Progressive erythrocytosis under lenvatinib treatment in patients with advanced hepatocellular carcinoma.

Purpose: This manuscript reports on the occurrence of early and frequent erythrocytosis in advanced hepatocellular carcinoma (HCC) patients treated with lenvatinib.

Methods: A cohort of 23 patients with advanced HCC, treated with this antiangiogenic drug for at least one month, was retrospectively analyzed.

Results: These patients (82.

Agreement between a regional pharmacovigilance centre and an adjudication committee regarding adverse drug reactions on a cohort of hospitalised children.

Background And Purpose: The EREMI project was set up to collect data on adverse drug reactions (ADRs) occurring due to off-label and/or unlicensed drugs prescribed to hospitalised children in France. These events were evaluated by a regional pharmacovigilance centre (RPC) and an adjudication committee (AC). The aim of this study was to assess the agreement between these two different entities on their evaluation of ADRs.

Characteristics of insulinopenic and non insulinopenic diabetes related to immune checkpoint inhibitors: A French pharmacovigilance study.

Introduction: Immune checkpoint inhibitor-induced diabetes mellitus (ICI-DM) is an immune-related adverse drug reaction (irADR). Hyperglycemia can be linked to endogenous insulin deficiency with ketoacidosis or associated with preserved beta-cell function.

Objectives: We aimed to identify the characteristics of both types of ICI-DM (type 1 and type 2 DM), to improve our understanding of this irADR and its management.

Severe immune-related hepatitis induced by immune checkpoint inhibitors: Clinical features and management proposal.

Background: Immune-related hepatitis (IRH) occurs in 1 to 18% of immune checkpoint inhibitor (ICI)-treated patients. Steroids are usually recommended for grade≥3 IRH, but their impact on IRH resolution and patient survival remains unclear.

Methods: We retrospectively analyzed a prospective cohort of 339 patients treated at Saint-Louis Hospital (Paris, France) with ICIs for advanced melanoma.

Baraitser-Winter cerebrofrontofacial syndrome: delineation of the spectrum in 42 cases.

Baraitser-Winter, Fryns-Aftimos and cerebrofrontofacial syndrome types 1 and 3 have recently been associated with heterozygous gain-of-function mutations in one of the two ubiquitous cytoplasmic actin-encoding genes ACTB and ACTG1 that encode β- and γ-actins. We present detailed phenotypic descriptions and neuroimaging on 36 patients analyzed by our group and six cases from the literature with a molecularly proven actinopathy (9 ACTG1 and 33 ACTB). The major clinical anomalies are striking dysmorphic facial features with hypertelorism, broad nose with large tip and prominent root, congenital non-myopathic ptosis, ridged metopic suture and arched eyebrows.

The use of dabigatran in elderly patients.

Recent approval by the US Food and Drug Administration (FDA) of dabigatran etexilate, an oral direct thrombin inhibitor, for the prevention of stroke in patients with atrial fibrillation will likely extend its administration in elderly patients. The risk of major overdosage of dabigatran etexilate in this population is, however, much increased owing to frequent renal function impairment, low body weight, drug interactions that cannot be detected with a routine coagulation test, and no antagonist available. We report herein 2 clinical cases, including 1 fatal case, illustrating our concern regarding the risk of bleeding events in elderly patients.

Interest in an original methodology to define the optimal dosage of interferon-alpha-2a in metastatic melanoma patients.

Interferon-α-2a (IFNa) has proven antitumor activity in a variety of neoplastic diseases, but no clear modality of administration has been validated. The aim of our study was to estimate the optimal dose of continuous subcutaneous administration of IFNa in stage IV metastatic melanoma patients. An innovative dose-finding approach, combining phase I and phase II trials, was planned to evaluate the toxicity and efficacy of four dose levels of IFNa (3, 6, 9, and 12 MIU/day).

[Treatment of erectile dysfunction by phosphodiesterase-5 inhibitors and nonarteritic anterior ischemic optic neuropathy (NOIAN)].

Introduction: Following the notification of forty cases of varying degrees of vision loss in patients using phosphodiesterase-5 (PDE-5) inhibitors, the FDA (Food and Drug Administration) examined the possible link between these treatments of erectile dysfunction and NOIAN (nonarteritic anterior ischemic optic neuropathy). Following this investigation, the FDA requested modification of the summary of product characteristics (SPC) for this therapeutic category. The authors review this problem, especially in France.

Child overdose: the value of the French pharmacovigilance causality assessment method.

Background: In France, the regional pharmacovigilance centres manage drug overdose as adverse drug reactions (ADRs) using the French ADR causality assessment method, and some poison control centres (e.g. in Paris) do likewise for the most serious cases.