Efficacy and safety of ruxolitinib after and versus interferon use in the RESPONSE studies.

Ruxolitinib was well tolerated and superior to best available therapy (including interferon [IFN]) in controlling hematocrit without phlebotomy eligibility, normalizing blood counts, and improving polycythemia vera-related symptoms in the Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 (INCB018424) Tablets Versus Best Available Care (RESPONSE) studies. This ad hoc analysis focuses on ruxolitinib in relation to IFN in the RESPONSE studies, with attention on the following: (1) safety and efficacy of ruxolitinib and best available therapy in patients who received IFN before study randomization, (2) safety and efficacy of IFN during randomized treatment in best available therapy arm, and (3) use of ruxolitinib after crossover from best available therapy in IFN-treated patients. IFN exposure before randomization had little effect on the efficacy or safety of ruxolitinib. In the randomized treatment arms, ruxolitinib was superior to IFN in efficacy [hematocrit control (RESPONSE = 60% of ruxolitinib vs 23% of IFN patients; RESPONSE-2 = 62% of ruxolitinib vs 15% of IFN patients)] and was tolerated better in hydroxyurea-resistant or hydroxyurea-intolerant patients. After crossing over to receive ruxolitinib, patients who had initially received IFN and did not respond had improved hematologic and spleen responses (62% of patients at any time after crossover) and an overall reduction in phlebotomy procedures. Rates and incidences of the most common adverse events decreased after crossover to ruxolitinib, except for infections (primarily grade 1 or 2). These data suggest that ruxolitinib is efficacious and well tolerated in patients who were previously treated with IFN. The RESPONSE (NCT01243944) and RESPONSE-2 (NCT02038036) studies were registered at clinicaltrials.gov .

The impact of myeloproliferative neoplasms (MPNs) on patient quality of life and productivity: results from the international MPN Landmark survey.

Myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET) are myeloproliferative neoplasms (MPNs) associated with high disease burden, reduced quality of life (QOL), and shortened survival. To assess how MPNs affect patients, we conducted a global MPN Landmark survey. This online survey of patients with MPNs and physicians was conducted in Australia, Canada, Germany, Japan, Italy, and the United Kingdom. The survey measured MPN-related symptoms and the impact of MPNs on QOL and the ability to work as well as disease-management strategies. Overall, 219 physicians and 699 patients (MF, n = 174; PV, n = 223; ET, n = 302) completed the survey; 90% of patients experienced MPN-related symptoms. The most frequent and severe symptom was fatigue. Most patients experienced a reduction in QOL, including those with low symptom burden or low-risk scores. A substantial proportion of patients reported impairment at work and in overall activity. Interestingly, physician feedback and blood counts were the most important indicators of treatment success among patients, with improvements in symptoms and QOL being less important. Regarding disease management, our study revealed a lack of alignment between physician and patient perceptions relating to communication and disease management, with patients often having different treatment goals than physicians. Overall, our study suggested that therapies that reduce symptom burden and improve QOL in patients with MPNs are crucial in minimizing disease impact on patient daily lives. Additionally, our findings showed a need for improved patient-physician communication, standardized monitoring of symptoms, and agreement on treatment goals.

Real-World Assessment of Clinical Outcomes in Patients with Lower-Risk Myelofibrosis Receiving Treatment with Ruxolitinib.

Few trial-based assessments of ruxolitinib in patients with lower-risk myelofibrosis (MF) have been conducted, and no studies have made such assessments in a real-world population. We assessed changes in spleen size and constitutional symptoms during ruxolitinib treatment using a retrospective, observational review of anonymized US medical record data of patients diagnosed with IPSS low-risk (n = 25) or intermediate-1-risk (n = 83) MF. The majority of patients were male (low risk, 60%; intermediate-1 risk, 69%). Most patients (92% and 77%) were still receiving ruxolitinib at the medical record abstraction date (median observation/exposure time, 8 months). The proportion of patients with moderate or severe palpable splenomegaly (≥10 cm) decreased from diagnosis (56%) to best response (12%). Fatigue was reported in 47% of patients and was the most common constitutional symptom. For most symptoms in both risk groups, shifts in the distribution of severity from more to less severe from diagnosis to best response were observed. Both patients with low-risk and intermediate-1-risk MF experienced a substantial decrease in spleen size with ruxolitinib treatment in real-world settings. For most symptoms examined, there were distinct improvements in the distribution of severity during ruxolitinib treatment. These findings suggest that patients with lower-risk MF may benefit clinically from ruxolitinib treatment.

[Incidence, resource use and costs associated with postherpetic neuralgia: a population-based retrospective study]. / Incidencia, uso de recursos y costes asociados a la neuralgia postherpetica: estudio retrospectivo de base poblacional.

AIM: To determine the incidence rate, the treatment administered and the use of health resources and health, and their respective costs in patients with postherpetic neuralgia (PHN). PATIENTS AND METHODS: We performed an observational design, made from retrospective review of patient records from six primary care centers and one hospital. All patients > 30 years consulting for PHN between 1/1/2007 and 31/12/2010 were included. Prepared two study groups according to presence / absence of PHN. Follow up was for one year. MAIN MEASURES: socio-demographic, treatment and co-morbidity. The cost model differed direct healthcare costs (primary care/specialist) and indirect (productivity). STATISTICAL ANALYSIS: logistic regression models and analysis of covariance (p < 0.05). RESULTS: 1506 patients were recruited, age: 61.2 years female: 59.2%. 15.1% (n = 228, 95% CI = 8.1-22.1%) had a PHN (incidence rate: 0.8/1,000 inhabitants/year; 95% CI = 0.7-0.9/1,000 population/year), and increased with age (≥ 65 years: 19.7%). The PHN was principally associated with: psychosis (OR = 3.9), dementia (OR = 2.3), depression (OR = 1.8) and age (OR = 1.1), p < 0.03. Drugs use was higher (5.3 vs. 3.3; p < 0.001). The cost in primary care was 63.1% and 24.7% indirect. Total cost €1827.1 vs. €457.5 (p = 0.003), respectively, due to higher labour productivity losses (€692.2 vs. €62.4) and health costs (€1135 vs. €395.1); p < 0.001. All cost components maintained these differences. CONCLUSIONS: PHN is a frequent complication. These patients have a significant economic burden. The cost increases with age.

Incidencia, uso de recursos y costes asociados a la neuralgia postherpética: estudio retrospectivo de base poblacional / Incidence, resource use and costs associated with postherpetic neuralgia: a population-based retrospective study

Objetivo. Determinar la tasa de incidencia, los tratamientos administrados y la utilización de recursos sanitarios y no sanitarios, y sus costes respectivos, en pacientes con neuralgia postherpética (NPH). Pacientes y métodos. Se efectuó un estudio observacional, realizado a partir de la revisión retrospectiva de registros de pacientes de seis centros de atención primaria y un hospital. Se incluyeron todos los pacientes mayores de 30 años que demandaron atención por NPH entre el 1 de enero de 2007 y el 31 de diciembre de 2010. Se elaboraron dos grupos de estudio según la presencia/ausencia de NPH. El seguimiento fue durante un año, y las principales medidas fueron sociodemográficas, tratamientos y comorbilidad. El modelo de costes diferenció los costes sanitarios directos (atención primaria/ especializada) e indirectos (productividad laboral). Se realizó un análisis estadístico con modelos de regresión logística y análisis de la covarianza (p < 0,05). Resultados. Se reclutaron 1.506 pacientes, con una edad media de 61,2 años, de los cuales el 59,2% eran mujeres. El 15,1% (n = 228; intervalo de confianza al 95%, IC 95% = 8,1-22,1%) presentó NPH (tasa de incidencia: 0,8/1.000 habitantes/ año; IC 95% = 0,7-0,9/1.000 habitantes/año) y el porcentaje aumentó con la edad (≥ 65 años: 19,7%). La NPH se asoció principalmente a psicosis (odds ratio, OR = 3,9), demencia (OR = 2,3), depresión (OR = 1,8) y edad (OR = 1,1); p < 0,03. El uso de medicamentos fue superior (5,3 frente a 3,3; p < 0,001). El coste en atención primaria fue del 63,1% y los costes indirectos del 24,7%. Los costes totales fueron de 1.827,1 frente a 457,5 euros (p = 0,003), respectivamente, debido a mayores pérdidas de productividad laboral (692,2 frente a 62,4 euros) y costes sanitarios (1.135 frente a 395,1 euros; p < 0,001). Todos los componentes del coste mantuvieron estas diferencias. Conclusiones. La NPH es una complicación frecuente. Estos pacientes presentan una importante carga económica y el coste aumenta con la edad (AU)

Aim. To determine the incidence rate, the treatment administered and the use of health resources and health, and their respective costs in patients with postherpetic neuralgia (PHN). Patients and methods. We performed an observational design, made from retrospective review of patient records from six primary care centers and one hospital. All patients > 30 years consulting for PHN between 1/1/2007 and 31/12/2010 were included. Prepared two study groups according to presence / absence of PHN. Follow up was for one year. Main measures: socio-demographic, treatment and co-morbidity. The cost model differed direct healthcare costs (primary care/ specialist) and indirect (productivity). Statistical analysis: logistic regression models and analysis of covariance (p < 0.05). Results. 1506 patients were recruited, age: 61.2 years female: 59.2%. 15.1% (n = 228, 95% CI = 8.1-22.1%) had a PHN (incidence rate: 0.8/1,000 inhabitants/year; 95% CI = 0.7-0.9/1,000 population/year), and increased with age (≥ 65 years:19.7%). The PHN was principally associated with: psychosis (OR = 3.9), dementia (OR = 2.3), depression (OR = 1.8) and age (OR = 1.1), p < 0.03. Drugs use was higher (5.3 vs. 3.3; p < 0.001). The cost in primary care was 63.1% and 24.7% indirect. Total cost €1827.1 vs. €457.5 (p = 0.003), respectively, due to higher labour productivity losses (€692.2 vs. €62.4) and health costs (€1135 vs. €395.1); p < 0.001. All cost components maintained these differences. Conclusions. PHN is a frequent complication. These patients have a significant economic burden. The cost increases with age (AU)