Delphi consensus recommendations for a treatment algorithm in pulmonary sarcoidosis

Rahaghi, Franck; Baughman, Robert P.; Saketkoo, Lesley Ann; Sweiss, Nadera J.; Barney, Joseph B.; Birring, Surinder S.; Costabel, Ulrich; Crouser, Elliott D.; Drent, Marjolein; Gerke, Alicia K.; Grutters, Jan C.; Hamzeh, Nabeel; Huizar, Isham; James, William; Kalra, Sanjay; Kullberg, Susanna; Li, Huiping; Lower, Elyse E.; Maier, Lisa A.; Mirsaeidi, Mehdi; Müller–Quernheim, Joachim; Porquera, Eva M. Carmona; Samavati, Lobelia; Valeyre, Dominique; Scholand, Mary Beth

doi:10.1183/16000617.0146-2019

Cited by 107 publications

(95 citation statements)

References 63 publications

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“…The authors participated in a Delphi expert consensus panel to develop consensus recommendations for phenotyping and appropriate therapies for different phenotypes. The design and results of the modified Delphi consensus process are presented elsewhere in this issue of the European Respiratory Review [21]. The Delphi group included 26 panellists.…”

Section: Phenotyping For Treatment Decisionsmentioning

confidence: 99%

Clinical phenotyping: role in treatment decisions in sarcoidosis

2020

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A variety of phenotypic categorisations have been developed for sarcoidosis. Phenotyping has been used for genetics studies and to guide treatment selection. The authors participated in a Delphi expert consensus panel to develop a proposed phenotype categorisation and treatment recommendations for pulmonary sarcoidosis patients. Panellists reached consensus that asymptomatic patients with normal pulmonary function and adenopathy alone or normal chest imaging do not require therapy, while symptomatic patients with impaired pulmonary function or infiltrates should be treated. The panel did not reach consensus on asymptomatic patients with abnormal chest imaging or reduced pulmonary function, or symptomatic patients with normal chest imaging and pulmonary function. The proposed phenotype categories and associated treatment recommendations are asymptomatic (no therapy), acute (disease duration <1–2 years, apparently self-limited, corticosteroids), chronic (antimetabolites and other second-line therapies) and advanced (biologics). Some clinical settings, such as dyspnoea/hypoxaemia at rest, severely impaired or rapidly decreasing pulmonary function tests, and severe cardiac, neurologic, ocular or renal involvement warrant immediate therapy.

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Section: Phenotyping For Treatment Decisionsmentioning

confidence: 99%

Clinical phenotyping: role in treatment decisions in sarcoidosis

2020

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“…Although RCI has been approved to treat inflammatory and autoimmune diseases for more than 60 years, recent progress in understanding melanocortin receptors and the effects of RCI in modulating immune responses has led to increased interest in RCI as a therapeutic choice [25]. The role of RCI in the overall treatment algorithm is considered in the article on the treatment algorithm for sarcoidosis in this issue of the European Respiratory Review [26]. RCI was considered a "fourth-line" therapy for patients who were not able to take "third-line" treatments such as infliximab or other anti-TNF therapies.…”

Section: Discussionmentioning

confidence: 99%

“…The companion articles in this issue of the European Respiratory Review, which concern phenotyping of sarcoidosis [36] and development of a treatment algorithm for sarcoidosis using the Delphi process [26], seem to suggest that the use of RCI, when considered, should be mainly for the advanced disease phenotype, where concomitant therapy with steroids and immunosuppressants has not successfully controlled the condition.…”

Section: Discussionmentioning

confidence: 99%

Management of repository corticotrophin injection therapy for pulmonary sarcoidosis: a Delphi study

et al. 2020

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In patients treated with repository corticotrophin injection (RCI) for pulmonary sarcoidosis, effective management of adverse events may improve adherence. However, management of adverse events may be challenging due to limitations in real-world clinical experience with RCI and available published guidelines.We surveyed 12 physicians with a modified Delphi process using three questionnaires. Questionnaire 1 consisted of open-ended questions. Panellists' answers were developed into a series of statements for Questionnaires 2 and 3. In these, physicians rated their agreement with the statements using a Likert scale.Key consensus recommendations included a starting dose of 40 units twice a week for patients with less severe disease, continued at a maintenance dose for patients who responded, particularly those with chronic refractory sarcoidosis. Panellists reached consensus that concomitant steroids should be quickly tapered in patients receiving RCI, but that concomitant use of immunosuppressive medications should be continued. Panellists developed consensus recommendations for adverse event management, and reached consensus that RCI should be down-titrated or discontinued if other interventions for the adverse effects fail or if the adverse effect is severe.In the absence of clinical evidence, our Delphi consensus opinions may provide practical guidance to physicians on the management of RCI to treat pulmonary sarcoidosis.

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“…Treatment of pulmonary sarcoidosis should be considered for patients with significant pulmonary symptoms and patients with an impaired or deteriorating lung function [ 3 , 4 ]. The ATS/ERS/WASOG guideline, dating from 1999, recommends oral corticosteroids as the first-choice therapy for pulmonary sarcoidosis and this is also reflected in a recent Delphi consensus statement and current practice [ 5 – 7 ]. These recommendations are mainly based on expert opinion and limited evidence from observational studies.…”

Section: Introductionmentioning

confidence: 99%

“…These recommendations are mainly based on expert opinion and limited evidence from observational studies. Both state that more research is needed to define the best treatment for patients with pulmonary sarcoidosis [ 5 – 7 ]. Although corticosteroid treatment leads to short-term improvement of pulmonary function, radiological improvement, and symptom reduction, previous studies have not conclusively demonstrated a beneficial effect in preventing disease progression in the long-term [ 3 , 4 , 7 ].…”

Section: Introductionmentioning

confidence: 99%

Design of a randomized controlled trial to evaluate effectiveness of methotrexate versus prednisone as first-line treatment for pulmonary sarcoidosis: the PREDMETH study

Kahlmann¹,

Janssen

Moor³

et al. 2020

BMC Pulm Med

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Background Treatment of pulmonary sarcoidosis is recommended in case of significant symptoms, impaired or deteriorating lung function. Evidence-based treatment recommendations are limited and largely based on expert opinion. Prednisone is currently the first-choice therapy and leads to short-term improvement of lung function. Unfortunately, prednisone often has side-effects and may be associated with impaired quality of life. Methotrexate is presently considered second-line therapy, and appears to have fewer side-effects. Objective The primary objective of this trial is to investigate the effectiveness and tolerability of methotrexate as first-line therapy in patients with pulmonary sarcoidosis compared with prednisone. The primary endpoint of this study will be the change in hospital-measured Forced Vital Capacity (FVC) between baseline and 24 weeks. Secondary objectives are to gain more insights in response to therapy in individual patients by home spirometry and patient-reported outcomes. Blood biomarkers will be examined to find predictors of response to therapy, disease progression and chronicity, and to improve our understanding of the underlying disease mechanism. Methods/design In this prospective, randomized, non-blinded, multi-center, non-inferiority trial, we plan to randomize 138 treatment-naïve patients with pulmonary sarcoidosis who are about to start treatment. Patients will be randomized in a 1:1 ratio to receive either prednisone or methotrexate in a predefined schedule for 24 weeks, after which they will be followed up in regular care for up to 2 years. Regular hospital visits will include pulmonary function assessment, completion of patient-reported outcomes, and blood withdrawal. Additionally, patients will be asked to perform weekly home spirometry, and record symptoms and side-effects via a home monitoring application for 24 weeks. Discussion This study will be the first randomized controlled trial comparing first-line treatment of prednisone and methotrexate and provide valuable data on efficacy, safety, quality of life and biomarkers. If this study confirms the hypothesis that methotrexate is as effective as prednisone as first-line treatment for sarcoidosis but with fewer side-effects, this will lead to improvement in care and initiate a change in practice. Furthermore, insights into the immunological mechanisms underlying sarcoidosis pathology might reveal new therapeutic targets. Trial registration The study was registered on the 19th of March 2020 in the International Clinical Trial Registry, www.clinicaltrials.gov; ID NCT04314193.

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Delphi consensus recommendations for a treatment algorithm in pulmonary sarcoidosis

Cited by 107 publications

References 63 publications

Clinical phenotyping: role in treatment decisions in sarcoidosis

Clinical phenotyping: role in treatment decisions in sarcoidosis

Management of repository corticotrophin injection therapy for pulmonary sarcoidosis: a Delphi study

Design of a randomized controlled trial to evaluate effectiveness of methotrexate versus prednisone as first-line treatment for pulmonary sarcoidosis: the PREDMETH study

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