Outcomes of adrenal-sparing surgery or total adrenalectomy in phaeochromocytoma associated with multiple endocrine neoplasia type 2: an international retrospective population-based study.

FREDERIC CASTINETTI,, ; XIAO-PING QI,; MARTIN K WALZ,; ANA LUIZA MAIA,; GABRIELA SANSÓ; MARIOLA PECZKOWSKA, , ; KORNELIA HASSE-LAZAR; THERA P LINKS,; SARKA DVORAKOVA,, ; RODRIGO A TOLEDO; CATERINA MIAN; MARIA JOAO BUGALHO; NELSON WOHLLK, ; OLEG KOLLYUKH,; LETIZIA CANU, ; PAOLA LOLI,; SIMONA R BERGMANN, , , ; JOSEFI NA BIARNES COSTA; OZER MAKAY; ATTILA PATOCS; MARIJA PFEIFER,; NALINI S SHAH, ; THOMAS CUNY,; MICHAEL BRAUCKHOFF; BIRKE BAUSCH, , ; ERNST VON DOBSCHUETZ; CLAUDIO LETIZIA,; MARCIN BARCZYNSKI; MARIA K ALEVIZAKI, ,; MALGORZATA CZETWERTYNSKA; M UMIT UGURLU; , GERLOF VALK; JOHN T M PLUKKER, ; PAOLA SARTORATO,; DEBORA R SIQUEIRA,; MARTA BARONTINI; MALGORZATA SZPERL,; BARBARA JARZAB; HANS H G VERBEEK; TOMAS ZELINKA; PETR VLCEK , ; SERGIO P A TOLEDO,; FLAVIA L COUTINHO, ; MASSIMO MANNELLI; MONICA RECASENS; LEA DEMARQUET, ; ,LUIGI PETRAMALA,; SVETLANA YAREMCHUK; DMITRY ZABOLOTNYI,; FRANCESCA SCHIAVI, ; GIUSEPPE OPOCHER,; KAROLY RACZ,; ANDRZEJ JANUSZEWICZ, ; GEORGES WERYHA; JEAN-FRANCOIS HENRY,;

LANCET

ELSEVIER SCIENCE INC

Lugar: Amsterdam; Año: 2014 vol. 15 p. 648 - 655

0140-6736

Background The prevention of medullary thyroid cancer in patients with multiple endocrine neoplasia type 2 syndromehas demonstrated the ability of molecular diagnosis and prophylactic surgery to improve patient outcomes. However,the other major neoplasia associated with multiple endocrine neoplasia type 2, phaeochromocytoma, is not as wellcharacterised in terms of occurrence and treatment outcomes. In this study, we aimed to systematically characterisethe outcomes of management of phaeochromocytoma associated with multiple endocrine neoplasia type 2.Methods This multinational observational retrospective population-based study compiled data on patients withmultiple endocrine neoplasia type 2 from 30 academic medical centres across Europe, the Americas, and Asia.Patients were included if they were carriers of germline pathogenic mutations of the RET gene, or were fi rst-degreerelatives with histologically proven medullary thyroid cancer and phaeochromocytoma. We gathered clinicalinformation about patients? RET genotype, type of treatment for phaeochromocytoma (ie, unilateral or bilateraloperations as adrenalectomy or adrenal-sparing surgery, and as open or endoscopic operations), and postoperativeoutcomes (adrenal function, malignancy, and death). The type of surgery was decided by each investigator and thetiming of surgery was patient driven. The primary aim of our analysis was to compare disease-free survival aftereither adrenal-sparing surgery or adrenalectomy.Findings 1210 patients with multiple endocrine neoplasia type 2 were included in our database, 563 of whom hadphaeochromocytoma. Treatment was adrenalectomy in 438 (79%) of 552 operated patients, and adrenal-sparingsurgery in 114 (21%). Phaeochromocytoma recurrence occurred in four (3%) of 153 of the operated glands afteradrenal-sparing surgery after 6?13 years, compared with 11 (2%) of 717 glands operated by adrenalectomy (p=0·57).Postoperative adrenal insuffi ciency or steroid dependency developed in 292 (86%) of 339 patients with bilateralphaeochromocytoma who underwent surgery. However, 47 (57%) of 82 patients with bilateral phaeochromocytomawho underwent adrenal-sparing surgery did not become steroid dependent.Interpretation The treatment of multiple endocrine neoplasia type 2-related phaeochromocytoma continues to rely onadrenalectomies with their associated Addisonian-like complications and consequent lifelong dependency onsteroids. Adrenal-sparing surgery, a highly successful treatment option in experienced centres, should be the surgicalapproach of choice to reduce these complications.