Real‐world experience on off‐label use of imiquimod 5% as an adjuvant therapy after surgery or as a monotherapy for lentigo maligna

Abstract We report off-label use patterns for medications repurposed for COVID-19 at 318 U.S. hospitals. Inpatient hydroxychloroquine use declined by 80%, while corticosteroids and tocilizumab were initiated two days earlier in May versus March 2020. Two-thirds of ventilated COVID-19 patients were already receiving corticosteroids during March-May, resembling pre-COVID use in mechanically ventilated influenza patients.

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Off-label use of intravenous thrombolysis for acute ischemic stroke: a critical appraisal of randomized and real-world evidence

Therapeutic Advances in Neurological Disorders ◽

10.1177/1756286421997368 ◽

2021 ◽

Vol 14 ◽

pp. 175628642199736

Author(s):

Georgios Tsivgoulis ◽

Odysseas Kargiotis ◽

Gianmarco De Marchis ◽

Martin Kohrmann ◽

Else Charlotte Sandset ◽

...

Keyword(s):

Ischemic Stroke ◽

Acute Ischemic Stroke ◽

Real World ◽

Intravenous Thrombolysis ◽

Therapeutic Window ◽

Off Label Use ◽

Exclusion Criteria ◽

Off Label ◽

Thrombolytic Agents ◽

Label Use

Intravenous thrombolysis (IVT) represents the only systemic reperfusion therapy able to reverse neurological deficit in patients with acute ischemic stroke (AIS). Despite its effectiveness in patients with or without large vessel occlusion, it can be offered only to a minority of them, because of the short therapeutic window and additional contraindications derived from stringent but arbitrary inclusion and exclusion criteria used in landmark randomized controlled clinical trials. Many absolute or relative contraindications lead to disparities between the official drug label and guidelines or expert recommendations. Based on recent advances in neuroimaging and evidence from cohort studies, off-label use of IVT is increasingly incorporated into the daily practice of many stroke centers. They relate to extension of therapeutic time windows, and expansion of indications in co-existing conditions originally listed in exclusion criteria, such as use of alternative thrombolytic agents, pre-treatment with antiplatelets, anticoagulants or low molecular weight heparins. In this narrative review, we summarize recent randomized and real-world data on the safety and efficacy of off-label use of IVT for AIS. We also make some practical recommendations to stroke physicians regarding the off-label use of thrombolytic agents in complex and uncommon presentations of AIS or other conditions mimicking acute cerebral ischemia. Finally, we provide guidance on the risks and benefits of IVT in numerous AIS subgroups, where equipoise exists and guidelines and treatment practices vary.

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Off-Label Use of Venetoclax in Patients With Acute Myeloid Leukemia: Single Center Experience and Data From Pharmacovigilance Database

Frontiers in Pharmacology ◽

10.3389/fphar.2021.748766 ◽

2021 ◽

Vol 12 ◽

Author(s):

Lucia Gozzo ◽

Calogero Vetro ◽

Serena Brancati ◽

Laura Longo ◽

Daniela Cristina Vitale ◽

...

Keyword(s):

Acute Myeloid Leukemia ◽

Febrile Neutropenia ◽

Real World ◽

Myeloid Leukemia ◽

Response Assessment ◽

Off Label Use ◽

Real World Data ◽

Off Label ◽

Acute Myeloid ◽

Label Use

The potent oral inhibitor of BCL2, venetoclax (VEN), used to treat adults with chronic lymphocytic leukaemia, has been approved in US for the treatment of naïve patients with acute myeloid leukemia (AML) unfit for intensive chemotherapy and recently in Europe, too. However, the drug has been used for years in combination with hypomethylating agents (HMAs) in patients not eligible to other treatment option, according to the so-called off-label use. We collected real-world data about patients treated with VEN + HMAs in the context of a pharmacovigilance project focused on the evaluation of the safety and effectiveness of drugs used for unapproved indication in Italian hospitals. From March to December 2020, 24 patients started treatment with VEN combined with HMAs. 21 patients have been assessed for response. Eleven (52%) patients reached complete remission (CR), and three patients (14%) CR with partial hematological recovery (CRh), with a median duration of response of 4.5 months (range 0.5–12.5). 19 patients experienced at least 1 adverse drug reaction (ADR), mostly serious, including 3 deaths (9% of ADRs; 12.5% of patients) in febrile neutropenia. Hematological toxicities and infections (cytopenia, neutropenia, febrile neutropenia, sepsis), were the most reported ADRs (84.4%). In general, neutropenic fever occurred more frequently in patients treated with decitabine (7 out of 9, 78%) compared to azacitidine (5 out of 15, 33%; p = 0.03), whereas response assessment did not differ based on used HMA (p = 0.1). These results confirm the benefit-risk profile of VEN in a real-world setting of patients with no adequate therapeutic options.

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