Waldenström巨球蛋白血症的一线治疗:基于荷兰国家指南的考虑

IF 0.9 Q4 HEMATOLOGY Hemato Pub Date : 2022-10-26 DOI:10.3390/hemato3040047
K. Amaador, M. Kersten, H. Visser, L. Nieuwenhuizen, R. Schop, M. Chamuleau, G. Velders, M. Minnema, J. Vos
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引用次数: 0

摘要

Waldenström巨球蛋白血症(WM)是一种罕见的b细胞非霍奇金淋巴瘤。只有很少的前瞻性随机临床试验来指导治疗建议,并且对于首选的一线治疗方法没有国际共识。在最近修订的荷兰WM指南中,我们描述了尽可能基于已知数据的实践建议。在这里,我们根据这些荷兰指南总结了一线治疗的注意事项。总结现有证据,包括疗效和毒性数据。利妥昔单抗联合化疗、蛋白酶体抑制或btk抑制都是有效的一线治疗选择。荷兰WM工作组认为,鉴于地塞米松/利妥昔单抗/环磷酰胺(DRC)方案的疗效、相对温和的毒性和广泛的经验,该方案适合许多WM患者的一线治疗。然而,刚果民主共和国的长期毒性尚不清楚,需要进一步澄清。其他方案,如r -苯达莫司汀,r -硼替佐米-地塞米松也是有效的选择,但具有特定的毒性。在荷兰WM指南中,由于需要长期治疗和毒性,btk抑制剂不是大多数患者的首选一线治疗方案。基于患者偏好研究,未来的临床试验应侧重于有效的固定疗程方案,并结合具有良好毒性的非细胞毒性疗法。BCL-2抑制剂、新型蛋白酶体抑制剂和btk抑制的进一步发展可能是有趣的。此外,t细胞定向治疗包括双特异性抗体作为单一疗法或与其他新型药物联合治疗值得在WM中进一步研究。
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First-Line Treatment of Waldenström’s Macroglobulinaemia: Considerations Based on the Dutch National Guideline
Waldenström macroglobulinemia (WM) is a rare B-cell Non-Hodgkin Lymphoma. There are only few prospective randomized clinical trials to guide treatment recommendations and there is no international consensus on a preferred first line treatment approach. In the recently revised Dutch guideline for WM, we describe recommendations for practice based as much as possible on the known data. Here, we summarize the considerations for first-line treatment based on these Dutch guidelines. Available evidence is summarized, including efficacy and toxicity data. Combinations of Rituximab with chemotherapy, proteasome inhibition or BTK-inhibition are all valid first line treatment options. The Dutch WM working group considers Dexamethasone/Rituximab/Cylofosfamide (DRC) a suitable first-line treatment for many WM patients, given the efficacy, the relatively mild toxicity profile and the extensive experience with this regimen. However, the long-term toxicities of DRC are unclear and need further clarification. Other regimens such as R-bendamustine, R-Bortezomib-dexamethason are also effective options, however with specific toxicities. BTK-inhibitors are not a preferred option in first line for most patients in the Dutch WM guidelines because of the need for longterm treatment and toxicities. Based on patient preferences research, future clinical trials should focus on effective fixed-duration regimens with non-cytotoxic therapies that have a favorable toxicity profile. Further development of (combinations with) BCL-2 inhibititors, novel proteasome inhibitors and BTK-inhibition could be interesting. In addition T-cell-directed treatments including bispecific antibodies as a monotherapy or combined with other novel agents deserve further study in WM.
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