[A case-control study of multiple myeloma patients with central nervous system involvement].

Objective: To investigate the treatment options for multiple myeloma patients with central nervous system involvement (CNS-MM) , as well as their clinical characteristics and prognostic factors. Methods: Between January 2011 and January 2022 our center diagnosed 18 people with CNS-MM. A retrospective analysis was done on the clinical information from the initial diagnosis and central nervous system involvement, and it was compared to 1∶3 matched newly diagnosed MM from the same period. Analysis was done on the clinical characteristics and survival rates of the two groups. Results: In patients with CNS-MM, the median time of onset was 14.2 (0.9-79.6) months and the median overall survival (OS) was 30.5 months from initial diagnosis and only 3.8 months in patients after CNS involvement. The CNS-MM patients showed more IgD type (P=0.010) , severer anemia (P=0.014) , a higher proportion of bone marrow plasma cells (P=0.013) , more extramedullary lesions (P=0.001) , and increased lactic dehydrogenase (LDH) (P=0.009) when compared to the control group. Lenalidomide or pomalidomide-based combinations had higher rates of hematology and CNS remission than bortezomib or daratumumab-based regimens (75.0% vs 16.7% , P=0.019) . Patients who received IMiD-based regimens and had 2 high-risk factors at initial diagnosis (high LDH and extramedullary lesions) had a significantly lower incidence of CNS-MM (P=0.026) . At the initial diagnosis, LDH (P=0.008, HR=7.319, 95% CI 1.663-32.219) and extramedullary lesions (P=0.006, HR=8.054, 95% CI 1.828-35.486) were independent risk factors for the occurrence of CNS-MM. Conclusion: Patients with CNS-MM had a poor prognosis. Patients with high LDH or extramedullary lesions at the time of the initial diagnosis are more likely to have CNS-MM. The prognosis of this patient may be improved by immunoregulator-based therapy.

[The efficacy and safety of daratumumab in relapsed and refractory multiple myeloma].

Objective: To investigate the efficacy and safety of daratumumab in relapsed and refractory multiple myeloma (RRMM) . Methods: The clinical characteristics, adverse reactions, efficacy, and prognosis of 46 patients with RRMM treated with daratumumab in Shanghai Changzheng Hospital from September 2017 to March 2020 were retrospectively analyzed. Results: All patients were treated with daratumumab-based regimen: 8 in the Dd group, 35 in the DRd group, and 3 in the DVd group. With a median follow-up of 9.6 months, the overall response rate (ORR) was 75% [complete remission (CR) rate 18.2% ] among the 44 patients available for evaluation. The ORRs of patients resistant to bortezomib, lenalidomide, and both were 70.6% , 69.2% , and 63.6% , respectively. The CR rates of patients resistant to bortezomib, lenalidomide, and both were 17.6% , 11.5% , and 13.6% , respectively. No significant difference was observed in ORR and CR rates among the three groups. The ORRs of the DRd, DVd, and Dd groups were 85.3% , 66.7% , and 28.6% , respectively (P=0.007) . The median PFS of 46 patients was 8.9 months, the median OS was not reached, and the 1-year OS rate was 74% . The median PFS and OS in the DRd group were longer than those in the Dd group (PFS: 14.4 months vs 2.0 months; OS: not reached vs 5.2 months) . After treatment with daratumumab, neutropenia is the most common hematological adverse reaction above grade 3. Non-hematological adverse reactions are mainly infusion-related adverse reactions and infections. Prognostic analysis showed that patients with extramedullary invasion had shorter PFS and OS compard with patients without extramedullary invasion (PFS: 5.7 vs 14.4 months, P=0.033; OS: 6.3 months vs not reached, P=0.029) . The OS of patients with an ECOG score of 3-4 was significantly shorter than patients with an ECOG score of 1-2 (5.9 months vs not reached, P=0.004) . Conclusion: Daratumumab-based regimens have good efficacy and safety in the treatment of RRMM.

[Clinical characteristics and prognosis of Waldenström's macroglobulinemia: a clinical data report from a single center].

Objective: To summarize the clinical characteristics and prognosis of 51 patients with Waldenström's macroglobulinemia (WM) and evaluate the efficacy and adverse reactions of ibrutinib in the treatment of WM. Methods: We carried out a single-center retrospective study, including 51 patients with WM of our single center from November 2008 to October 2019. Results: The median age at diagnosis was 65 years with a male-to-female ratio of 2.64∶1. There were 9 (18%) , 21 (41%) , and 21 (41%) ISSWM stage low-, intermediate- and high-risk patients identified, respectively. A total of 27 (73%) patients harbored MYD88(L265P) mutation. The median follow-up time was 38.6 (0.3-120.0) months, the median progression free survival was 46.4 months, and the median overall survival was not reached. The overall remission and major remission rates of patients who received ibrutinib were 87% and 80%, respectively. The median time to achieve at least partial remission of patients treated with ibrutinib was 8 weeks, which was earlier than those treated with other drugs (P<0.05) . Conclusion: WM is often seen in elderly men. MYD88(L265P) had a high frequency in WM. The findings of our study validate the efficacy of ibrutinib monotherapy. Even in patients with advanced age and at high risk of ISSWM, the overall remission rate and major remission rate are high. Ibrutinib is a safe and effective therapy because of its rapid onset and rare serious adverse reactions.