Off-Label Use and Safety of Drug Use in Vascular Anomalies

Dermatology ◽  
2021 ◽  
pp. 1-9
Author(s):  
Niina Kleiber ◽  
Laurence Gariépy-Assal ◽  
Jérôme Coulombe ◽  
Simon Marcoux ◽  
Sandrine Essouri ◽  
...  
Keyword(s):  
Drug Use ◽  
Oral Solution ◽  
Vascular Anomalies ◽  
Significant Safety ◽  
Safety Issues ◽  
Off Label ◽  
Safety Concerns ◽  
Increased Risk ◽  
Threatening Condition ◽  
Life Threatening

<b><i>Background:</i></b> Off-label drug use is associated with an increased risk of adverse drug reactions. It is common in pediatrics and in rare diseases, which are two characteristics applying to vascular anomalies (VA). <b><i>Objectives:</i></b> The aim of this work was to quantify off-label drug use in VA and assess its safety. <b><i>Methods:</i></b> A review was conducted to extract a list of drugs used in VA management. A drug was considered to have significant safety concerns if a black box warning was present or if a serious adverse drug reaction (SADR) was reported in at least 1% of the patients (SADR is defined as a noxious and unintended response to a drug that occurs at any dose and results in hospitalization, prolongation of existing hospitalization, congenital malformation, persistent or significant disability or incapacity, life-threatening condition, or death). The labelling status and safety of each drug was assessed based on the product monograph, Micromedex, and the FDA data. <b><i>Results:</i></b> We found that 98.9% of the inventoried drugs were used off-label or unlicensed for VA management. Only the oral solution of propranolol hydrochloride (Hemangeol®) for the treatment of infantile hemangiomas is approved. Significant safety issues concerned 73% of the drugs and were more frequent among systemic than locally delivered drugs. <b><i>Conclusions:</i></b> Off-label drug use in VA is the rule and not the exception. Significant safety concerns are common. It is necessary to carefully weigh risk and benefits for every patient when using systemic and local treatments carrying safety concerns. Patients should be openly informed and involved in the decision-making process.

73 Off-label use and safety of drug use in vascular anomalies

2021 ◽  
Vol 26 (Supplement_1) ◽  
pp. e53-e55
Author(s):  
Laurence Gariépy-Assal ◽  
Simon Marcoux ◽  
Jerome Coulombe ◽  
Julie Powell ◽  
Sandrine Essouri ◽  
...  
Keyword(s):  
Drug Use ◽  
Vascular Anomalies ◽  
Abnormal Development ◽  
Off Label Use ◽  
Significant Safety ◽  
Off Label ◽  
Safety Concerns ◽  
Increased Risk ◽  
Life Threatening ◽  
Label Use

Abstract Primary Subject area Clinical Pharmacology and Toxicology Background Vascular anomalies (VA) represent a heterogeneous group of disorders associated with an abnormal development and proliferation of blood and/or lymphatic vessels displaying variable clinical presentations and severity. Infantile hemangiomas, venous, and lymphatic malformations, for example, are commonly encountered in children. Other, less frequent diagnostics include Klippel-Trenaunay syndrome and PIK-3CA-related overgrowth spectrum (PROS). Severe phenotypes can alter organ function and/or lead to pain and chronic functional impairment, and are associated with significant morbidity and mortality. Management includes surgical, interventional radiology, and pharmacologic modalities. Drugs are administered by systemic (e.g., oral, intravenous) or local (topical, intralesional) routes, or by sclerotherapy (endovascular or percutaneous venous, lymphatic, or arterial injection). Off-label drug use is common in pediatrics and in rare diseases, two characteristics applying to vascular anomalies (VA). Off-label use is associated with an increased risk of adverse drug reactions. Objectives To quantify off-label drug use in VA and assess its safety. Design/Methods A guidelines search was conducted to extract a list of drugs used in VA management. The labelling status and safety of each drug was assessed based on the product monograph, Micromedex, and the FDA data. A drug was considered to have significant safety concerns if a black box warning (the FDA’s most stringent warning dedicated to serious or life-threatening risks) or if a serious adverse drug reaction was reported in at least 1% of the patients (leading to hospitalization, congenital malformation, persistent or significant disability or incapacity, life-threatening condition, or death). Results Among 87 drugs, 13 were unlicensed and 73 off-label. Figure 1 describes the reason for considering the 73 drugs off-label. Among 74 licensed drugs, only the oral solution of propranolol hydrochloride (Hemangeol®) for the treatment of infantile hemangiomas (IH) is approved. 98.9% of the drugs are used off-label or unlicensed. Except infantile hemangioma, all other VA are exclusively treated with off-label drugs. Significant safety issues concerned 73% of the drugs and were more frequent among systemic than locally delivered drugs (Figure 2). Conclusion This first study determining the rate of off-label drug use in vascular anomalies shows that off-label drug use in VA is the rule and not the exception. Significant safety concerns are common. It is needed to carefully weigh risk and benefits for every patient when using systemic and local treatments carrying safety concerns. Patients and families should be openly informed and involved in the decision-making process.

scholarly journals P13 Serious adverse reactions and off label and unlicensed drug use in children – decade of pharmacovigilance study in Serbia

2019 ◽  
Vol 104 (6) ◽  
pp. e22.2-e22
Author(s):  
M Bajcetic ◽  
J Joksimovic
Keyword(s):  
Drug Use ◽  
Adverse Reactions ◽  
Drug Formulation ◽  
System Organ Class ◽  
List Type ◽  
Age Group ◽  
Sources Of Information ◽  
Antimicrobial Drugs ◽  
Off Label ◽  
Life Threatening

BackgroundOff label (OL) and unlicensed (UL) drug use in children is a widespread global problem. Previous study showed that only 66% of all available drugs for children is with licence in Serbia.1 Data on safety of medicines in children remain lacking, so the key intervention for the effective use of medicine is safety monitoring. Therefore, the aim of this study is to evaluate safety implication of OL and UL drug use in children up to 12 years old.MethodWe conducted a retrospective study based on reports of suspected adverse reactions (ADRs) collected from 2008. to 2018. by Medicines and Medical Devices Agency of Serbia, using the Medical Dictionary for Regulatory Activities and organized by System Organ Class. Sources of information about medicines including vaccines (license, drug formulation, etc.) are the Summary of Product Characteristics and Serbia´s official drug registry.ResultsWithin 10 years, we observed 1595 ADRs. Vaccines, antineoplastic and antimicrobial drugs were the most frequently pharmacotherapeutic subgroups involved. Out of total number of observed ADRs, 433 (28%) were serious; 189 of them led to hospitalization, 31 to life threatening conditions and 7 were fatal. More than a half (63%) of serious ADRs were detected in children for the age group of 28 days - 23 months, followed by the age group of 2 to 11 years (34%) and finally by age group of 0 to 27 days (3%). Serious ADRs were detected in boys (55%) as well as in girls (45%). Out of total number of registered only 3% (46) of ADRs were associated with off-label use; 18 of them were serious, 7 led to life threatening conditions and 3 were fatal.ConclusionThis research provided new insight on the factors such as OL and UL use, that might increase the risk of serious ADRs in children.ReferencesBo&zbreve;i&cacute; B, Stupar S, Stupar D, Babi&cacute; U, Bajčeti&cacute; M. Availability of pediatric-evaluated formulations in Serbia. Indian J Pharmacol 2017 Mar-Apr;49(2):189–193Disclosure(s)Nothing to disclose

scholarly journals Aortic Dissection in a Pregnant Patient without Other Risk Factors

2019 ◽  
Vol 2019 ◽  
pp. 1-3
Author(s):  
Priya V. Patel ◽  
Raanan Alter ◽  
Recia Frenn ◽  
Thaddeus P. Waters
Keyword(s):  
Risk Factors ◽  
Aortic Dissection ◽  
Aortic Rupture ◽  
Treatment Options ◽  
Pregnant Patient ◽  
Physical Exam ◽  
Vascular Events ◽  
Increased Risk ◽  
Threatening Condition ◽  
Life Threatening

Background. An aortic dissection is a life-threatening condition in which the intima of the artery tears causing separation of the intima and media. Pregnancy places women at a significantly increased risk of common vascular events including venous thromboembolism, myocardial infarction, and stroke, while also increasing the risk of rarer vascular events such as aortic dissection and aortic rupture. Case. A 30-year-old previously healthy multiparous woman presenting at 36 weeks of pregnancy with a Type A aortic dissection. She underwent a combined emergent cesarean delivery followed by repair of her aortic root. Conclusions. Aortic dissection should be high on the differential for pregnant patients presenting with the characteristic complaints and physical exam findings given the high mortality rate associated with this vascular event. Teaching Points. (1) This report reviews the characteristic presentation, risk factors, and physical exam findings in a patient with an aortic dissection. (2) The report includes treatment options for pregnant patients based on the classification of the dissection.

Off-label drug use and the risk of medication errors in critically ill neonates: A conceptual pilot study

2021 ◽  
pp. 1-15
Author(s):  
Kannan Sridharan ◽  
Muna Al Jufairi ◽  
Eman Al Ansari
Keyword(s):  
Intensive Care Unit ◽  
Intensive Care ◽  
Birth Weight ◽  
Drug Use ◽  
Critically Ill ◽  
Medication Errors ◽  
Off Label ◽  
Increased Risk ◽  
Critically Ill Neonates ◽  
Medication Risk

BACKGROUND: Off-label drug (OLD) use is common in neonates. There is a dearth of information associating the OLD use and the risk of medication errors in critically ill neonates. Hence, the present study was carried out. METHODS: Drug prescriptions in neonates admitted to the intensive care unit of a tertiary care hospital between September 2018 and June 2019 were evaluated. Details on their demographics, reason for admission in intensive care unit, drug-related information and serum creatinine were extracted. United States Food and Drug Administration approved drug labels were compared. World Health Organization (WHO) anatomy, therapeutic and chemical (ATC) classification was used for drug categorization. We assessed the risk of medication errors using a validated tool, medication risk score (MERIS). RESULTS: One hundred and seventy-one neonates with 2394 prescriptions were included in this study. Seventy one percent of the neonates in the present study received at least one OLD/unlicensed prescription item. A trend in increased numbers of OLD/unlicensed drug use in more premature and lower birth weight neonates were observed. Medication risk score was significantly higher in neonates receiving OLD/unlicensed drugs compared to those with only labelled drugs. Very and extreme pre-term (along with very low and extremely low birth weight) neonates were at higher risk of medication errors compared to others. Presence of OLD/unlicensed prescribed items is associated with an increased risk of medication errors by an odds ratio of 20.4 compared to labelled drugs. CONCLUSION: Significant proportions of critically ill neonates received at least one OLD/unlicensed drug and such use was associated with potentially increased risk of medication errors.

scholarly journals Masivno krvavljenje i mehanizmi koagulopatije u traumi

2006 ◽  
Vol 53 (4) ◽  
pp. 89-92
Author(s):  
N. Popovic ◽  
Z. Blagojevic ◽  
V. Nikolic ◽  
Lj. Arsenijevic ◽  
A. Karamarkovic ◽  
...  
Keyword(s):  
Massive Bleeding ◽  
Vascular Integrity ◽  
Small Vessels ◽  
Civilian Trauma ◽  
Risk Of Mortality ◽  
Lethal Triad ◽  
Increased Risk ◽  
Threatening Condition ◽  
Life Threatening ◽  
Dilutional Coagulopathy

Trauma is disease of the young, mainly affecting people between 15-40 years of age. Uncontrolled massive bleeding is the leading cause of early in-hospital mortality, within 48h of admission, and the second leading cause of prehospital death in victims of both military and civilian trauma, accounting for 40-45% of the total fatalities. Coagulopathy develops early after injury and is present in 25- 36% of trauma victims upon admission to the emergency department. Coagulopathy correlates to the severity of trauma and is associated with an increased risk of mortality. The aim of this paper is to explain pathophysiology of developing coagulopathy in trauma. The coagulopathy in the trauma patient is complex and multifactorial. It includes: dilutional coagulopathy, hypothermia, acidosis, hyperfibrinolysis, anemia and consumption coagulopathy. When the patient develops the so called ?lethal triad? of hypothermia, acidosis and coagulopathy, surgical restoration of vascular integrity may be insufficient to achieve a definitive control of blood loss and non-mechanical bleeding from small vessels, usually terminated by spontaneous coagulation, becomes a life-threatening condition.

scholarly journals Coronavirus Disease 2019 (Covid-19) Outbreak and Pituitary Apoplexy

2021 ◽  
Author(s):  
Rafael Martinez-Perez ◽  
Benjamin W Carroll ◽  
Daniel Duran ◽  
James S Neill ◽  
Gustavo D Luzardo ◽  
...  
Keyword(s):  
Pituitary Adenoma ◽  
Molecular Mechanisms ◽  
Pituitary Apoplexy ◽  
Care Center ◽  
Tertiary Care ◽  
Perioperative Period ◽  
Common Symptom ◽  
Increased Risk ◽  
Threatening Condition ◽  
Life Threatening

Abstract Background. Pituitary apoplexy (PA) is a rare and potentially life-threatening condition characterized by pituitary hemorrhage, often in the setting of a preexisting pituitary adenoma. Risk factors and mechanisms associated to PA are poorly understood. Although involvement of the nervous system in SARS-CoV-2 infection causing intracranial hemorrhagic complications has been documented, the association between COVID-19 infection and PA has yet to be determined. Methods . From a prospectively collected database of patients with pituitary adenomas, we retrospectively reviewed the electronical medical records and scans of patients with pituitary apoplexy during the COVID-19 outbreak, since March 2020 to December 2020, that were treated at a tertiary care center. Results. Herein, we report three consecutive cases of patients with PA and concomitant COVID-19 infection. Most common symptom at presentation was headache and visual worsening. Included patients were successfully treated with surgical decompression and medical management of the hormonal deficits, experiencing moderate to significant improvement of their visual symptoms at last follow up. COVID-19 infection in the perioperative period was corroborated throughout polymerase chain reaction testing in all patients. Conclusions. COVID-19 positive patients may be at an increased risk of developing PA. Angiotensin-converting enzyme 2 receptors expressed in cerebrovascular endothelium may potentially play a role in the molecular mechanisms that induce changes of the vascular autoregulation and cerebral blood flow and predispose to pituitary hemorrhage in patients harboring a pituitary adenoma.

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