Paediatric desmoid tumour of the head and neck: a complete response to chemotherapy and the paradigm shift towards non-surgical management

2021 ◽  
Vol 14 (1) ◽  
pp. e237509
Author(s):  
Filipa Proença ◽  
Claudia Constantino ◽  
Maria Manuel Lemos ◽  
Alexandra Borges

Desmoid tumours are rare locally aggressive mesenchymal tumours with a high local recurrence rate, particularly in the head and neck locations. We present the case of a 5-year-old child with a locally invasive inoperable desmoid tumour of the masticator space who underwent a low-dose chemotherapy regimen for 17 months, obtaining a long-term complete response. Definitive evidenced-based treatment guidelines are lacking. Therefore, paediatric patients should be managed by specialised multidisciplinary teams to try to achieve the best tumour control while minimising treatment associated morbidity and mortality. Available treatment options include surgery, radiotherapy and different systemic medical therapies. Whereas traditionally, surgery was the mainstay of treatment, more conservative options have increasingly shown adequate tumour control with little associated morbidity. In this decision making it is mandatory to take into account the patient’s age, tumour location and extension, and potential short-term and long-term treatment-related sequelae to minimise functional and cosmetic compromise.

2021 ◽  
Vol 5 (19) ◽  
pp. 3799-3806
Author(s):  
Xiaoling Cheng ◽  
LingLing Fu ◽  
Jingyao Ma ◽  
Hao Gu ◽  
Zhenping Chen ◽  
...  

Abstract Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder with isolated thrombocytopenia and risk of hemorrhage. Treatment with eltrombopag increases and maintains hemostatic platelet counts; however, to date, long-term data are lacking on the outcome of children with ITP who are treated with eltrombopag. This prospective, observational, longitudinal cohort study evaluated the efficacy and safety of eltrombopag in pediatric patients with persistent or chronic ITP. For the 116 pediatric patients enrolled, duration of eltrombopag treatment was at least 3 months. Median effective dose was 25 mg/day, 50 mg/day, and 50 mg/day, respectively, for children age 5 years or younger, 6 to 11 years, or 12 years or older. In all, 89 patients (76.7%) achieved overall response, 53 (45.7%) achieved complete response, and 36 (31.0%) achieved response. Median platelet counts increased by week 1 and were sustained throughout the treatment period. During treatment with eltrombopag, the proportion of patients with grade 1 to 4 bleeding symptoms decreased from 83.61% at baseline to 9.88% at 6 months when only grade 1 was reported. Forty-three patients (37.1%) reported using concomitant medications at study entry, which was reduced to 1 patient (2.5%) who needed concomitant medications at 12 months. All adverse events were grade 1 or 2 according to Common Terminology Criteria for Adverse Events. No serious adverse events, cataracts, malignancies, or thromboses were reported during the study. Long-term treatment with eltrombopag was generally safe, well tolerated, and effective in maintaining platelet counts and reducing bleeding in most pediatric patients with persistent or chronic ITP. Combined with future studies, these findings will help establish how eltrombopag should best be used in the management of pediatric patients with East Asian ancestry.


Author(s):  
Marci Lee Nilsen ◽  
Michael A. Belsky ◽  
Nicole Scheff ◽  
Jonas T. Johnson ◽  
Dan P. Zandberg ◽  
...  

2008 ◽  
Vol 31 (5) ◽  
pp. 465-469 ◽  
Author(s):  
Ramesh Rengan ◽  
David G. Pfister ◽  
Nancy Y. Lee ◽  
Dennis H. Kraus ◽  
Jatin P. Shah ◽  
...  

Oral Oncology ◽  
2017 ◽  
Vol 74 ◽  
pp. 115-122 ◽  
Author(s):  
Shiran Sun ◽  
Xiaodong Huang ◽  
Li Gao ◽  
Ye Zhang ◽  
Jingwei Luo ◽  
...  

2000 ◽  
Vol 12 (3) ◽  
pp. 115-119 ◽  
Author(s):  
R.W. Licht

ABSTRACTBackground: In clinical practice, typical antipsychotics are widely used in the treatment of bipolar disorder, albeit in treatment guidelines often considered as adjunctive agents only. Recently, focus has shifted towards the use of atypical antipsychotics. This paper reviews the advantages and disadvantages associated with the use of antipsychotics in bipolar disorder.Methods: Randomised controlled trials (RCTs) were selected for review. A few review articles were also cited.Results: Typical antipsychotics, at least some of them, are powerful antimanics, beneficial for severe agitation in particular. However, in the long term treatment, typical antipsychics may precipitate depression. Among the atypical antipsychotics, both risperidone and olanzapine are clearly antimanic alternatives, although olanzapine is the best studied. Clozapine seems to be useful when other treatments fail to work.Conclusions: Antipsychotics are beneficial for some clinical presentations of mania. To minimize side effets, atypical agents should be preferred before typical agents, unless parenteral administration is needed. Despite the lack of RCTs, antipsychotics also seem to be useful as adjunctive agents in the treatment of psychotic bipolar depression. For the long term treatment of bipolar disorder, typical antipsychotics should be used only under certain circumstances. The place of atypical antipsychotics in the long term treatment of bipolar disorder remains to be studied.


2020 ◽  
Vol 79 (8) ◽  
pp. 1098-1104 ◽  
Author(s):  
Filippo Fagni ◽  
Alessandra Bettiol ◽  
Rosaria Talarico ◽  
Giuseppe Lopalco ◽  
Elena Silvestri ◽  
...  

ObjectiveTo evaluate the effectiveness and safety of secukinumab in patients with a mucosal and articular Behçet’s phenotype resistant to conventional and biologic treatment.MethodsA multicentre retrospective study was performed on 15 patients with a mucosal and articular phenotype of Behçet’s syndrome fulfilling the International Criteria for Behçet’s Disease and refractory to treatment with colchicine, disease-modifying antirheumatic drugs and at least one antitumour necrosis factor-α agent. Minimum follow-up was set at 6 months. Six patients with a polyarticular involvement were treated with secukinumab 300 mg/month, while all other cases received secukinumab 150 mg/month. Dose increase from 150 to 300 mg per month and shortening of administration frequency were allowed for poor disease control. Response evaluation was based on the number of oral ulcers in the previous 28 days and Disease Activity Score-28 for articular manifestations.ResultsAt 3 months of follow-up, nine (66.7%) patients achieved a response (complete or partial), and this proportion further increased to 86.7% at 6 months, 76.9% at 12 months, 90.0% at 18 months and 100.0% after 24 months. Notably, all patients who started with secukinumab 300 mg/month achieved complete response by month 6. Seven (46.7%) patients could achieve a response only after switching to a higher dosage.ConclusionsOur study suggests that secukinumab at a dose of 150 and 300 mg per month is safe and effective for the long-term treatment of patients with Behçet’s syndrome with a mucosal and articular phenotype refractory to previous treatments. Notably, secukinumab 300 mg/month resulted in superior complete mucosal and articular responses with no serious or dose-related adverse effects.


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