First-choice Therapy Research Articles

Diagnostic mystery—a rare right ventricular cardiac hemangioma: a case report

BackgroundCardiac hemangiomas are rare in all kinds of benign cardiac tumors. Although cardiac hemangiomas affect all ages and may occur anywhere within the heart, right ventricular hemangiomas are extremely uncommon.Case presentationWe report a 56-year-old woman presented with chest tightness and breath shortness for 3 months. Transthoracic echocardiography and coronary computed tomography angiography showed a mass located adjacent to the apex of the right ventricle but both failed to figure out where the mass originated from, remaining a diagnostic mystery preoperatively. The mass was removed successfully and the histopathological examination confirmed it was hemangioma.ConclusionsCardiac magnetic resonance should be the ultimate diagnostic tool of cardiac tumors. Surgical removal, associated with a low recurrence rate and long-term survival benefits, should be the first choice of therapy for cardiac hemangiomas.

Gastric MALT lymphoma - from pathogenetic insights to consequent deescalation of therapy

Gastric MALT- (mucosa-associated-lymphoid-tissue) lymphoma represents the most frequent gastrointestinal lymphoma. For decades, surgery and later on radiation and chemotherapy were regarded as established therapy. Some 30 years ago, the pathogenetic role of Helicobacter pylori infection for the development of gastric MALT-lymphoma became evident. During the following years, the pathogenetic insights were consequently implemented into clinical medicine. This lead to a radical change of the therapeutic approach to these lymphoma. Nowadays, Helicobacter pylori eradication is the internationally established therapy of first choice. It is followed by lymphoma regression in most cases. The long-term prognosis of patients after exclusive eradication therapy is excellent, even if endoscopic and/or histological residuals persist and a watch-and-wait strategy is favored.The pathogenetic insights und their clinical application implicated a consequent deescalation of therapy of gastric MALT-lymphoma. This review summarizes the single steps of this development and gives a recommendation for the actual management of patients with gastric MALT lymphoma.

Low-dose immune tolerance induction therapy in children of Arab descent with severe haemophilia A, high inhibitor titres and poor prognostic factors for immune tolerance induction treatment success.

IntroductionImmune Tolerance Induction (ITI) is the first‐choice therapy to eradicate Factor VIII (FVIII) neutralizing antibodies in patients with haemophilia A (HA). There is limited published data on ITI from East Mediterranean countries.AimTo assess the effectiveness of a low‐dose ITI regimen to eradicate FVIII neutralizing antibodies in children with severe HA and high‐titre inhibitors.MethodsA prospective, single‐arm study was conducted in children with HA (FVIII < 1 IU/dl), high‐titre inhibitors and poor prognostic factors for successful ITI. Patients were treated with ∼50 IU/kg plasma‐derived FVIII containing von Willebrand factor (pdFVIII/VWF) concentrate (Koate‐DVI, Grifols) three times a week. Time to achieve tolerance, total and partial success were analysed after ITI. Annual bleeding rate (ABR), number of target joints, FVIII recovery and school absence were compared before and after ITI.ResultsTwenty patients with median (range) age of 6.2 (3–12) years and pre‐ITI inhibitor titre of 36.5 (12–169) BU were enrolled. ITI lasted ≤12 months (early tolerization) in 45% of patients. Median follow‐up was 12 months (3–22) and total response rate was 80% (60% total success; 20% partial success). Patients with two and three poor prognosis factors achieved overall success rate of 60% and 50%, respectively. ABR, target joints and school absence were reduced after ITI by 60%, 50% and 44.1%, respectively. In successful ITI tolerized patients, FVIII recovery was 90 (60–100)%.ConclusionA low‐dose ITI therapy using a pdFVIII/VWF concentrate achieved at least partial tolerance in 80% of patients, and reduced annual bleeds in children with high inhibitor titres and at least one poor prognosis factor for ITI treatment success.

Statin-Associated Myopathy: Emphasis on Mechanisms and Targeted Therapy.

Hyperlipidemia is a major risk factor for cardiovascular morbidity and mortality. Statins are the first-choice therapy for dyslipidemias and are considered the cornerstone of atherosclerotic cardiovascular disease (ASCVD) in both primary and secondary prevention. Despite the statin-therapy-mediated positive effects on cardiovascular events, patient compliance is often poor. Statin-associated muscle symptoms (SAMS) are the most common side effect associated with treatment discontinuation. SAMS, which range from mild-to-moderate muscle pain, weakness, or fatigue to potentially life-threatening rhabdomyolysis, are reported by 10% to 25% of patients receiving statin therapy. There are many risk factors associated with patient features and hypolipidemic agents that seem to increase the risk of developing SAMS. Due to the lack of a “gold standard”, the diagnostic test for SAMS is based on a clinical criteria score, which is independent of creatine kinase (CK) elevation. Mechanisms that underlie the pathogenesis of SAMS remain almost unclear, though a high number of risk factors may increase the probability of myotoxicity induced by statin therapy. Some of these, related to pharmacokinetic properties of statins and to concomitant therapies or patient characteristics, may affect statin bioavailability and increase vulnerability to high-dose statins.

Phospholipid Derivatives of Cinnamic Acid Restore Insulin Sensitivity in Insulin Resistance in 3T3-L1 Adipocytes.

Background: Insulin resistance (IR) is a condition in which the physiological amount of insulin is insufficient to evoke a proper response of the cell, that is, glucose utilization. Metformin is the first choice for therapy, thanks to its glycemic efficacy and general tolerability. In addition, various natural compounds from plant extracts, spices, and essential oils have been shown to provide health benefits regarding insulin sensitivity. In the present study, we analyzed the effect of phospholipid derivatives of selected natural aromatic acids on insulin action and their potential use to overcome insulin resistance. Methods: The 3T3-L1 fibroblasts were differentiated into mature adipocytes; next, insulin resistance was induced by palmitic acid (16:0). Cells were further cultured with phenophospholipids at appropriate concentrations. To assess insulin sensitivity, we measured the insulin-stimulated glucose uptake, using a glucose uptake test. Results: We showed that cinnamic acid (CA) and 3-methoxycinnamic acid (3-OMe-CA) restored the proper insulin response. However, 1,2-dicinnamoyl-sn-glycero-3-phosphocholine (1,2-diCA-PC) and 1-cinnamoyl-2-palmitoyl-sn-glycero-3-phosphocholine (1-CA-2-PA-PC) improved insulin sensitivity in insulin-resistant adipocytes even stronger, exhibiting more beneficial effects. Conclusions: The binding of aromatic acids to phosphatidylcholine increases their beneficial effect on insulin sensitivity in adipocytes and expands their potential practical application as nutraceutical health-promoting agents.

Fingolimod in pediatric multiple sclerosis: Six case reports

Treatment for pediatric-onset multiple sclerosis (POMS) currently reflects treatment for adult-onset MS, despite some differences in its clinical course. First-choice treatment of POMS generally consists of interferon β-1a or glatiramer acetate, with therapies such as natalizumab or fingolimod reserved for second-choice treatment. In cases of severe disease, both fingolimod and natalizumab can be considered first-choice therapy.

New developments and perspectives in acromegaly

Acromegaly is a rare but severe disorder which is usually due to an excessive secretion of growth hormone (GH) by a pituitary adenoma. Screening mainly relies on the measurement of insulin-like growth factor 1, and confirmatory diagnostics includes a GH suppression test. As delayed diagnosis results in increased morbidity and mortality, we here discuss recently published suggestions regarding the biochemical work-up of suspected cases and the follow-up of co-morbidities. Moreover, new analytical tools (such as automatic identification of typical facial changes using artificial intelligence) are presented, hopefully allowing for an earlier diagnosis in the future. So far, surgery is still regarded as therapy of first choice. In cases without postoperative remission, a new imaging approach (combining sellar magnetic resonance imaging and position emission tomography) may improve the results of repeated surgery. The pharmaceutical arsenal now includes the first orally available somatostatin analogue, and recent data on possible drug combinations and the outcome of radiotherapy are presented. Finally, special attention is paid to older and pregnant patients, as well as certain considerations during the COVID-19 pandemic (where appropriate diagnosis and management of acromegaly is particularly challenging).

Middle Cerebral Artery M2 Thrombectomy in the STRATIS Registry.

The safety and benefit of mechanical thrombectomy in the treatment of acute ischemic stroke patients with M2 segment middle cerebral artery occlusions remain uncertain. Here, we compare clinical and angiographic outcomes in M2 versus M1 occlusions in the STRATIS (Systematic Evaluation of Patients Treated With Neurothrombectomy Devices for Acute Ischemic Stroke) Registry. The STRATIS Registry was a prospective, multicenter, nonrandomized, observational study of acute ischemic stroke large vessel occlusion patients treated with the Solitaire stent-retriever as the first-choice therapy within 8 hours from symptoms onset. Primary outcome was defined as functional disability at 3 months measured by dichotomized modified Rankin Scale. Secondary outcomes included reperfusion rates and rates of symptomatic intracranial hemorrhage. A total of 984 patients were included, of which 538 (54.7%) had M1 and 170 (17.3%) had M2 occlusions. Baseline demographics were well balanced within the groups, with the exception of mean baseline National Institutes of Health Stroke Scale score which was significantly higher in the M1 population (17.3±5.5 versus 15.7±5.0, P≤0.001). No difference was seen in mean puncture to revascularization times between the cohorts (46.0±27.8 versus 45.1±29.5 minutes, P=0.75). Rates of successful reperfusion (modified Thrombolysis in Cerebral Infarction≥2b) were similar between the groups (91% versus 95%, P=0.09). M2 patients had significantly increased rates of symptomatic ICH at 24 hours (4% versus 1%, P=0.01). Rates of good functional outcome (modified Rankin Scale score of 0–2; 58% versus 59%, P=0.83) and mortality (15% versus 14%, P=0.75) were similar between the 2 groups. There was no difference in the association of outcome and onset to groin puncture or onset to successful reperfusion in M1 and M2 occlusions. In the STRATIS Registry, M2 occlusions achieved similar rates of successful reperfusion, good functional outcome, and mortality, although increased rates of symptomatic ICH were demonstrated when compared with M1 occlusions. The time dependence of benefit was also similar between the 2 groups. Further studies are needed to understand the benefit of mechanical thrombectomy for M2 occlusions. URL: https://www.clinicaltrials.gov; Unique identifier: NCT02239640.

Everolimus versus sirolimus for angiomyolipoma associated with tuberous sclerosis complex: a multi-institutional retrospective study in China

PurposeTo evaluate the efficacy and safety of everolimus and sirolimus in patients with tuberous sclerosis complex-associated angiomyolipomas (TSC-AML).Materials and methods We performed a multi-institutional retrospective study of TSC-AML patients treated with oral everolimus 10 mg or sirolimus 2 mg per day for at least 3 months. Angiomyolipoma volume was estimated using orthogonal measurements by MRI or CT. Adverse events (AEs) were assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events. All analyses were performed using SPSS 19.0 software.ResultsResponse rates were high in both groups. With the prolonged medication durations, the therapeutic efficacy of both agents became more significant. The TSC-AML volume reduction after 6 and 12 months was more pronounced in patients with everolimus than those with sirolimus. More than half of the patients treated with everolimus had ≥ 50% reduction, and approximately 80% of them had ≥ 30% reduction, which was higher than that in patients treated with sirolimus. Regarding safety, there was no significant difference in the incidence of AEs between the two groups.ConclusionsBoth everolimus and sirolimus are excellent therapeutic options for TSC-AML. However, everolimus has a better therapeutic efficacy than sirolimus, particularly in reducing TSC-AML volume. Everolimus is therefore recommended as the first choice of therapy for TSC-AML.

Management of Gestational Diabetes Mellitus with Medical Nutrition Therapy

Gestational diabetes mellitus (GDM) also referred to as hyperglycemia during pregnancy is posing to be a big health concern for women and is emerging as a major public health problem in India. Early diagnosis and tight glycemic control during pregnancy play a pivotal role in improving pregnancy outcomes in women with GDM. Once diagnosis is made, lifestyle behavioral changes with medical nutrition therapy (MNT) and physical activity form the first choice of therapy for the management of GDM. Failure to meet glycemic goals with these requires the addition of medication/insulin. Regular blood glucose monitoring and record keeping must be encouraged to improve adherence and review treatment efficacy. MNT for GDM is defined as a “carbohydrate-controlled” meal plan that provides adequate nutrition along with appropriate weight gain and fetal well-being to meet increased requirements of energy, protein, and micronutrients, achieve normoglycemia, and prevent nutritional ketosis. Choosing nutrient-dense carbohydrate sources which are low to moderate in glycemic index and glycemic load with focus on the correct amount and distribution of carbohydrates at meals helps control postprandial glucose excursions which is the biggest challenge in GDM. Blood glucose levels can be difficult to control in the morning due to increased insulin resistance secondary to dawn phenomenon seen in women with GDM. Splitting meals at breakfast has shown to improve post-breakfast blood glucose levels. This article is a comprehensive review of guidelines and scientific literature for the dietary management of GDM aimed at achieving normoglycemia, ensuring fetal and maternal wellbeing, and preventing adverse outcomes in pregnancy. The literature has been retrieved from various databases such as “Google Scholar,” “PubMed,” and “Cochrane Database of Systematic Reviews” using relevant keywords related to the topics discussed in this manuscript.

Awareness and practice of solution focused therapy in mental health professionals

ABSTRACT Solution Focused Therapy (SFT) is a paradigm shift from the traditional psychotherapy which is practised more by counsellors and social workers. Formal training of this therapy is observed across countries. The objective of the study was to assess SFT knowledge and its practice among mental health professionals in India post their educational degree. A descriptive cross-sectional design was used. Convenience sampling was used with an online survey mailed to 100 known professionals from July to September, 2018. The sample comprised of practicing Clinical Psychologists and Psychiatric Social Workers who held a minimum qualification of the superspecialty degree MPhil (Master of Philosophy) in their respective fields. Analysis was done both qualitatively and quantitatively. Descriptive statistics found that only 5.55% of Clinical Psychologists and 22.22% of Psychiatric Social Workers used it as a sole therapy. About 72.2% of the mental health professionals felt that Solution Focused Therapy should be incorporated in the traditional specialized training program. The two themes obtained from thematic content analysis were the understanding and the efficacy of this form of therapy. The awareness and practice of Solution Focused Therapy among mental health professional appears low. Despite awareness of SFTs’ principles and techniques, it was not the first choice of therapy for mental health professionals

Taking a Rational Approach to a Reported Antibiotic Allergy.

Up to 10% of hospitalized patients have an antibiotic allergy label in their medical file, most frequently concerning penicillins. However, the vast majority of reported allergies to antibiotics does not represent a "true" allergy but are due to drug intolerance, idiosyncratic reactions or symptoms of the concurrent infectious disease. Since antibiotic allergy labels result in deviation from first-choice antimicrobial therapy, tackling the issue of incorrect antibiotic allergy labelling, already at young age, is a core element of antibiotic stewardship. In this article, we describe the structured approach to the patient with a presumed antibiotic allergy with emphasis on key elements of allergy-specific history taking and the limited risk of cross-allergic reactions between beta-lactam subclasses.

Bleomycin sclerotherapy following doxycycline lavage in the treatment of ranulas: A retrospective analysis and review of the literature.

A ranula is a mucus-filled salivary pseudocyst that forms in the floor of the mouth, commonly arising from the sublingual or submandibular salivary glands following obstruction or trauma. Complete excision of the injured gland and removal of the cyst content is the first-choice therapy, but has the potential for complications related to injury to nearby structures. As such, minimally invasive approaches such as percutaneous sclerotherapy have been investigated. We aim to contribute to the literature by assessing the efficacy and safety of our technique through our experience with 18 patients over the last decade. This retrospective study evaluated 18 patients with intraoral and plunging ranulas treated by percutaneous bleomycin ablation. The primary endpoint was the treatment result. Secondary endpoints included bleomycin dosage and complications. The study evaluated 12 males and six females with a median age of 23.5 years (range 13-39 years). At a final follow-up of at least 2 months (6.5±5.5 months), four patients demonstrated complete response (22%) and 14 patients demonstrated residual presence, recurrence, or regrowth of the lesion (78%). There were no statistically significant associations between outcomes and history of prior treatment, number of treatments, and size or type of ranula. No complications were noted. Our findings indicate that bleomycin, while safe for use in various head and neck malformations, is of limited utility in ranula therapy when the offending gland is not addressed primarily.

Pharmacological Management of Gastro-Esophageal Reflux Disease: An Update of the State-of-the-Art.

Gastro-esophageal reflux disease (GERD) is a highly prevalent, chronic disorder, whose knowledge remains limited and the management of these patients changes continuously. This review provides a summary of the most recent advancements in the pathogenesis of this disease and the new drugs introduced into the market to overcome some of the unmet needs of traditional therapies. Nowadays, the most fruitful diagnostic examinations are 24-hour impedance-pH monitoring, which allows us to separate true NERD from esophageal functional disorders and high-resolution manometry, which helps to exclude the existence of motility disorders sharing the same symptoms of GERD. Proton pump inhibitors (PPIs) remain the first-choice therapy in the treatment of GERD, but a consistent proportion of these patients continue to experience symptoms despite their intake. These cases pertain mainly to the subpopulation with non-erosive reflux disease (NERD) and represent very challenging clinical situations, because it is mandatory to understand the reasons for PPI failure. The management of these difficult patients requires necessarily to test them and avoid the use of empiric treatments that are often unsuccessful, costly and potentially dangerous. Recently, several new drugs have been used to increase the defensive properties of this mucosa with promising results in randomized clinical trials.

Arthroscopic Management of Synovial Osteochondromatosis of the Elbow

Primary synovial chondromatosis is characterized by newly formed chondral or osteochondral nodules in the synovial membrane, which may detach and form loose bodies. The loose bodies can be calcified or ossified, and the condition is termed synovial osteochondromatosis. Three distinct stages can be identified in primary synovial chondromatosis : phase I is active synovitis without loose bodies, phase II shows nodular synovitis along with loose bodies, and phase III is characterized by the presence of loose bodies with the resolution of synovitis. Surgical treatment has been recommended as the first choice of therapy in phases II and III disease. Complete synovectomy and removal of all loose bodies is advisable for prevention of recurrence of the disease. In this technical note, the technical details of arthroscopic removal of loose bodies and synovectomy for the management of synovial osteochondromatosis of the elbow is described. Compared with open procedures, the arthroscopic approach has many advantages, including a shorter rehabilitation period and higher patient satisfaction.

A comparative study to evaluate the efficacy of metformin monotherapy on glycemic markers in normal weight, over weight, and obese patients with Type 2 diabetes mellitus

Background: Metformin has been recommended as pharmacological therapy of first choice in Type 2 diabetes mellitus (T2DM) but there remains a gap in the present literature regarding relative efficacy and choice of metformin as monotherapy in patients who are non-obese when compared to obese. Aim and Objectives: The aim of the study was to evaluate the efficacy of Metformin as monotherapy on glycemic markers in normal weight, over weight, and obese T2DM patients. Materials and Methods: After obtaining permission from institutional ethics committee, 90 treatment naive patients with T2DM who met inclusion criteria were included in this study. They were categorized into normal weight, over weight, and obese based on BMI. Efficacy was measured by reduction in glycemic parameters at end of weeks 4, 8, 12, and 16 from baseline. Safety was assessed by monitoring adverse events. Data analyzed using analysis of variance, Student t-test for continuous data and Chi-square test for categorical data. Results: There was a significant decrease in glycosylated hemoglobin, fasting blood sugar, and postprandial blood sugar levels from baseline to end of 16 weeks in all three groups (P 0.05). A significant decrease in body weight was observed in overweight and obese group whereas the reduction (0.3 Kg) is not significant in normal weight group. The treatment was well tolerated in all three groups. Conclusion: Metformin in normal weight group was found to be as efficacious as that of overweight and obese group for treating newly detected T2DM. Furthermore, the weight loss in normal weight group is negligible compared to overweight and obese group patients.

Контроль симптомов аллергического ринита у взрослых лиц в Российской Федерации: результаты онлайн-опроса

Aim: subjective assessment by patients with allergic rhinitis (AR) of the efficacy in control of the main symptoms, seeking medical care for AR symptoms and general treatment methods. Patients and Methods: from April to August 2020, a study was conducted of 328 adult respondents (buyers of medicines for the AR treatment: antihistamines and/or intranasal corticosteroids), who voluntarily agreed to an online survey. 164 respondents purchased medicines based on the prescription or doctor recommendation, while other 164 chose medicines independently. Visual analog scale (VAS) was used to assess the severity of individual AR symptoms (nasal congestion, runny nose/nasal discharge, sneezing, itchy nose, cough), as well as eye and general symptoms (general malaise, drowsiness, headache). Results: 56% of respondents had persistent AR symptoms. Herewith, 60% of respondents suffered from seasonal AR, 40% — perennial AR. 52% of respondents had a mild AR form. However, the main triad of AR symptoms (nasal congestion, rhinorrhea and sneezing), despite the non-severe AR form in the absolute majority of respondents corresponded to moderate or severe severity (VAS&gt;5) and indicated a lack of AR control. 31% of those surveyed experienced severe AR symptoms that disrupted daytime activity and sleep. The respondents were most concerned about nasal congestion, runny nose/rhinorrhea, and sneezing (the average VAS score was 7.3, 7.1, and 6.5, respectively). The frequency of visits to the doctor for AR was very low: 95% of respondents visited their doctor no more than once a year, while 48% of them — every few years. The first choice in therapy was antihistamines, regardless of the presence or absence of doctor’s recommendation. In second place were decongestants, in third — intranasal corticosteroids, which were more commonly purchased on the doctor’s recommendation. Conclusion: adult respondents with a suspected diagnosis of AR, despite uncontrolled and marked symptoms, underestimate their disease, rarely consult a doctor, and prefer to follow the recommendations of relatives and friends or pharmacists for treatment. The lack of AR control among adults in our country is obvious and requires the active participation of the medical and pharmaceutical community for salvation of such problem. KEYWORDS: allergic rhinitis, visual analog scale, control of allergic rhinitis symptoms, survey, therapy choice, severity of allergic rhinitis symptoms. FOR CITATION: Nenasheva N.M., Shilenkova V.V. Control of allergic rhinitis symptoms in adults in the Russian Federation: online survey results. Russian Medical Inquiry. 2021;5(1):25–31. DOI: 10.32364/2587-6821-2021-5-1-25-31.

Should metformin remain the first-line therapy for treatment of type 2 diabetes?

Metformin is a biguanide that is used as first-line treatment of type 2 diabetes mellitus and is effective as monotherapy and in combination with other glucose-lowering medications. It is generally well-tolerated with minimal side effects and is affordable. Although the safety and efficacy of metformin have been well-established, there is discussion regarding whether metformin should continue to be the first choice for therapy as other anti-hyperglycemic medications exhibit additional advantages in certain populations. Despite a long-standing history of metformin use, there are limited cardiovascular outcomes data for metformin. Furthermore, the available studies fail to provide strong evidence due to either small sample size or short duration. Recent data from glucagon-like peptide-1 receptor agonist and sodium-glucose cotransporter-2 inhibitor cardiovascular and renal outcomes trials demonstrated additional protection from diabetes complications for some high-risk patients, which has impacted the guidelines for diabetes management. Post-hoc analyses comparing hazard ratios for participants taking metformin at baseline versus not taking metformin are inconclusive for these two groups. There are no data to suggest that metformin should not be initiated soon after the diagnosis of diabetes. Furthermore, the initiation of newer glycemic-lowering medications with cardiovascular benefits should be considered in high-risk patients regardless of glycemic control or target HbA1c. However, cost remains a major factor in determining appropriate treatment.

Endorectal formalin instillation or argon plasma coagulation for hemorrhagic radiation proctopathy therapy: a prospective and randomized clinical trial

Radiotherapy may cause hemorrhagic radiation proctopathy (HRP). For conservative treatment of refractory HRP, argon plasma coagulation (APC) is the first-choice therapy. Endorectal formalin instillation (EFI), in turn, is an attractive treatment option because of its satisfactory results, great availability, and low cost. Nevertheless, comparative studies between these procedures are rather scarce. This study aims to make a prospective and randomized comparison of the outcomes in 2 HRP patient groups treated with either APC or EFI. Twenty-seven patients (11 women), with a mean age of 67 years (range, 36-83), were randomized to receive either APC (n= 14) or EFI (n= 13). On completion of the treatment, comparisons were made in relation to the baseline for each patient and between groups for endoscopic findings according to the Vienna score and the telangiectasia distribution pattern score (TDP); the impact of radiation proctitis on patients' lives was made according to the modified radiation toxicity score (MRTS) and hemoglobin levels. Number of sessions, duration of therapy, and adverse events were also compared between groups. The endoscopic therapeutic success (ETS) was defined by the absence or only few residual telangiectasias (TDP≤1) on conclusion. An ETS of 92.8% was achieved in patients treated with APC and 92.3% for those treated with EFI (P > .05); there was an MRTS improvement of 85.7% in APC patients and 69.2% in EFI patients (P > .05). Mild adverse events occurred, respectively, in 23% and 28.5% in the EFI and APC groups (P > .05). The study showed that APC and EFI have similar efficacy and a high safety profile for HRP treatment. (Clinical trial registration number: 3.120.353.).

Hemangiol in infantile haemangioma: A paediatric post-marketing surveillance drug study.

Infantile haemangioma (IH) is the most common benign tumour in children. Since 2014, propranolol has become the first-choice therapy and currently Hemangiol is the only approved drug for complicated haemangioma. This post-marketing study reports the use of Hemangiol for IH in paediatric practice. From January 2014 to November 2018, 94 children (median age 4 [0; 21] months; 75% female) treated with Hemangiol for proliferative IH were enrolled in the study. The systematic paediatric cardiology consultation never contraindicated beta-blockers. Two Hemangiol initiation protocols were used: a conventional ambulatory 3-week titration phase protocol (n = 76, 80.9%), and a rapid initiation protocol with a 48-hour dose escalation in conventional hospitalization for severe proliferative or ulcerated IH (n = 18, 19.1%). In both protocols, the haemodynamic tolerance was good. The mean maintenance dose of Hemangiol was 2.7 ± 0.8 mg/kg/day, with a median treatment duration of 7 [1.5; 19] months. Adverse events (AEs) have been found in 25 (26,6%) patients, including 8 (8.5%) patients with serious AEs (uncontrolled bronchial hyperreactivity, n = 5; serious hypoglycaemia, n = 3). Some patients had one or more AEs, a total of 24 nonserious AEs was reported in 19 patients (sleep disturbances, n = 9; respiratory disorders, n = 5; digestive disorders, n = 6). No cardiac adverse event was reported. This post-marketing surveillance drug study supports the good tolerance of Hemangiol in children with IH. A rapid initiation protocol is of interest when treatment is urgent. The pretherapeutic paediatric cardiology consultation should not be systematic but only indicated for specific patients. CLINICALTRIALS.GOV: NCT04105517.