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. 2018 Oct 1;21(5):1189–1198. doi: 10.1038/s41436-018-0297-9

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© The Author(s) 2018

Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/.

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Fig. 1 — CONSORT flow-chart and schematic of nonrandomized open-label pilot study. a Schematic of number of participants assessed for eligibility and excluded or allocated to the study, treated, followed, and analyzed. Of the 39 subjects enrolled, 30 completed 26 weeks of sirolimus therapy, and 23/30 had anatomy that permitted analysis of the primary outcome measure. Safety and tolerability were evaluated in all treated participants. b Overview of study design including schedule of procedures. AE/SAEs adverse events/serious adverse events, CXR chest X-ray, DXA/MRI dual energy X-ray absorptiometry/magnetic resonance imaging, QOL quality of life.