RESUMO
Background: Prior clinical trials have suggested that home-based Ig treatment in multifocal motor neuropathy (MMN) and chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) and its variant Lewis-Sumner syndrome (LSS) is safe and effective and is less costly than hospital-administered intravenous immunoglobulin (IVIg). Methods: A French prospective, dual-center, cost minimization analysis was carried out to evaluate IVIg administration (5% concentrated) at home versus in hospital with regard to costs, patients' autonomy, and patients' quality of life. The primary endpoint was the overall cost of treatment, and we adopted the perspective of the payer (French Social Health Insurance). Results: Twenty-four patients aged 52.3 (12.2) years were analyzed: nine patients with MMN, eight with CIDP, and seven with LSS. IVIg (g/kg) dosage was 1.51 ± 0.43 in hospital and 1.52 ± 0.4 at home. Nine-month total costs per patient extrapolated to 1 year of treatment were 48,189 ± 26,105 versus 91,798 ± 51,125 in the home and hospital groups, respectively (p < .0001). The most frequently reported factors for choosing home treatment were the good tolerance and absence of side effects of IVIg administration, as well as a good understanding of the advantages and drawbacks of home treatment (75% of respondents). The mRankin scores before and after switch to home treatment were 1.61 ± 0.72 and 1.36 ± 0.76, respectively (p = .027). Discussion: The switch from hospital-based to home-based IVIg treatment for patients with immune neuropathy represents potentially significant savings in the management of the disease.
Assuntos
Serviços de Assistência Domiciliar/economia , Hospitalização/economia , Imunoglobulinas Intravenosas , Polineuropatias , Polirradiculoneuropatia Desmielinizante Inflamatória Crônica , Qualidade de Vida , Adulto , Autoimunidade , Custos e Análise de Custo , Feminino , França/epidemiologia , Humanos , Imunoglobulinas Intravenosas/economia , Imunoglobulinas Intravenosas/uso terapêutico , Masculino , Pessoa de Meia-Idade , Polineuropatias/economia , Polineuropatias/imunologia , Polineuropatias/psicologia , Polineuropatias/terapia , Polirradiculoneuropatia Desmielinizante Inflamatória Crônica/economia , Polirradiculoneuropatia Desmielinizante Inflamatória Crônica/imunologia , Polirradiculoneuropatia Desmielinizante Inflamatória Crônica/psicologia , Polirradiculoneuropatia Desmielinizante Inflamatória Crônica/terapia , Estudos ProspectivosRESUMO
The efficacy of intravenous immunoglobulins (IVIg) in patients with autoimmune diseases (AID) has been known for several decades. Majority of these patients received IVIg in hospital. A retrospective study was conducted in 22 centers in France to evaluate the feasibility of the administration of Tegeline, an IVIg from LFB Biomedicaments, and assess its safety at home, compared to in hospital, in patients with AID. The included patients were at least 18 years old, suffering from AID, and treated with at least 1 cycle of Tegeline at home after receiving 3 consecutive cycles of hospital-based treatment with Tegeline at a dose between 1 and 2 g/kg/cycle. Forty-six patients with AID, in most cases immune-mediated neuropathies, received a total of 138 cycles of Tegeline in hospital and then 323 at home. Forty-five drug-related adverse events occurred in 17 patients who received their cycles at home compared to 24 adverse events in hospital in 15 patients. Serious adverse events occurred in 3 patients during home treatment, but they were not life-threatening and did not lead to discontinuation of Tegeline. Forty-five patients continued their treatment with Tegeline at home or in hospital; 39 (84.8%) were still receiving home treatment at the end of the study. In conclusion, the study demonstrates the good safety profile of Tegeline administered at home at high doses in patients with AID who are eligible for home administration of Tegeline.
Assuntos
Doenças Autoimunes/tratamento farmacológico , Imunoglobulinas Intravenosas/administração & dosagem , Síndromes de Imunodeficiência/tratamento farmacológico , Doenças do Sistema Nervoso Periférico/tratamento farmacológico , Adulto , Idoso , Doenças Autoimunes/imunologia , Doenças Autoimunes/patologia , Feminino , França , Humanos , Imunoglobulinas Intravenosas/efeitos adversos , Síndromes de Imunodeficiência/imunologia , Síndromes de Imunodeficiência/patologia , Masculino , Pessoa de Meia-Idade , Doenças do Sistema Nervoso Periférico/imunologia , Doenças do Sistema Nervoso Periférico/patologia , Estudos Retrospectivos , Resultado do TratamentoRESUMO
OBJECTIVE: To assess the efficacy and safety of intraveinous immunoglobulin (IV Ig) in patients with chronic inflammatory demyelinating polyneuropathy (CIDP) at 4, 7 and 12months. METHODS: A national multicenter retrospective study was conducted by LFB Biotehcnologies in patients with CIDP who had received at least one cycle of a 5% polyvalent IV Ig, Tegeline(®), from LFB biomédicaments between 1995 and 2004. The primary endpoint was the efficacy of IV Ig at 4 months, which was defined as the responder rate based on the modified Rankin scale. Several secondary endpoints were assessed: safety and efficacy (i.e., responders according to the investigators' overall assessment of the patients' status) at 4, 7 and 12 months. The analysis was performed at 7 months only (due to missing data for 12 months and few patients). RESULTS: A total of 26 patients were included who had received between 1 and 6 cycles of IV Ig (mean 3±2) with a median follow-up of 9.9 months. The responder rate at 4 months based on the modified Rankin scale was 52% (95% CI 0.313-0.722), whereas the responder rate with placebo reported in the literature (meta-analysis including results from van Schaik and an ICE study) is 18% (P<0.001). Responder patients at 4 months were still responders at 7 months. The overall safety of IV Ig was good, with adverse events of mild to moderate severity, which resolved without sequelae and were expected adverse events of IV Ig. CONCLUSION: This retrospective study confirmed both the efficacy of IV Ig at 4 months in the treatment of chronic inflammatory demyelinating polyneuropathy and the favorable safety profile of the product.
Assuntos
Imunoglobulinas Intravenosas/uso terapêutico , Imunoterapia/métodos , Polirradiculoneuropatia Desmielinizante Inflamatória Crônica/terapia , Feminino , Seguimentos , Humanos , Imunoglobulinas Intravenosas/efeitos adversos , Masculino , Polirradiculoneuropatia Desmielinizante Inflamatória Crônica/imunologia , Estudos Retrospectivos , Resultado do TratamentoRESUMO
INTRODUCTION: Primary Immunodeficiencies (PIDs) represent a heterogeneous group of rare diseases characterized by increased susceptibility to infections, often accompanied by a diverse immuno-pathological manifestations (autoimmunity, inflammation, benign or malignant lymphoproliferative disorders). The precise prevalence of PIDs in France is not known but is estimated to represent approximately 5 000 patients. METHODS: To better understand the situation of PID in France and gain an insight as to the care of these patients, we conducted a national survey by sending a questionnaire to physicians potentially involved in the care of PID patients. RESULTS: The majority of physicians follow only a few PID patients but the diagnostic and therapeutic attitudes are generally satisfactory. DISCUSSION: These results underscore the need to coordinate the care of PID patients in France as part of national networks. The approach adopted by the CEREDIH (French reference center for hereditary immune deficiencies) and DEF-I french national cohort identifying patients with DIP, will optimize the management of PID by defining diagnostic and therapeutic guidelines. In addition, these networks will provide valuable data regarding the incidence of PID and their complications.