Response after treatment with pembrolizumab in a patient with myelophthisis due to melanoma: The role of checkpoint inhibition in the bone Journal Article


Authors: Rosner, S.; Sen, F.; Postow, M.
Article Title: Response after treatment with pembrolizumab in a patient with myelophthisis due to melanoma: The role of checkpoint inhibition in the bone
Abstract: Background: Myelophthisis due to melanoma is a rare phenomenon. Treatment strategies for patients with this serious complication of malignancy have not been well documented, and none have previously reported efficacy of immune checkpoint inhibition. Since bone metastases are not measurable lesions per standard response criteria, the efficacy of immune checkpoint inhibition in the bones is also not well described. Case presentation: We describe a patient with widespread melanoma metastases involving the bone marrow causing myelophthisis and pancytopenia who responded to immune checkpoint inhibition with the anti-programmed cell death-1 (PD-1) inhibitor pembrolizumab. Conclusions: This is the first report to our knowledge of disease response to immune checkpoint inhibition in a patient with myelophthisis. Clinical trials have recently emerged describing the efficacy of PD-1 inhibition for disorders regularly involving the bone marrow, such as hematologic malignancies, suggesting the importance of better understanding the bone marrow as an immunologically active compartment. Clinicians should be aware that immune checkpoint inhibition alone may be effective in treating malignancy involving the bone marrow, even in cases of extensive involvement resulting in pancytopenia due to myelophthisis from a solid tumor as our case suggests. © 2017 The Author(s).
Keywords: immunohistochemistry; adult; human tissue; middle aged; case report; solid tumor; systemic therapy; sentinel lymph node biopsy; melanoma; computer assisted tomography; bone marrow; hemoglobin; fever; immune response; thrombocyte count; cell cycle checkpoint; gene inactivation; pancytopenia; liver injury; programmed death 1 receptor; vitiligo; local excision; melanoma metastasis; human; male; priority journal; article; pembrolizumab; pd-1 inhibitor; immune checkpoint inhibition; myelophthisis; spinal cord atrophy
Journal Title: Journal for ImmunoTherapy of Cancer
Volume: 5
Issue: 1
ISSN: 2051-1426
Publisher: Biomed Central Ltd  
Date Published: 2017-04-18
Start Page: 34
Language: English
DOI: 10.1186/s40425-017-0236-3
PROVIDER: scopus
PUBMED: 28428883
PMCID: PMC5394614
DOI/URL:
Notes: Article -- Export Date: 2 June 2017 -- Source: Scopus
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  1. Michael Andrew Postow
    361 Postow
  2. Filiz   Sen
    26 Sen