Expert consensus document: A consensus on the medical treatment of acromegaly (original) (raw)
Related papers
The role of primary pharmacological therapy in acromegaly
Pituitary, 2013
Background and objectives Primary pharmacological therapy may be the only viable treatment option for many patients with acromegaly, especially those presenting with advanced disease with large inoperable tumors. Long-acting somatostatin analogs are currently the first-line treatment of choice in this setting, where they provide biochemical control and reduce tumor size in a significant proportion of patients. We herein present a brief overview of the role of primary pharmacological therapy in the treatment of acromegaly within the context of Latin America and support this with a representative case study. Case description A 20 year old male presented with clinical and biochemical evidence of acromegaly. The glucose-suppressed growth hormone (GH) was 5.3 lg/L, his insulin-like growth factor-1(IGF-1) was 3.5 times the ULN and serum prolactin greater than 4,000 lg/L. Pituitary MRI revealed a large and invasive mass, extending superiorly into the optic chiasm and laterally into the left cavernous sinus. He was treated with a combination of octreotide and cabergoline with remarkable clinical improvement, normalization of GH and IGF-1 values and striking shrinkage of the adenoma. Conclusion This case illustrates how effective the pharmacological therapy of acromegaly can be and yet at the same time, raises several important issues such as the need for lifelong treatment with costly medications such as the somatostatin analogs. Access to these agents may be limited in regions where resources are restricted and clinicians face challenges in order to make the most efficient use of available options.
Limitations of Current Approaches For The Treatment of Acromegaly
Endocrine Practice, 2016
Objective: Acromegaly is a rare disease characterized by hypersecretion of growth hormone (GH), typically from a benign pituitary somatotroph adenoma, that leads to subsequent hypersecretion of insulin-like growth factor 1 (IGF-1). Patients with acromegaly have an increased risk of mortality and progressive worsening of comorbidities. Surgery, medical therapy, and radiotherapy are currently available treatment approaches for patients with acromegaly, with overall therapeutic goals of lowering GH levels and achieving normal IGF-1 levels, reducing tumor size, improving comorbidities, and minimizing mortality risk. Although surgery can lead to biochemical remission in some patients with acromegaly, many patients will continue to have uncontrolled disease and require additional treatment. Methods: We reviewed recently published reports and present a summary of the safety and efficacy of current treatment modalities for patients with acromegaly. Results: A substantial proportion of patients who receive medical therapy or radiotherapy will have persistently elevated GH and/or IGF-1. Because of the serious health consequences of continued elevation of GH and IGF-1, there is a need to improve therapeutic approaches to optimize biochemical control, particularly in high-need patient populations for whom current treatment options provide limited benefit. Conclusion: This review discusses current treatment options for patients with acromegaly, limitations associated with each treatment approach, and areas within the current treatment algorithm, as well as patient populations for which improved therapeutic options are needed. Novel agents in development were also highlighted, which have the potential to improve management of patients with uncontrolled or persistent acromegaly.
The Journal of Clinical Endocrinology & Metabolism, 2003
Acromegaly is associated with significant morbidities and a 2to 3-fold increase in mortality because of the excessive metabolic action of GH and IGF-I, a marker of GH output. Reductions in morbidity correspond with decreases in IGF-I, and mortality is lowered following normalization of IGF-I or GH levels. Therefore, this has become an important end point. Current guidelines for the treatment of acromegaly have not considered recent advances in medical therapy, in particular, the place of pegvisomant, a GH receptor antagonist. Treatment goals include normalizing biochemical markers, controlling tumor mass, preserving pituitary function, and relieving signs and symptoms. Surgery reduces tumor volume and is considered first-line therapy. Radiation reduces tumor volume and GH and IGF-I levels, but the onset of action is slow and hypopituitarism typically develops. Therefore, pharmacotherapy is often used following surgery or as first-line ther-apy for nonresectable tumors. Dopamine agonists can be considered in patients exhibiting minimal disease or those with GH-prolactin-cosecreting tumors but will not achieve hormone normalization in most patients. Somatostatin analogs effectively suppress GH and IGF-I in most patients, but intolerance (e.g. diarrhea, cramping, gallstones) can occur. Pegvisomant, the newest therapeutic option, blocks GH action at peripheral receptors, normalizes IGF-I levels, reduces signs and symptoms, and corrects metabolic defects. Pegvisomant does not appear to affect tumor size and has few adverse effects. Pegvisomant is the most effective drug treatment for acromegaly in normalizing IGF-I and producing a clinical response; it is the preferred agent in patients resistant to or intolerant of somatostatin analogs. (J Clin Endocrinol Metab 88: 4759 -4767, 2003)
Consensus statement: medical management of acromegaly
European Journal of Endocrinology, 2005
In November 2003, the Pituitary Society and the European Neuroendocrine Association sponsored a consensus workshop in Seville to address challenging issues in the medical management of acromegaly. Participants comprised 70 endocrinologists and neurosurgeons with international expertise in managing patients with acromegaly. All participants participated in the workshop proceedings, and the final document written by the scientific committee reflects the consensus opinion of the interactive deliberations. The meeting was supported by an unrestricted educational grant from Ipsen. No pharmaceutical representatives participated in the program planning or in the scientific deliberations. European Journal of Endocrinology 153 737-740 European Journal of Endocrinology (2005) 153 737-740
The place of medical treatment of acromegaly: current status and perspectives
Expert Opinion on Pharmacotherapy, 2013
The past two decades have brought significant advancement in treatment of acromegaly, primarily due to discovery of new medications and broadening of their availability. Accordingly, mortality rate of patients with acromegaly has decreased significantly and in adequately treated patients it approximates the general population. Treatment of acromegaly requires a combination of several modalities: neurosurgical operation, medical treatment and radiotherapy. Treatment of acromegaly in Serbia is conducted in accordance to international guidelines and recommendations. Recent availability of novel medical therapies for acromegaly in Serbia, demanded a need for creation of recommendations for optimal application of these new treatment options. Medical treatment of acromegaly encompasses three groups of drugs: 1) somatostatin receptor ligands, 2) dopamine agonists, 3) GH receptor antagonists. Every patient requires individual approach in treatment selection, usually involving a combination of multiple treatment modalities. Considerable cost of some medications, even in the economically developed countries, poses an additional challenge for the endocrinologist responsible for selection of treatment. Individualized approach to treatment ensures better disease control, reduction or elimination of comorbidities and reduction in cost of treatment of this chronic disease.
Acromegaly: current therapies benefits and burdens
Clinical Practice, 2018
Acromegaly is a rare disease most often caused by the prolonged secretion of excess growth hormone from a pituitary adenoma. The disease is associated with multiple significant comorbidities and increased mortality. The delay to diagnosis is often long. This may be because of low disease awareness among health care professionals, the insidious onset of differentiating features, and because patients are likely to present with complaints typical of other conditions more frequently seen in primary care. Early identification of acromegaly facilitates prompt treatment initiation and may minimize the permanent effects of excess growth hormone. The primary treatment for many patients will be pituitary surgery, although not all patients will be eligible for surgery or achieve a surgical cure. If biochemical control is not achieved following surgery, other treatment options include medical therapy and radiation therapy. Improved biochemical control may only alleviate rather than reverse the associated comorbidities. Thus, lifelong monitoring of patient health is needed, with particular attention to the management of cardiovascular risk factors. It is additionally important to consider the impact of both disease and treatment on patients' quality of life and minimize that impact where possible, but particularly for chronic therapies. For the majority of patients, chronic therapy is likely to include somatostatin analog injections. In some circumstances, it may be possible to extend the dosing interval of the analog once good biochemical control is achieved. Additional convenience may be gained from the possibility of self-/partner administration of treatment or administration of treatment by a health care professional at home. Overall, it is clear that the care of patients with acromegaly requires a highly coordinated approach involving numerous specialties (eg, endocrinology, surgery, cardiology). Further, patients' needs must be at the core of management and every effort must be made to improve health care experiences and minimize treatment burdens.
Pharmacological management of acromegaly: a current perspective
Neurosurgical FOCUS, 2010
Acromegaly is a chronic disorder of enhanced growth hormone (GH) secretion and elevated insulin-like growth factor–I (IGF-I) levels, the most frequent cause of which is a pituitary adenoma. Persistently elevated GH and IGF-I levels lead to substantial morbidity and mortality. Treatment goals include complete removal of the tumor causing the disease, symptomatic relief, reduction of multisystem complications, and control of local mass effect. While transsphenoidal tumor resection is considered first-line treatment of patients in whom a surgical cure can be expected, pharmacological therapy is playing an increased role in the armamentarium against acromegaly in patients unsuitable for or refusing surgery, after failure of surgical treatment (inadequate resection, cavernous sinus invasion, or transcapsular intraarachnoid invasion), or in select cases as primary treatment. Three broad drug classes are available for the treatment of acromegaly: somatostatin analogs, dopamine agonists, an...
Endocrinología y Nutrición (English Edition), 2011
Background and objectives: The reported efficacy of treatments for acromegaly varies depending on reference centers and national registries. The aim of this study was to describe clinical management of this disease and to assess the efficacy of treatments used in standard clinical practice. Material and methods: An epidemiological, observational, longitudinal, multicenter study was performed in adult patients with newly diagnosed acromegaly (n = 74) seen by 38 Spanish endocrinologists who collected during routine clinic visits data on disease treatment and control during 2 years of follow-up. Results: Pituitary surgery and treatment with somatostatin analogs were the first choice therapies in 76% and 24% of patients, respectively, with no differences related to tumor size. ଝ Please cite this article as: Luque-Ramírez M, et al. Estudio OASIS: manejo terapéutico de la acromegalia en un escenario de práctica clínica habitual. Evaluación de la eficacia de las diversas estrategias de tratamiento aplicadas. Endocrinol Nutr. 2011;58:478---86. ARTICLE IN PRESS +Model 2 M. Luque-Ramírez et al.
Current therapies and mortality in acromegaly
Journal of Medicine and Life, 2015
Acromegaly is a rare disease most frequently due to a GH secreting pituitary adenoma. Without an appropriate therapy, life of patients with acromegaly can be shortened with ten years. Pituitary surgery is usually the first line therapy for GH secreting pituitary adenomas. A meta-analysis proved that mortality is much lower in operated patients, even uncured, than the entire group of patients and is similar with the general population in patients with GH<1 μg/ L. For the patients with hypersecreting postoperative remnant tumor, those with low chance of surgical cure or with life-threatening comorbidities, medical therapies are available: somatostatin receptor analogues (SRA), dopamine agonists (DA) and GH receptor antagonists. Studies with >30% utilization of SRAs reported a lower mortality ratio than studies with lower percentages of SRA administration. Although therapy with DA has long been used in patients with acromegaly, there are no studies reporting its effect on mortali...
Review of current and emerging treatment options in acromegaly
The Netherlands journal of medicine, 2015
In almost every patient, acromegaly is caused by a growth hormone secreting pituitary adenoma. Clinical features are the result of excessive growth hormone secretion and the consecutive excess in insulin-like growth factor I levels. This results in somatic overgrowth and metabolic disturbances with a higher morbidity and mortality than in the general population. With optimal disease management, mortality can be reduced to that seen in the general population. The current treatment of acromegaly is based on a combination of surgery, radiotherapy and medical therapy. This review provides an overview of the current and upcoming therapies with a focus on medical therapy.