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Peptide receptor radionuclide therapy as a new tool in treatment-refractory sarcoidosis - initial experience in two patients

机译:肽受体放射性核素疗法作为治疗难治性结节病的新工具-两名患者的初步经验

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摘要

Sarcoidosis is a multisystem granulomatous disorder of unknown etiology that can involve virtually all organ systems. Whereas most patients present without symptoms, progressive and disabling organ failure can occur in up to 10% of subjects. Somatostatin receptor (SSTR)-directed peptide receptor radionuclide therapy (PRRT) has recently received market authorization for treatment of SSTR-positive neuroendocrine tumors.>Methods: We describe the first case series comprising two patients with refractory multi-organ involvement of sarcoidosis who received 4 cycles of PRRT.>Results: PRRT was well-tolerated without any acute adverse effects. No relevant toxicities could be recorded during follow-up. Therapy resulted in partial response accompanied by a pronounced reduction in pain (patient #1) and stable disease regarding morphology as well as disease activity (patient #2), respectively.>Conclusion: Peptide receptor radionuclide therapy in sarcoidosis is feasible and might be a new valuable tool in patients with otherwise treatment-refractory disease. Given the long experience with and good tolerability of PRRT, further evaluation of this new treatment option for otherwise treatment-refractory sarcoidosis in larger patient cohorts is warranted.
机译:结节病是一种病因不明的多系统肉芽肿性疾病,实际上可能涉及所有器官系统。尽管大多数患者没有症状,但多达10%的受试者会发生进行性和致残性器官衰竭。生长抑素受体(SSTR)指导的肽受体放射性核素治疗(PRRT)最近已获得市场许可,用于治疗SSTR阳性神经内分泌肿瘤。>方法:我们描述了第一例病例,其中包括两名难治性多发性多结节病的器官受累接受了4个PRRT周期。>结果:PRRT具有良好的耐受性,没有任何急性不良反应。随访期间没有相关毒性记录。该疗法导致部分反应,并伴随着形态和疾病活动性方面的疼痛显着减轻(患者1)和疾病稳定(患者2)。>结论:结节病中的肽受体放射性核素治疗是可行的,并且可能是治疗难治性疾病患者的新的有价值的工具。考虑到PRRT的长期经验和良好的耐受性,因此有必要对该更大的患者队列中治疗难治性结节病的新治疗方法进行进一步评估。

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