Acromegaly, a syndrome of excess growth hormone (GH) secretion typically caused by a GH-secreting pituitary adenoma, reduces life expectancy by approximately 10 years when left untreated. Treatment of acromegaly involves combinations of one or more discrete therapeutic modalities to achieve biochemical control. Unfortunately, data capable of informing decisions among alternate management strategies are presently lacking.
The authors performed a comparative effectiveness research (CER) review integrating efficacy, cost, and quality of life (QOL) analysis for treatment strategies comprising various combinations of surgery, radiotherapy, stereotactic radiosurgery, and pharmacotherapy in patients with acromegaly caused by a pituitary microadenoma. A management decision tree was used to identify 5 treatment strategies, each with up to 4 potential treatment steps. Efficacy was assessed using recent literature reports of biochemical control rates for each modality. Cost estimations were derived from wholesale drug prices and from the Healthcare Cost and Utility Project. Quality of life data were obtained from studies utilizing the Acromegaly Quality of Life Questionnaire.
Individual treatment modalities were analyzed and ranked in each of 3 domains: highest rate of success, lowest cost, and highest QOL, and these scores were combined to facilitate comparison of overall effectiveness of each of the management strategies. These aggregate effectiveness scores were used to compare the 5 strategies from the decision tree, and a novel strategy was also proposed.
The choice of management strategy must be individualized for each patient with acromegaly. This CER analysis provides a comprehensive framework to inform clinical decisions among alternate management strategies in patients with GH-secreting pituitary microadenomas.
Abbreviations used in this paper:AcroQoL = Acromegaly Quality of Life Questionnaire; AWP = average wholesale price; CER = comparative effectiveness research; GH = growth hormone; HCUP = Healthcare Cost and Utility Project; IGF-1 = insulin-like growth factor–1; LAR = long-acting repeatable; SRS = stereotactic radiosurgery.
BarkanALHalaszIDornfeldKJJaffeCAFribergRDChandlerWF: Pituitary irradiation is ineffective in normalizing plasma insulin-like growth factor I in patients with acromegaly. J Clin Endocrinol Metab82:3187–31911997
BarrandeGPittino-LungoMCosteJPonvertDBertagnaXLutonJP: Hormonal and metabolic effects of radiotherapy in acromegaly: long-term results in 128 patients followed in a single center. J Clin Endocrinol Metab85:3779– 37852000
BevanJSAtkinSLAtkinsonABBoulouxPMHannaFHarrisPE: Primary medical therapy for acromegaly: an open, prospective, multicenter study of the effects of subcutaneous and intramuscular slow-release octreotide on growth hormone, insulin-like growth factor-I, and tumor size. J Clin Endocrinol Metab87:4554–45632002
BiermaszNRvan ThielSWPereiraAMHoftijzerHCvan HemertAMSmitJW: Decreased quality of life in patients with acromegaly despite long-term cure of growth hormone excess. J Clin Endocrinol Metab89:5369–53762004
BuchfelderMWeigelDDrosteMMannKSallerBBrübachK: Pituitary tumor size in acromegaly during pegvisomant treatment: experience from MR re-evaluations of the German Pegvisomant Observational Study. Eur J Endocrinol161:27–352009
ChansonPBorson-ChazotFKuhnJMBlumbergJMaisonobePDelemerB: Control of IGF-I levels with titrated dosing of lanreotide Autogel over 48 weeks in patients with acromegaly. Clin Endocrinol (Oxf)69:299–3052008
ColaoAFeroneDMarzulloPCappabiancaPCirilloSBoerlinV: Long-term effects of depot long-acting somatostatin analog octreotide on hormone levels and tumor mass in acromegaly. J Clin Endocrinol Metab86:2779–27862001
Fallon Community Health Plan: Lanreotide (Somatuline Depot). Prior Authorization Approval CriteriaWorcester, MAFallon Community Health Plan2008. (http://www.fchp.org/~/media/Files/FCHP/Imported/SomatulineDepot_lanreotide.pdf.ashx) [Accessed April 25 2012]
Fallon Community Health Plan: Octreotide (Sandostatin). Prior Authorization Approval CriteriaWorcester, MAFallon Community Health Plan2008. (http://www.fchp.org/~/media/Files/FCHP/Imported/Sandostatin_octreotide.pdf.ashx) [Accessed April 25 2012]
JalladRSMusolinoNRKodairaSCescatoVABronsteinMD: Does partial surgical tumour removal influence the response to octreotide-LAR in acromegalic patients previously resistant to the somatostatin analogue?. Clin Endocrinol (Oxf)67:310–3152007
JenkinsPJBatesPCarsonMNStewartPMWassJA: Conventional pituitary irradiation is effective in lowering serum growth hormone and insulin-like growth factor-I in patients with acromegaly. J Clin Endocrinol Metab91:1239–12452006
MattaMPCoutureECazalsLVezzosiDBennetACaronP: Impaired quality of life of patients with acromegaly: control of GH/IGF-I excess improves psychological subscale appearance. Eur J Endocrinol158:305–3102008
MinnitiGTraishDAshleySGonsalvesABradaM: Risk of second brain tumor after conservative surgery and radiotherapy for pituitary adenoma: update after an additional 10 years. J Clin Endocrinol Metab90:800–8042005
NeggersSJde HerderWWJanssenJAFeeldersRAvan der LelyAJ: Combined treatment for acromegaly with long-acting somatostatin analogs and pegvisomant: long-term safety for up to 4.5 years (median 2.2 years) of follow-up in 86 patients. Eur J Endocrinol160:529–5332009
NeggersSJvan AkenMOde HerderWWFeeldersRAJanssenJABadiaX: Quality of life in acromegalic patients during long-term somatostatin analog treatment with and without pegvisomant. J Clin Endocrinol Metab93:3853–38592008
NeggersSJvan AkenMOJanssenJAFeeldersRAde HerderWWvan der LelyAJ: Long-term efficacy and safety of combined treatment of somatostatin analogs and pegvisomant in acromegaly. J Clin Endocrinol Metab92:4598–46012007
NicolucciAMaioneAFranciosiMAmorettiRBusettoECapaniF: Quality of life and treatment satisfaction in adults with Type 1 diabetes: a comparison between continuous subcutaneous insulin infusion and multiple daily injections. Diabet Med25:213–2202008
RowlesSVPrietoLBadiaXShaletSMWebbSMTrainerPJ: Quality of life (QOL) in patients with acromegaly is severely impaired: use of a novel measure of QOL: acromegaly quality of life questionnaire. J Clin Endocrinol Metab90:3337–33412005
SchreiberIBuchfelderMDrosteMForssmannKMannKSallerB: Treatment of acromegaly with the GH receptor antagonist pegvisomant in clinical practice: safety and efficacy evaluation from the German Pegvisomant Observational Study. Eur J Endocrinol156:75–822007
Walgreens Health Initiatives: Volume 7 Number 3 March 2003 (http://www.springsgov.com/units/empservices/Benefits_2003/Walgreens_News_Mar03.pdf) [Accessed May 1 2012]
WebbSMBadiaXSurinachNL: Spanish AcroQoL Study Group: Validity and clinical applicability of the acromegaly quality of life questionnaire, AcroQoL: a 6-month prospective study. Eur J Endocrinol155:269–2772006