Lack of awareness of systemic lupus erythematosus and its consequences in a cohort of moderate and severe patients in Spain: The LupusVoice study.

BACKGROUND AND OBJECTIVES: Systemic lupus erythematosus (SLE) is an autoimmune condition that can highly impact patients' quality of life (QoL). However, there is a lack of knowledge about SLE, affecting the general population and health care professionals (HCPs) alike. This lack of knowledge has negative implications for patients and the healthcare system, worsening prognosis, negatively impacting QoL, and increasing healthcare utilization. The aim of this paper is to draw attention, according to the perspective of the participants of this study, to the lack of awareness of SLE and its consequences in Spain, and to suggest improvements. PATIENTS AND METHODS: This qualitative, descriptive, observational, multicenter, and cross-sectional study included 40 patients with moderate or severe SLE, recruited during their routine visits in six university hospitals in Spain. The study also included 11 caregivers and 9 HCPs. All participants were individually interviewed. Data from the interviews were coded and analyzed thematically by two anthropologists following a phenomenological perspective. RESULTS: Our study identified a lack of disease awareness among primary care physicians, emergency medicine doctors, and other specialists treating SLE symptomatology. This led to diagnostic delays, which had a clinical and emotional impact on patients. Furthermore, symptom awareness was found to be context dependent. Differences in symptom awareness between HCPs and patients led to a mismatch between the severity evaluation made by doctors and patients. Some HCPs did not consider the limitations of the current severity evaluation of SLE, and therefore attributed symptoms potentially caused by SLE to the unfavorable socioeconomic conditions patients lived in. Finally, a lack of social awareness among friends, family members, and romantic partners led to lower social support, increased isolation, and negative physical and emotional impact for patients. Gender differences in the provision of support were identified. CONCLUSION: This study highlights the need to increase SLE awareness among patients, HCPs, and the broader public in order to improve patient QoL. Being aware of the clinical and emotional impact of such lack of awareness, as well as the role played by context on the patient experience of SLE, is a crucial step towards achieving this goal.

Integration of persistence in the 5P-medicine approach for age-related chronic diseases.

5P medicine is defined as Personalized, Predictive, Preventive, Participatory, and Population-based. 5P medicine may be improved by including a factor that could provide information about the therapeutic value of a particular drug treatment and measure its effectiveness in clinical practice. We propose that this factor may be treatment persistence, and that its addition to 5P medicine would allow to define a new improved 6P medicine. Persistence is the length of time between initiation and the last dose, which immediately precedes discontinuation, that is, a definitive suspension of the treatment. By including this sixth P, the persistence, we would be able to present the value of a treatment for each individual patient with its own characteristics, state of the disease, with more than one age-related diseases and patient journey. Persistence is a concept of the value of a treatment that includes the three main stakeholders of the pharmacotherapeutic process: Patient, Physician, and Pharmacist. Persistence is becoming a useful measure to evaluate the long-term effectiveness of therapies in real-world setting in chronic diseases. Drug treatments with longer persistence are more likely to provide better disease control and to be amenable to dose adjustment in order to optimize treatment cost in age-related chronic diseases. Long-term persistence could be a measure of a drug´s real-world performance and has been shown to aid in clinical decision-making.

Six months multicentre pilot open label single-arm study to evaluate patient experience, acceptability and satisfaction of switching certolizumab pegol from a prefilled syringe or autoinjection pen to an AVA® e-Device in rheumatoid arthritis, psoriatic arthritis and axial spondyloarthritis patients.

WHAT IS KNOWN AND OBJECTIVE: The study aimed to assess acceptability and patient experience of Certolizumab (CZP) self-injection with AVA® and clarify patient device preference after switching CZP from the syringe or auto-injection pen to AVA® in rheumatoid arthritis (RA), psoriatic arthritis (PsA) and axial spondyloarthritis (axSpA) patients. METHOD: A multicentre open-label, cross-sectional and prospective study among four Spanish hospitals was performed. Adult RA, PsA, axSpA patients treated for at least 6 months with the CZP syringe or pen were recruited. At the first visit, patients completed Pre-AVA® questionnaire. Patients were instructed on proper administration of CZP by AVA®. After 2 and 6 months of CZP self-injections using the AVA®, patient experience, adherence, preference and safety of each administration was assessed using post-AVA® questionnaire. RESULTS AND DISCUSSION: Thirty four patients were included (28 women). All patients self-administered CZP AVA® the full dose of CZP was injected. Patients reported >90% adherence to CZP AVA® assessed with the injection log. Pain at the injection site was reduced after switching to AVA®. Twenty nine patients preferred CZP AVA® and five patients preferred the CZP pen. No safety-related findings related to AVA® CZP administration were identified. WHAT IS NEW AND CONCLUSION: The AVA® is an advantageous delivery option for CZP in patients with RA, PsA, axSpA.

Successful use of golimumab in a patient with ulcerative colitis refractory to infliximab and adalimumab.

OBJECTIVE: To report a case of successful use of golimumab (GLB) in a patient with ulcerative colitis (UC) refractory to infliximab (IFX) and adalimumab (ADA). CASE SUMMARY: A 60-year-old man was diagnosed with left UC and was given azathioprine 2.5 mg/kg to control UC symptoms and decrease corticosteroid patient dependence. Four years later, he developed adverse reaction to azathioprine and began treatment with mercaptopurine 1.5 mg/kg/day. Despite this treatment, he developed a severe relapse (Truelove-Witts modified: 15 points). Treatment with IFX 5 mg/kg at weeks 0, 2, 6, and every 8 weeks was started. After 1 year in clinical remission, the patient developed an infusion reaction to IFX, and IFX was suspended. The patient started treatment with ADA 40 mg every other week. After 2 years in clinical remission, ADA was suspended. 20 months after ADA discontinuation, the patient developed an acute episode of UC with a Truelove-Witts modified score of 16 points. ADA plus corticosteroid therapy was restarted. Despite these treatments, the patient's clinical condition did not improved. ADA 40 mg per week was started with not clinical improvement and with corticosteroid dependence after 4 months of ADA intensive therapy. The patient denied surgery, and cyclosporine was discarded because of its inability to be used as a maintenance drug. The patient started GLB with an induction dosage regimen of 200 mg subcutaneous at week 0, followed by 100 mg at week 2, and then maintenance therapy with 100 mg every 4 weeks (patient's weight = 84 kg), combined with mercaptopurine and corticosteroids. After 6 weeks of treatment, the patient achieved clinical remission, with just three non-bleeding stools per day, without stomach ache, apyretic, and no urgency or tenesmus rectal symptoms. One year later, the patient continued to be asymptomatic with a Truelove-Witts modified score of 2 points, corticoid-free treatment, and a complete clinical and endoscopic remission and normal calprotectin levels (< 15 µg/g). We decided to suspend mercaptopurine in order to avoid side effects derived from the combined treatment. After 1 year on GLB therapy, the patient continued in clinical remission. CONCLUSIONS: Based on our case, GLB could be selected as an effective approach for patients with UC refractory to IFX and ADA. However, further studies need to be performed to evaluate the efficacy of GLB therapy as a rescue treatment.

Successful use of infliximab and tacrolimus combination therapy in a patient with ulcerative colitis refractory to infliximab dose intensification plus azathioprine.

OBJECTIVE: To report of a case successful use of infliximab (IFX) and tacrolimus (TAC) in a patient with ulcerative colitis (UC). CASE SUMMARY: A 22-year-old woman diagnosed with UC started treatment with azathioprine 2.5 mg/kg. After 3 years of therapy, she developed a severe relapse. A colonoscopy was performed showing diffuse continuous mucosal disease and multiple erosions (< 5 mm) with no signs of spontaneous bleeding. Treatment with IFX 5 mg/kg at weeks 0, 2, and 6 was started. After IFX induction, she remained with symptoms: six stools per day, as well as presenting bloody diarrhea, tenesmus, and no abdominal pain. An IFX dose intensification of 5 mg/kg every 6 weeks was prescribed. After 6 months of azathioprine plus IFX therapy, patient's clinical condition was improved: 3 - 4 stools per day, 20% of bloody diarrhea, tenesmus, and no abdominal pain. Her Mayo endoscopic subscore was 6.3 months later, and a severe relapse of ulcerative colitis was presented. The patient refused a surgical treatment. Azathioprine 2.5 mg/kg/day was suspended and TAC 0.2 mg/kg/day (12 mg/day) as a compassionate use was added to IFX dose intensification of 10 mg/kg every 8 weeks and mesalamine 800 mg 3 times daily. After the first month of combined therapy, the patient's clinical condition improved with no bloody stools and abdominal pain. After 6 months of combination therapy, the patient was in remission, with two stools per day, no tenesmus and no abdominal pain. Due to the patient's clinical remission, IFX was suspended. Tacrolimus was continued on 10 mg/day. After 6 months of TAC monotherapy, the patient continued without symptoms (1 - 2 normal stools per day). CONCLUSIONS: Based on our case, the combination therapy of IFX and TAC could be selected as an effective approach for the patients with UC refractory to IFX dose intensification plus AZA. However, further studies need to be performed to evaluate the efficacy of this combination therapy.

Successful use of infliximab and tacrolimus in a patient with Crohn's disease.

OBJECTIVE: To report a case of successful use of infliximab (IFX) and tacrolimus (TAC) in a patient with Crohn's disease (CD). CASE SUMMARY: A 42-year-old man with no significant previous medical history was referred to our emergency department because of a 3-month history of weight loss, severe abdominal pain, and bloody diarrhea. His Harvey Bradshaw Index was 39. Ileocolonoscopic revealed severe Crohn colitis. Treatment with IFX 5 mg/kg, azathioprine 2.5 mg/kg/day and corticosteroids was started. After a second IFX infusion, he remained with symptoms with a Harvey Bradshaw Index of 17. An IFX dose intensification of 10 mg/kg every 8 weeks was prescribed. After 16 weeks, a new colonoscopic examination revealed multiple deep ulcerations in sigma and rectum. IFX was intensified to 10 mg/kg every 6 weeks. After 4 doses of IFX intensified dose, the patient's clinical condition was not improved, with a Harvey Bradshaw Index of 10. Azathioprine (AZA) 2.5 mg/kg/day was suspended. Tacrolimus 0.2 mg/kg/day as a compassionate use was added to IFX 10 mg/kg every 6 weeks. After 6 months of combination therapy, the patient was in clinical remission. His Harvey Bradshaw Index was 3. After 1 year on combination IFX and TAC therapy, the patient continued in clinical remission. CONCLUSIONS: This case documents that the combination therapy of IFX and TAC could be selected as an effective approach for patients with CD refractory to IFX dose-intensification plus AZA. However, further studies need to be performed to evaluate the efficacy of this combination therapy.

Dealing with the high cost of biological therapies: developing and implementing a biological therapy prioritization protocol for ankylosing spondylitis patients in a tertiary hospital.

OBJECTIVE: In January 2011, a biological therapies commission was created in our hospital to fully address the management of biological drugs. A biological therapy prioritization protocol was developed for ankylosing spondylitis (AS) patients. Here, we describe it and report on its economic impact to illustrate how we are optimizing the use of these expensive new drugs. METHODS: The biological therapies commission established several procedures for the rational use of biological drugs such as cost-efficiency therapeutic protocols, pharmacovigilance, and therapeutic drug monitoring programs. The AS protocol was based on clinical and economic aspects. We estimated the economic impact of the protocol by comparing the cost of treating AS patients with biological drugs in the pre-commission (2009 - 2010) vs. post-commission period (2011 - 2013). AS patients treated with adalimumab (ADA), etanercept (ETN) or infliximab (IFX) for at least 6 months in the 2009 - 2013 period were included. RESULTS: 107 patients were included. In the pre-commission period, total expenses increased by +30,944 Euro (+4%). After protocol implementation, total expenses decreased by 11,441 Euro (-1%) during 2011, and by an additional 36,781 Euro (-4%) and 53,872 Euro (-8%) in 2012 and 2013, respectively. In the 2010 - 2013 period the cost of biological therapy per patient-year decreased by 869 €, suggesting the positive effects of the biological therapy prioritization protocol instauration. CONCLUSION: We describe the establishment of a multidisciplinary biological therapy commission to optimize the use of biological therapies. We illustrate its work in developing a protocol for the management of AS patients with such therapies. We show that after 3-years of implementation, the biological therapy prioritization protocol allowed us to steadily decrease the direct cost of biological drug therapies per patient, up to 869 Euro.

Effectiveness of infliximab, adalimumab and golimumab for non-infectious refractory uveitis in adults.

AIM: To discuss the available data regarding the off-label uses of anti-TNF agents in non-infectious uveitis. DATA SOURCE: A literature search was performed in Medline through PubMed from January 2001 to January 2014. STUDY SELECTION AND DATA EXTRACTION: English-language articles about uveitis treatment with anti-TNF drugs in adult patients were reviewed. DATA SYNTHESIS: The use of anti-TNF-Î ± drugs for treatment of several refractory manifestations of refractory uveitis in adult patients is increasing. However, due to the lack of evidence from randomized controlled trials, the use of anti-TNF in uveitis remains “off-label” in most countries. There is no trial-based evidence to support it except for the experience provided by cases and case series. This experience, which is continuously increasing, has yielded encouraging results. Anti-TNF-Î ± drugs, such as infliximab, adalimumab, and golimumab, are reasonably effective for controlling ocular inflammation and sparing patients corticosteroid treatment in non-infectious refractory uveitis. Approximately 80% of patients on infliximab, adalimumab, or golimumab were able to achieve sustained control of inflammation by 6 months. CONCLUSION: Anti-TNF-Î ± therapy is effective in inducing clinical remission for refractory uveitis, with a relatively low rate of treatment-ending adverse events. However, randomized and controlled trials are required to adequately assess the maintained clinical efficacy and safety profile in the long term of anti-TNF agents for non-infectious refractory uveitis.

Patient preference after switching guselkumab from prefilled syringe to an autoinjection pen in psoriasis and psoriatic arthritis patients.

BACKGROUND: We assessed pain, acceptability, patient preference, and tolerability of patients with psoriasis and psoriatic arthritis after switching guselkumab from a prefilled syringe to One-Press autoinjector pen. METHODS: Patients with psoriasis and psoriatic arthritis treated for at least 6 months with guselkumab syringe were recruited from Jan 2019 to Dec 2022. Gender, age, diagnosis, self-administration, and pain perception of guselkumab prefilled syringe were recorded. At the first visit, patients completed a post-auto-injection syringe questionnaire before starting auto-injection pen administration. After 2 and 6 months of guselkumab self-injection using the One-Press autoinjector pen, patient experience, adherence, preference, pain, and safety of each administration were assessed using post-guselkumab by One-Press autoinjector pen questionnaire. RESULTS: 40 patients [psoriasis n=34, psoriatic arthritis n=6] were included. All patients self-administered guselkumab by One-Press autoinjector pen. Pain at the injection site was significantly reduced with the use of the One-Press autoinjector pen. All patients considered that using One-Press autoinjector pen was easier than the syringe, 98% chose the pen as their preferred delivery system. CONCLUSION: The One-Press autoinjector pen for guselkumab administration is presented as a preferred option, with a high satisfaction and less painful compared to the administration of guselkumab in a prefilled syringe.

Biosimilars and access to biologic therapy in immune-mediated diseases.

BACKGROUND: The rise of biologic agents has been a major breakthrough in treating immune-mediated inflammatory diseases (IMIDs). However, their high cost underscores the need for strategies to optimize treatment efficiency. Biosimilars offer cost-effective alternatives to biologics. This study aimed to assess biosimilar drug availability's impact on biologic therapy access for IMIDs. RESEARCH DESIGN AND METHODS: A retrospective observational study in 15 Spanish hospitals analyzed IMID patients (arthropathies, inflammatory bowel disease and psoriasis) initiating biologic therapy with originator or biosimilar drugs (infliximab, etanercept, adalimumab). Time to availability and initiation of biologic therapy were assessed. RESULTS: 267 patients were included, with 58.4% starting on biosimilars. The mean time to availability of the biologic drugs in the hospitals was 15.9 ± 6.7 months, (20.0 ± 12.4 for originator and 11.8 ± 5.2 for biosimilars). Mean time to biologic treatment was 7.7 ± 9.0 years (8.6 ± 8.9 for originators and 7.0 ± 9.0 for biosimilars). Showing statistically significant differences among conditions. CONCLUSION: The emergence of biosimilar drugs has enhanced market competition and accelerated their adoption into hospitals' therapeutic regimens over original reference drugs. This has significantly improved access to biologic therapy for patients with IMIDs, evidenced by a notable 1.6-year reduction in access time for biosimilar drugs.