sildenafil-citrate and Vascular-Malformations

Vascular malformations of the head and neck are complex lesions that are notoriously difficult to manage. Treatment of these lesions often requires a multispecialty and multimodal approach. In the modern era of evidence-based medicine, it has become imperative for clinicians to incorporate evidence-based treatment algorithms into their everyday practices. With general widespread inundation of the literature with levels IV and V clinical evidence, however, it is often difficult to draw meaningful conclusions that can be practically applied to the clinical question at hand. When asking how best to manage the most common vascular malformations, we are faced with this large volume of lower level studies conducted in drastically different ways without consistency in outcomes reporting, thus making direct comparison nearly impossible. Furthermore, much of the evidence shows mixed results, adding to confusion over what the optimal evidence-based treatment approaches truly are. In attempt to derive consensus from available literature discussing the management of vascular malformations, we reviewed the current literature detailing modern-day treatment approaches for lymphatic malformations, venous malformations, and arteriovenous malformations of the head and neck.

Topics: Ablation Techniques; Arteriovenous Malformations; Embolization, Therapeutic; Evidence-Based Medicine; Humans; Immunosuppressive Agents; Laser Therapy; Lymphatic Abnormalities; Lymphatic Vessels; Phosphodiesterase 5 Inhibitors; Sclerotherapy; Sildenafil Citrate; Sirolimus; Vascular Malformations; Veins; Watchful Waiting

We have entered an exciting era in the care of patients with vascular anomalies. These disorders require multidisciplinary care and coordination and dedicated centers have emerged to address this need. Vascular tumors have been treated with medical therapies for many years, while malformations have been historically treated with endovascular and operative procedures. The recent serendipitous discoveries of propranolol and sirolimus for vascular anomalies have revolutionized this field. In particular, sirolimus responses are challenging the dogma that vascular malformations are not biologically active. While initially explored for lymphatic anomalies, sirolimus is now being used broadly throughout the spectrum of vascular anomalies. Whether medical therapies are reserved for refractory patients or used first line is currently dependent on the experience and availability of alternative therapies at each institution. On the horizon, we anticipate new drugs targeting genes and pathways involved in vascular anomalies to be developed. Also, combinations of medications and protocols combining medical and procedural approaches are in development for refractory patients.

Topics: Adrenergic beta-Antagonists; Angiogenesis Inhibitors; Bevacizumab; Clinical Decision-Making; Drug Therapy, Combination; Humans; Immunosuppressive Agents; Interferons; Propranolol; Sildenafil Citrate; Sirolimus; Thalidomide; Vascular Malformations

To explore the differences in the efficacy and safety of oral sirolimus and sildenafil in the treatment of pediatric intractable lymphatic malformations (LMs).. From January 2014 to May 2022, we retrospectively enrolled children with intractable LMs treated with oral drugs (sirolimus or sildenafil) and divided the patients into sirolimus and sildenafil groups from Beijing Children's Hospital (BCH). Clinical features, treatment, and follow-up data were collected and analyzed. The indicators were the ratio of reduction in lesion volume pre and posttreatment, the number of patients with improved clinical symptoms, and adverse reactions to the two drugs.. Twenty-four children in the sildenafil group and 31 children in the sirolimus group were included in the present study. The effective rate in the sildenafil group was 54.2% (13/24), with a median lesion volume reduction ratio of 0.32 (-0.23, 0.89) and clinical symptoms improved in 19 patients (79.2%). On the contrary, the effective rate in the sirolimus group was 93.5% (29/31), with a median lesion volume reduction ratio of 0.68 (0.34, 0.96), and clinical symptoms improved in 30 patients (96.8%). There were significant differences (p < 0.05) between the two groups. Regarding safety, four patients in the sildenafil group and 23 patients in the sirolimus group with mild adverse reactions were reported.. Both sildenafil and sirolimus can reduce the volume of LMs and improve clinical symptoms in partial patients with intractable LMs. Sirolimus is more effective than sildenafil and the adverse reactions associated with both drugs are mild and controllable.. III Laryngoscope, 133:3192-3199, 2023.

Topics: Child; Humans; Lymphatic Abnormalities; Retrospective Studies; Sildenafil Citrate; Sirolimus; Treatment Outcome; Vascular Malformations

Congenital extrahepatic portocaval shunt (CEPS), also known as Abernethy malformation, is an extremely rare anomaly of the splanchnic venous system, especially when accompanied by pulmonary arterial hypertension.. We report a case of a 15-year-old female who was diagnosed with CEPS (Abernethy type Ib) accompanied by pulmonary arterial hypertension. This case was incidentally identified during abdominal ultrasound examination and confirmed by mesenteric and splenic arteriography. During more than 4 years of follow-up, after receiving sildenafil (80 mg/day), the patient's condition improved in the first year after discharge. However, one year later, the patient's conditions start to deteriorate.. This article presents a rare case of Abernethy malformation accompanied by pulmonary arterial hypertension, which can be diagnosed by using abdominal ultrasonography, portal vein computed tomography angiography or mesenteric and splenic arteriography. This malformation had limited treatment and poor prognosis.

Topics: Adolescent; Antihypertensive Agents; Arterial Pressure; Disease Progression; Female; Humans; Incidental Findings; Portal Vein; Pulmonary Arterial Hypertension; Pulmonary Artery; Sildenafil Citrate; Treatment Outcome; Vascular Malformations; Vasodilator Agents; Vena Cava, Inferior

Patients with extensive lymphatic malformations associated with tissue overgrowth syndromes (such as Klippel-Trenaunay syndrome and CLOVES) often pose a therapeutic challenge for physicians. In recent years, it has been suggested that oral sildenafil therapy might be used to treat congenital lymphatic malformations. However, this possible new therapy has not yet been used in patients with lymphatic malformations associated with tissue overgrowth syndromes. A 30-year-old man with extensive capillary-lymphatic malformations of the right leg and thorax, and a tissue overgrowth syndrome caused by a somatic mutation in the PIK3CA gene, was treated with oral sildenafil due to symptoms of pain, dyspnea, and functional impairment. Several weeks after the start of the treatment, the patient reported softening of the lymphatic malformation and a significant improvement of his symptoms and physical condition. So far, sildenafil is still considered a last resort in the treatment of complex treatment-resistant lymphatic malformations. With this case report, we demonstrate that sildenafil could also be an alternative treatment option for lymphatic malformations in patients with syndromes belonging to the PIK3CA-related overgrowth spectrum.

Topics: Administration, Oral; Adult; Capillaries; Class I Phosphatidylinositol 3-Kinases; Genetic Predisposition to Disease; Humans; Lymphatic Abnormalities; Male; Mutation; Phenotype; Phosphatidylinositol 3-Kinases; Sildenafil Citrate; Syndrome; Treatment Outcome; Vascular Malformations