Albert Einstein College of Medicine, 1300 Morris Park Ave, Bronx, NY 10461 USA.
Memorial Sloan Kettering Cancer Center, 1275 York Ave, New York, NY 10065 USA.
J Immunother Cancer. 2017 Apr 18;5:34. doi: 10.1186/s40425-017-0236-3. eCollection 2017.
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.
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.
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.
由黑色素瘤引起的骨髓浸润是一种罕见现象。对于这种恶性肿瘤严重并发症的患者，尚未有明确的治疗策略，也没有先前关于免疫检查点抑制疗效的报道。由于骨转移不是根据标准反应标准测量的病变，因此免疫检查点抑制在骨骼中的疗效也描述得不够清楚。
我们描述了一例广泛的黑色素瘤转移累及骨髓导致骨髓浸润和全血细胞减少的患者，该患者对免疫检查点抑制剂抗程序性细胞死亡蛋白-1（PD-1）抑制剂 pembrolizumab 有反应。
据我们所知，这是首例骨髓浸润患者对免疫检查点抑制有疾病反应的报道。最近出现了一些临床试验，描述了 PD-1 抑制对经常涉及骨髓的疾病的疗效，如血液系统恶性肿瘤，这表明更好地理解骨髓作为一个免疫活跃的隔室的重要性。临床医生应该意识到，仅免疫检查点抑制可能对治疗骨髓受累的恶性肿瘤有效，即使在广泛受累导致骨髓浸润性全血细胞减少的情况下也是如此，正如我们的病例所表明的那样。
