme (initial training and follow-up). METHODS: A 48-week model-based cost-minimization analysis from a societal perspective was performed. Resources included immunoglobulin (IVIg: 1 g/kg/3 weeks; SCIg: 0.4 g/kg/week initially and 0.2 g/kg/week in the maintenance phase), hospital charges, time of professionals, infusion material, transport and losses of productivity for patients. Costs were expressed in Swiss francs (CHF) (1 CHF = 0.93€ = US$1.10, www.xe.com, 2020/10/28). RESULTS: The total costs of IVIg were higher than those of SCIg for health insurance and other payers: 114,747 CHF versus 86,558 CHF and 8,762 CHF versus 2,401 CHF, respectively. The results were sensitive to the immunoglobulin doses, as this was the main cost driver. The SCIg daily cost in the initial phase was higher for health insurance than hospital-based IVIg was, but the additional costs were compensated during the maintenance phase (from week 28). The professional costs associated with the switch were not fully covered by the insurance and were borne by the pharmacist and the nurse. CONCLUSIONS: SCIg for CIDP patients reinforced by an interprofessional drug therapy management programme may be a cost-effective and sustainable alternative to IVIg in the Swiss system context. From an economic perspective, this therapy alternative should be more widely supported by healthcare systems and proposed to eligible patients by professionals.

Economic Costs of Myasthenia Gravis: A Systematic Review.

OBJECTIVES: The objective of our study was to conduct a systematic literature review of economic costs (henceforth costs) associated with myasthenia gravis (MG). METHODS: We searched MEDLINE (through PubMed), CINAHL, Embase, PsycINFO, and Web of Science for studies reporting costs of MG published from inception up until March 18, 2020, without language restrictions. Two reviewers independently screened records for eligibility, extracted the data, and assessed included studies for risk of bias using the Newcastle-Ottawa Scale. Costs were inflated and converted to 2018 United States dollars ($). RESULTS: The search identified 16 articles for data extraction and synthesis. Estimates of costs of MG were found for samples from eight countries spanning four continents (Europe, North America, South America, and Asia). Across studies, the mean per-patient annual direct medical cost of illness was estimated at between $760 and $28,780, and cost per hospitalization between $2550 and $164,730. The indirect cost of illness was estimated at $80 and $3550. Costs varied considerably by patient characteristics, and drivers of the direct medical cost of illness included intravenous immunoglobulin and plasma exchange, myasthenic crisis, mechanical ventilatory support, and hospitalizations. CONCLUSIONS: We show that the current body of literature of costs of MG is sparse, limited to a few geographical settings and resource categories, mostly dated, and subject to non-trivial variability, both within and between countries. Our synthesis will help researchers and decision-makers identify gaps in the local health economic context of MG and inform future cost studies and economic evaluations in this patient population.

Treosulfan-based reduced toxicity hematopoietic stem cell transplantation in X-linked agammaglobulinemia: A cost-effective alternative to long-term immunoglobulin replacement in developing countries.

X-linked agammaglobulinemia (XLA) is a primary antibody disorder due to a mutation in the Bruton tyrosine kinase gene that requires lifelong immunoglobulin replacement resulting in a significant economic burden and treatment abandonment. Hematopoietic stem cell transplantation (HSCT) offers an alternative option for complete cure. In our series, two children with XLA underwent successful HSCT using a myeloablative conditioning with thiotepa, treosulfan, and fludarabine from a matched sibling donor. The second child had rejected his first graft following a busulfan-based regimen with resultant autologous reconstitution. At 6 months post-HSCT, serum IgG were normal, off IVIG, and had no infections. Both children after a median follow-up of 20 months have 100% chimerism. Treosulfan-based reduced toxicity myeloablative HSCT has encouraging results with a positive impact on the socioeconomics in developing countries.

Optimization of intravenous immune globulin use at a comprehensive cancer center.

PURPOSE: Intravenous immune globulin (IVIG) is a high-cost medication used in a diverse range of settings. At many institutions, IVIG is dosed using total body weight (TBW). Recent evidence suggests that alternative dosing weights reduce waste without compromising clinical outcomes. The objective of this study was to quantify the waste reduction potential generated through the use of alternative IVIG dosing weights. METHODS: We performed a retrospective analysis of all IVIG doses administered from January 2011 through January 2016 to adults (≥18 years). TBW and height at the time of administration were used to calculate prescribed dose (g/kg), ideal body weight (IBW), and adjusted body weight (AdjBW). Three dosing methods were analyzed, as follows: use of AdjBW if TBW is >120% IBW (method 1), AdjBW for all doses (method 2), and IBW for all doses (method 3). Outcomes included potential IVIG use averted, direct drug cost savings, and reductions in outpatient infusion times for each method. RESULTS: A total of 9,918 doses were administered to 2,564 patients over 5 years, representing an average usage of 75,994 g/year. If dosing methods 1, 2, and 3 had been used, the annual use of IVIG would have decreased by 21.9% (16,658 g/year, p < 0.001), 24.2% (18,371 g/year, p < 0.001), and 35.9% (27,252 g/year, p < 0.001), respectively. This translates into average annual cost differences of $2.37 million, $2.62 million, and $3.89 million and average annual outpatient infusion time savings of 841 hours, 920 hours, and 1,366 hours, respectively. CONCLUSION: IVIG dosing optimization through use of alternative dosing weights represents a significant source of waste reduction and cost reduction.

Medication stewardship using computerized clinical decision support: A case study on intravenous immunoglobulins.

Background: Healthcare delivery organizations face increasing pressure to manage the use of medications in terms of safety, waste reduction, and cost containment. Objective: To describe a computerized provider order entry (CPOE) system intervention to optimize use of a commonly ordered, high-cost therapeutic: intravenous immune globulin (IVIG). Design: Description of IVIG order configuration, medication use patterns, and subsequent order set configuration development in a CPOE system. Measurements: IVIG orders were extracted from the CPOE system before and after the implementation of a specialty orderset to determine the indications for use, dosing, and duration of therapy. Orders were compared to a theoretical dosing schedule created from published evidence and data from a prior medication use evaluation. Results: During 36 months before the implementation of the IVIG order set, 1965 IVIG orders were reviewed. The prescribed IVIG dose varied considerably from the expected dose (mean = -1.8, range = -4.9-1.5). In the 27 months after order set implementation, 848 IVIG orders were reviewed. The prescribed IVIG dose was closer to the expected dose (mean = -1.2, range = -3.9-2.6, P < .0001). Conclusions: Order configuration processes are cumbersome and time-consuming, but can be streamlined to enhance a medication's usage in the healthcare system. A better understanding of institution-specific ordering patterns may facilitate more efficient and effective order configuration and optimize drug use.

Intravenous Immunoglobin Utilization Study in a Teaching Hospital.

BACKGROUND: Intravenous immunoglobulin (IVIG) is an immunomodulating agent that has several actions. The aim of this study was to investigate the indications of IVIG according to available evidence and the cost in our center. METHODS: This retrospective study was conducted between September 2017 and June 2018 at a teaching hospital affiliated with Iran University of Medical Sciences, Tehran, Iran. Patients' demographic data and disease, indication for IVIG use, its dosage and treatment regimen and previous and concurrent treatments were assessed. The collected data were compared with the present criteria for the pattern of IVIG usage. The last version of Lexicomp® was used as the reference for indications of the administrated drug and its dosage. RESULTS: A total of 119 patients received IVIG during the study period. The wards with the most frequent IVIG prescription were the neurology (46.2%) and neonatal intensive care unit (21%). The most common reasons of IVIG therapy were various inflammatory neurological disorders. IVIG was used in 22, 43 and 54 cases according to on-label, off- label and other indications, respectively. The total price was higher for off-label indications for IVIG ($254343.75) than on-label indications ($152625). As well, $107250 was exhausted for cases in which there was not sufficient evidence. CONCLUSION: One important aspects of this study was the use of IVIG in cases other than on-label indications. Although a number of studies support IVIG therapy in some diseases, further trials are needed to establish efficacy and safety in these fields.

Managing cost of care and healthcare utilization in patients using immunoglobulin agents.

The introduction of human immunoglobulin (Ig) therapies 40 years ago reduced the risk of often life-threatening infections for individuals with one of several immune-related conditions known as primary immunodeficiencies. Since then, the use of Ig has expanded to numerous other conditions. However, even though less than 1% of covered lives under Medicare or commercial insurers require Ig, it is in the top 5 drug categories in terms of annual spending. The cost of Ig is directly related to the type of delivery method used and the site of care. Numerous studies attest to the efficacy and cost savings of shifting Ig to the home setting, as well as shifting patients from intravenous Ig (IVIG) to subcutaneous Ig (SCIG). In addition, surveys find that patients with primary immunodeficiencies prefer home delivery, with patient evaluations also finding a preference for SCIG. Payers have numerous options to ensure Ig is used appropriately for the right patient in the right setting. These include formulary management, site-of-care programs, education for providers and patients on the possibility of switching from IVIG to SCIG, preauthorization policies that restrict the use of Ig to certain specialties for specific indications, implementation of evidence-based coverage criteria, and shifting coverage from the medical to the pharmacy benefit.

Cost-utility analysis comparing hospital-based intravenous immunoglobulin with home-based subcutaneous immunoglobulin in patients with secondary immunodeficiency.

BACKGROUND AND OBJECTIVE: Immunoglobulin replacement therapy (IRT) is often used to support patients with primary immunodeficiency disease (PID) and secondary immunodeficiency disease (SID). Home-based subcutaneous immunoglobulin (SCIg) is reported to be a cheaper and more efficient option compared to hospital-based intravenous immunoglobulin (IVIg) for PID. In contrast, there is little information on the cost-effectiveness of IRT in SID. However, patients who develop hypogammaglobulinaemia secondary to other conditions (SID) have different clinical aetiology compared to PID. This study assesses whether SCIg provides a good value-for-money treatment option in patients with secondary immunodeficiency disease (SID). METHODS: A Markov cohort simulation model with six health states was used to compare cost-effectiveness of IVIg with SCIg from a healthcare system perspective. The costs of treatment, infection and quality-adjusted life years (QALYs) for IVIg and SCIg treatment options were modelled with a time horizon of 10 years and weekly cycles. Deterministic and probabilistic sensitivity analyses were performed around key parameters. RESULTS: The cumulative cost for IVIg was A$151 511 and for SCIg A$144 296. The QALYs with IVIg were 3·07 and with SCIg 3·51. Based on the means, SCIg is the dominant strategy with better outcomes and at lower cost. The probabilistic sensitivity analysis shows that 88·3% of the 50 000 iterations fall below the nominated willingness to pay threshold of A$50 000 per QALY. Therefore, SCIg is a cost-effective treatment option. CONCLUSION: For SID patients in Queensland (Australia), the home-based SCIg treatment option provides better health outcomes and cost savings.

Clinical and economic comparison of an individualised immunoglobulin protocol vs. standard dosing for chronic inflammatory demyelinating polyneuropathy.

BACKGROUND: The clinical and economic implications of an individualised intravenous immunoglobulin (IVIg) protocol for chronic inflammatory demyelinating polyneuropathy (CIDP) are unknown. Comparison with standard dosing regimens has not been performed. METHODS: We retrospectively studied 47 IVIg-treated subjects with CIDP over 4 years with an individualised, outcome-measured, dose-modifying protocol. We evaluated responder and remission rates, clinical improvement levels and dose requirements. We compared clinical benefits and costs with those reported with standard dosing at 1 g/kg every 3 weeks. RESULTS: The IVIg-responder rate was 83% and the 4-year remission rate was 25.6%. Mean IVIg dose requirements were 22.06 g/week (SD:15.29) in patients on ongoing therapy. Dose range was wide (5.83-80 g/week). Mean infusion frequency was every 4.34 weeks (SD:1.70) and infusion duration of 2.79 days (SD:1.15). Mean Overall Neuropathy Limitation Scale improvement was 2.54 (SD:1.89) and mean MRC sum score improvement of 12.23 (SD:7.17) in IVIg-responders. Mean modified-INCAT (Inflammatory Neuropathy Cause and Treatment) score improvement was similar (p = 0.47) and mean MRC sum score improvement greater (p < 0.001) in our cohort, compared to the IVIg-treated arm of the ICE Study. Mean drug costs were GBP 37,660/patient/year (€ 43,309) and mean infusion-related costs of GBP 17,115/patient/year (€ 19,682), totalling GBP 54,775/patient/year (€ 62,991). Compared to standard dosing using recorded weight, mean savings were of GBP 13,506/patient/year (€ 15,532). Compared to standard dosing using dosing weight, savings were of GBP 6,506/patient/year (€ 7,482). CONCLUSION: Our results indicate that an individualised IVIg treatment protocol is clinically non-inferior and 10-25% more cost-effective than standard dosing regimens in CIDP.

Clinical and economical impacts of guideline implementation by the pharmaceutical care unit for high cost medications in a referral teaching hospital.

BACKGROUND: Irrational drug use is a global health challenge in all healthcare settings, such as hospitals. This study evaluated the impact of an intervention by the pharmaceutical care unit on the use pattern of high-value medications and their direct costs in a referral hospital. METHODS: This interventional, prospective study was carried out in clinical wards of Namazi Hospital (Shiraz University of Medical Sciences) during six months from May 2015 to October 2015. Clinical pharmacists completed the checklists for albumin, intravenous (IV) pantoprazole, and IV immune globulin (IVIG), as three high-cost medications. When ordering these medications, the physicians were asked to complete the checklists. Then, trained pharmacists examined the checklists, based on the clinical and paraclinical conditions. RESULTS: The total number of administered medications and their relative cost decreased by 50.76% through guideline implementation; the difference was significant (P <  0.001). In addition, the direct cost of albumin and IV pantoprazole significantly decreased (55.8% and 83.92%, respectively). In contrast, the direct cost of IVIG increased by 40.9%. After guideline implementation, the monthly direct cost of all three medications decreased by $77,720 (55.88%). The all-cause in-hospital mortality rate did not change significantly due to the intervention. The median length of hospital stay was six and seven days, respectively in the pre- and post-intervention periods. CONCLUSION: Based on the findings, implementation of guidelines by the pharmaceutical care unit caused a significant reduction in albumin and IV pantoprazole consumption and reduced their direct costs in a referral center in Iran.

Cost-minimization analysis in the Indian subcontinent for treating Guillain Barre Syndrome patients with therapeutic plasma exchange as compared to intravenous immunoglobulin.

BACKGROUND: Therapeutic Plasma Exchange (TPE) and Intravenous Immunoglobulin both are first-line treatments for Guillain Barre Syndrome; however, there is a significant difference in cost. We undertook this study to assess the cost minimization for treating Guillain Barre Syndrome patients. METHODS: A prospective randomized controlled trial was undertaken, in which 40 Guillain Barre Syndrome (GBS) patients with a GBS disability score of grade four and five were enrolled. A societal perspective was adopted for the analysis and assessment of both the health system cost and out-of-pocket expenditures. Cost-minimization analysis was undertaken as both the treatments were equally effective at the end of 12 weeks. RESULTS: No statistically significant differences were observed in the GBS Disability scores during overall treatment course in both treatment groups. The Out-of-pocket cost for the immunoglobulin (IVIG) group was INR 219 247 (4298 USD) and for the TPE group was INR 104 070 (2040.5 USD). Overall INR 86 685 ($1700), that is, 53% higher cost was observed in IVIG group without any concomitant health outcome benefit. CONCLUSION: In comparison with IVIG, TPE appears to be the better option for treatment of GBS in cost-constraint countries like ours to provide an economic treatment option to most average people.