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Haematopoietic stem cell transplantation (HSCT) has been proposed for refractory autoimmune diseases, including systemic sclerosis (SSc). A sequential Bayesian phase I-II clinical trial was conducted in SSc patients to assess the feasibility, the tolerance and the efficacy of autologous HSCT. Peripheral blood stem cells (PBSC) were collected using(More)
We review the rationale behind the statistical design of dose-finding studies as used in phase I and phase I/II clinical trials. We underline what the objectives of such dose-finding studies should be and why the widely used standard design fails to meet any of these objectives. The standard design is a "memoryless" design and we discuss how this impacts on(More)
PURPOSE It is generally accepted that early visual deprivation from monocular enucleation (ME; the surgical removal of one eye) results in intact spatial vision. Yet, motion perception studies in this population yield inconsistent findings. Here, we investigated speed and luminance contrast perception in a group of ME individuals. METHODS Twelve ME(More)
The aim of a phase I oncology trial is to identify a dose with an acceptable safety profile. Most phase I designs use the dose-limiting toxicity, a binary endpoint, to assess the unacceptable level of toxicity. The dose-limiting toxicity might be incomplete for investigating molecularly targeted therapies as much useful toxicity information is discarded. In(More)
The continual reassessment method (CRM) provides a Bayesian estimation of the maximum tolerated dose (MTD) in phase I clinical trials and is also used to estimate the minimal efficacy dose (MED) in phase II clinical trials. In this paper we propose Bayesian stopping rules for the CRM, based on either posterior or predictive probability distributions that(More)
OBJECTIVE Systemic sclerosis (SSc) is a generalised autoimmune disease, causing morbidity and a reduced life expectancy, especially in patients with rapidly progressive diffuse cutaneous SSc. As no proven treatment exists, autologous haematopoietic stem cell transplantation (HSCT) is employed as a new therapeutic strategy in patients with a poor prognosis.(More)
Oligodeoxynucleotides containing CpG motifs (CpG ODNs) display a strong immunostimulating activity and drive the immune response toward the Th1 (T helper type 1) phenotype. These ODNs have shown promising efficacy in preclinical studies when injected locally in several cancer models. We conducted a phase 1 trial to define the safety profile of CpG-28, a(More)
OBJECTIVE Using high resolution computed tomography (HRCT), to assess the lung involvement outcome after autologous hematopoietic stem cell transplant (AHSCT) in patients with scleroderma (systemic sclerosis, SSc). METHODS HCRT scans prospectively performed before (n = 9 patients) and after (n = 47) AHSCT were blindly reviewed by 2 independent(More)
OBJECTIVE The aim of this study was to report our experience in the management of late morbidity after colonic interposition for caustic injury and to assess the influence of coloplasty dysfunction on patient outcome. SUMMARY BACKGROUND DATA Reports on coloplasty dysfunction after colon interposition for corrosive esophageal injuries are scarce in the(More)
OBJECTIVE To determine the minimal effective dose (MED) of intravenous midazolam, required for appropriate sedation in 95% of patients, 1 h after drug administration. METHODS A double-blind dose-finding study using the continual reassessment method, a Bayesian sequential design. Twenty-three newborn infants hospitalized in intensive care unit(More)