[Autologous hematopoietic stem cell transplantation in a patient with refractory Crohn's disease].

Crohn's disease is a chronic condition for which sometimes there is no effective medical or surgical treatment. Autologous hematopoietic stem cell transplantation may be a therapeutic option for these patients to restore immune tolerance. Consequently, this may lead to remission of the disease or decrease its activity, making drugs that have previously failed be effective. Due to the safety profile of the procedure and the fact that it is a non-curative treatment, patient selection must be rigorous. We report our experience with the first patient selected in our centre for autologous hematopoietic stem cell transplantation: 27 years old male with Crohn's disease (A1L3B1p) refractory to multiple lines of medical treatment and not a candidate for surgical treatment. Two years after the transplantation, the patient remains asymptomatic.

Effectiveness and Safety of Ustekinumab in Elderly Patients with Crohn's Disease: Real World Evidence From the ENEIDA Registry.

BACKGROUND AND AIMS: Clinical trials and real-life studies with ustekinumab in Crohn's disease [CD] have revealed a good efficacy and safety profile. However, these data are scarcely available in elderly patients. Therefore, we aim to assess the effectiveness and safety of ustekinumab in elderly patients with CD. METHODS: Elderly patients [>60 years old] from the prospectively maintained ENEIDA registry treated with ustekinumab due to CD were included. Every patient was matched with two controls under 60 years of age, according to anti-tumour necrosis factor use and smoking habit. Values for the Harvey-Bradshaw Index [HBI], endoscopic activity, C-reactive protein [CRP] and faecal calprotectin [FC] were recorded at baseline and at weeks 16, 32 and 54. RESULTS: In total, 648 patients were included, 212 of whom were elderly. Effectiveness was similar between young and elderly patients during the follow-up. Steroid-free remission was similar at week 16 [54.6 vs 51.4%, p = 0.20], 32 [53.0% vs 54.5%, p = 0.26] and 54 [57.8% vs 51.1%, p = 0.21]. Persistence of ustekinumab as maintenance therapy was similar in both age groups [log-rank test; p = 0.91]. There was no difference in the rate of adverse effects [14.2% vs 11.2%, p = 0.350], including severe infections [7.1% vs 7.3%, p = 1.00], except for the occurrence of de novo neoplasms, which was higher in older patients [0.7% vs 4.3%, p = 0.003]. CONCLUSIONS: Ustekinumab is as effective in elderly patients with CD as it is in non-elderly patients. The safety profile also seems to be similar except for a higher rate of de novo neoplasms, probably related to the age of the elderly patients.

Ustekinumab in Crohn's disease: effectiveness and safety in clinical practice. / Ustekinumab en enfermedad de Crohn: efectividad y seguridad en práctica clínica.

INTRODUCTION: Ustekinumab (UST) is a monoclonal antibody against IL-12/23 approved in Spain (2017) to treat moderate / severe Crohn's disease. OBJECTIVE: To evaluate the effectiveness and safety in real clinical practice in patients treated with UST in our center. METHODS: This is a prospective observational study including patients who started UST from 08/01/2017 to 02/28/2019 with follow-up up to that date. We analyze response and remission in weeks 16, 24 and 52, using "Crohn's Disease Activity Index" (response if 100 point decrease and remission if <150) and Physician's Global Assessment. RESULTS: We included 61 patients with a median duration of Crohn's disease of 14,6 years (0-36). The 83,6% of patients without steroids and 73,8% without associated immunosuppressors. Previously all patients had received anti-TNF and 14,8%, in addition, vedolizumab. We observed a good correlation between Crohn's Disease Activity Index and Physician's Global Assessment (r = 0,89, p <.001). In week 16 (n = 45) 75,6% response (57,8% remission), in week 24 (n = 35) 69,9% response (45,7% remission) and in week 52 (n = 12) 75% response (58.3% remission). There were no statistically significant differences in the response/remission rates at week 16 or 24 depending on the reason for the onset of UST or the number of previous biologics. In 2 patients it was withdrawn due to toxicity (arthralgia / myalgia). CONCLUSION: UST is an effective and safe treatment in real clinical practice with high rates of clinical remission at week 16, 24 and 52 regardless of the order of biological used and the reason for starting UST.

Effectiveness of adalimumab for the treatment of ulcerative colitis in clinical practice: comparison between anti-tumour necrosis factor-naïve and non-naïve patients.

BACKGROUND: Ulcerative colitis (UC) treatment is focused to achieve mucosal healing, avoiding disease progression. The study aimed to evaluate the real-world effectiveness of adalimumab (ADA) in UC and to identify predictors of remission to ADA. METHODS: This cohort study used data from the ENEIDA registry. Clinical response, clinical remission, endoscopic remission, adverse events (AE), colectomy, and hospitalisations were evaluated; baseline characteristics and biological parameters were compared to determine predictors of response. RESULTS: We included 263 patients (87 naïve and 176 previously exposed to anti-tumour necrosis factor alpha, TNF). After 12 weeks, clinical response, clinical remission, and endoscopic remission rates were 51, 26, and 14 %, respectively. The naïve group demonstrated better response to treatment than the anti-TNF-exposed group at short-term. Clinical and endoscopic remission within 1 year of treatment was better in the naïve group (65 vs. 49 and 50 vs. 35 %, respectively). The rates of AE, dose-escalation, hospitalisations, and colectomy during the first year were higher in anti-TNF-exposed patients (40, 43, and 27 % vs. 26, 21, and 11 %, respectively). Patients with primary failure and intolerance to the first anti-TNF and severe disease were associated with worse clinical response. Primary non-response to prior anti-TNF treatment and severe disease were predictive of poorer clinical remission. Low levels of C-reactive protein (CRP) and faecal calprotectin (FC) at baseline were predictors of clinical remission. CONCLUSIONS: In clinical practice, ADA was effective in UC, especially in anti-TNF naïve patients. FC and CRP could be predictors of treatment effectiveness.