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  • Abstract: Autoimmune x
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J Gebauer Department of Internal Medicine I, University Hospital of Schleswig-Holstein, Campus Luebeck and Institute for Endocrinology and Diabetes, University of Luebeck, Luebeck, Germany

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R Skinner Department of Paediatric and Adolescent Haematology and Oncology and Children’s BMT Unit, Great North Children’s Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, and Translational and Clinical Research Institute, Newcastle University Centre for Cancer, Newcastle University, Newcastle upon Tyne, UK

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R Haupt DOPO Clinic, Department of Hematology/Oncolgy, IRCCS Istituto Giannina Gaslini, Genova, Italy

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L Kremer Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands
Amsterdam UMC, Emma’s Children’s Hospital, Amsterdam, The Netherlands

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H van der Pal Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands

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G Michel Department of Health Sciences and Medicine, University of Lucerne, Luzern, Switzerland

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G T Armstrong Department of Epidemiology and Cancer Control, St. Jude Children’s Research Hospital, Memphis, Tennessee, USA

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M M Hudson Department of Oncology, St. Jude Children’s Research Hospital, Memphis, Tennessee, USA

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L Hjorth Department of Clinical Sciences Lund, Paediatrics, Lund University, Skane University Hospital, Lund, Sweden

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H Lehnert Paris Lodron University of Salzburg, Salzburg, Austria

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T Langer Pediatric Hematology and Oncology, University Hospital of Schleswig-Holstein, Campus Luebeck, Luebeck, Germany

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Many long-term childhood cancer survivors suffer from treatment-related late effects, which may occur in any organ and include a wide spectrum of conditions. Long-term follow-up (LTFU) is recommended to facilitate early diagnosis and to ensure better health outcomes. Due to the heterogeneity of these sequelae, different specialists work together in the diagnosis and treatment of these conditions. Experts from both pediatric and internal medicine are involved in age-appropriate care by providing a transition process. Hence, LTFU of childhood cancer survivors is a prototypic example of multidisciplinary care for patients with complex needs treated in a specialized setting. International collaborations of healthcare professionals and scientists involved in LTFU of childhood cancer survivors, such as the International Guideline Harmonization Group, compile surveillance recommendations that can be clinically adopted all over the world. These global networks of clinicians and researchers make a joint effort to address gaps in knowledge, increase visibility and awareness of cancer survivorship and provide an excellent example of how progress in clinical care and scientific research may be achieved by international and multidisciplinary collaboration.

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Hanna Karhapää Medical Faculty, University of Helsinki, Helsinki, Finland
Department of Oncology, Comprehensive Cancer Centre, Helsinki University Hospital, Helsinki, Finland

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Siru Mäkelä Medical Faculty, University of Helsinki, Helsinki, Finland
Department of Oncology, Comprehensive Cancer Centre, Helsinki University Hospital, Helsinki, Finland

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Hanna Laurén Medical Faculty, University of Helsinki, Helsinki, Finland
Department of Radiology, HUS Medical Imaging Centre, University of Helsinki and Helsinki University Hospital, Helsinki, Finland

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Marjut Jaakkola Medical Faculty, University of Helsinki, Helsinki, Finland
Department of Radiology, HUS Medical Imaging Centre, University of Helsinki and Helsinki University Hospital, Helsinki, Finland

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Camilla Schalin-Jäntti Medical Faculty, University of Helsinki, Helsinki, Finland
Endocrinology, Abdominal Centre, University of Helsinki and HUS, Helsinki, Finland

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Micaela Hernberg Medical Faculty, University of Helsinki, Helsinki, Finland
Department of Oncology, Comprehensive Cancer Centre, Helsinki University Hospital, Helsinki, Finland

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Objective

Immune checkpoint inhibitors (ICI) can cause endocrine adverse events. However, endocrine AEs could be related to better treatment outcomes. Our aim was to investigate whether this holds true in a real-world setting of metastatic melanoma patients.

Design

A retrospective single-institution study.

Methods

We included 140 consecutive metastatic melanoma patients treated with ICI between January 2012 and May 2019. We assessed the endocrine toxicity and the best possible treatment outcomes from electronic patient records, including laboratory parameters and radiological images.

Results

Of the treated patients, 21 patients (15%) were treated with ipilimumab, 46 (33%) with nivolumab, 67 (48%) with pembrolizumab, and 6 (4%) with combination therapy (ipilimumab + nivolumab). Endocrine AEs appeared in 29% (41/140) patients. Three patients had two different endocrine AEs. Thyroid disorders were the most common: 26% (36/140), followed by hypophysitis: 4% (5/140). Three subjects (2%, 3/140) were diagnosed with autoimmune diabetes. Three patients had to terminate treatment due to endocrine toxicity. Radiological manifestations of endocrine AEs were found in 16 patients (39%, 16/41). Endocrine toxicity was associated with significantly better treatment outcomes. Median progression-free survival (8.1 months, range 5.1–11.1 months vs 2.7 months, range 2.4–3.0 months, P < 0.001), and median overall survival (47.5 months, range 15.5–79.5 months vs 23.7 months, range 15.3–32.1 months, P = 0.035) were longer for patients experiencing endocrine AEs.

Conclusions

The higher number of endocrine AEs suggest that regular laboratory monitoring aids in AE detection. Endocrine AEs in metastatic melanoma may correlate with better treatment outcomes.

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