In the era of chimeric antigen receptor T-cell (CAR-T) therapy, the prognostic impact of bulky disease in relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL) remains unclear, prompting this study that enrolled 27 patients with bulky disease and 27 with non-bulky disease to compare clinical outcomes. Over the follow‑up period, patients with non‑bulky disease achieved a higher overall response rate (77.8% vs. 70.4%) and better one‑year progression‑free survival (PFS) and overall survival (OS), with bulky disease significantly associated with worse OS (P = 0.040) and a median OS of 12 months versus 24 months in non‑bulky patients. Multivariable Cox regression identified autologous hematopoietic stem cell transplantation combined with CAR‑T (ASCT + CAR‑T) as an independent variable, and this combination was correlated with superior PFS (HR = 7.363; 95% CI: 2.653–20.437; P < 0.001) and OS (HR = 0.208; 95% CI: 0.070–0.615; P < 0.05) compared to CAR‑T alone, irrespective of bulky disease status. Collectively, these findings indicate that R/R DLBCL patients with bulky disease have worse outcomes after CAR‑T therapy than those without, and that ASCT + CAR‑T represents a promising alternative for such patients regardless of bulky disease presence.
Background Polatuzumab vedotin, an antibody-drug conjugate targeting CD79b, has become the standard of care (SOC) for both treatment-naive (TN) and relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL) patients, administered via the Pola-R-CHP (rituximab, cyclophosphamide, doxorubicin, prednisone) and Pola-BR (bendamustine plus rituximab) regimens. Unlike the strict inclusion criteria of clinical trials, real-world patients often present with poorer baseline conditions and higher disease burdens, with treatment modalities individualized based on their disease characteristics. Thus, evaluating the efficacy and safety of Pola-based regimens in real-world settings is of critical importance. Based on this, we retrospectively analyzed consecutive patients treated with Pola-based regimens at our center to investigate their disease characteristics, treatment patterns, and clinical outcomes. Methods This retrospective study enrolled consecutive patients from Changhai Hospital Affiliated to Naval Military Medical University who received Pola-based regimens. Efficacy was evaluated via whole-body 18F-FDG PET/CT or contrast-enhanced CT scans during and after treatment. Some patients were considered for autologous stem cell transplantation (ASCT) as consolidation therapy. Treatment responses were assessed using the 2014 Lugano criteria, and adverse events (AEs) were graded according to the NCI-CTCAE v5.0. Efficacy endpoints included complete response (CR) rate, objective response rate (ORR), safety profile, progression-free survival (PFS), and overall survival (OS). Results A total of 30 patients were enrolled between October 2024 and August 2025. The overall baseline characteristics indicated a high-risk profile: median age was 68.5 years (range, 46–81 years); 4 patients (13.3%) had transformed follicular lymphoma (tFL), and 2 (6.7%) had T-cell/histiocyte-rich large B-cell lymphoma (THRLBCL). Twenty-four patients (80.0%) were staged Ann-Arbor III–IV, and 16 (53.3%) were classified as high-risk by the International Prognostic Index (IPI). Additional high-risk factors included extranodal involvement (25 patients, 83.3%), double-expressor lymphoma (DEL, 17 patients, 56.7%), double-hit/triple-hit lymphoma (DH/TH, 5 patients, 16.7%), and high P53 expression (11 patients, 36.7%). Regarding treatment history: 14 patients (46.7%) were previously untreated, and 16 (53.3%) had received at least one prior line of therapy. Among the latter, 5 had early relapse after their last treatment, 6 were refractory, and 1 had prior exposure to ASCT and chimeric antigen receptor T-cell therapy (CAR-T). To date, the median number of Pola-based regimen cycles administered was 2 (range, 1–6). Ten patients were evaluable for efficacy, with 5 achieving CR and 3 achieving partial response (PR); 3 patients (10%) proceeded to ASCT afterward. In this high-risk population, Pola-based regimens showed manageable toxicity, with 37.5% of patients experiencing grade 3–4 AEs. The most common AEs were anemia (100.0%), decreased white blood cell count (41.7%), and decreased neutrophil count (41.7%), most of which were grade 1–2. The most frequent grade 3–4 AE was anemia (25.0%). With a median follow-up of 3.8 months (95% CI: 5.7–8.9), only 2 patients progressed: one was CD5-positive, and the other had high P53 expression. Conclusion Our results characterize the real-world disease features, treatment patterns, and outcomes of DLBCL patients with high-risk prognostic factors, treated with Pola-based regimens. Even in this high-risk population, Pola-based therapy demonstrated promising efficacy and manageable toxicity. However, detailed data on response rates and survival outcomes require further reporting following extended follow-up.
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative strategy for patients with chronic myelomonocytic leukemia (CMML). However, few reports have investigated the outcomes of patients receiving haploidentical HSCT. To this end, we included 117 patients with haploidentical donors (HID) and 75 patients with matched related donors (MRD) from 28 centers across China to explore the prognostic impact of different transplantation modalities. We found no significant difference between these two groups in terms of event-free survival (EFS, p = .211), overall survival (OS, p = .503), cumulative incidence of relapse (CIR, p = .076) or non-relapse mortality (NRM, p = .794). The predominance of peripheral blood (PB) graft source over bone marrow and PB since 2020 may have contributed to the worse outcomes in the MRD group. Moreover, CMML-specific prognostic scoring system (CPSS) lower-risk patients benefited more from the HID modality with superior EFS (p = .006). Multivariate analysis indicated that advanced age (p = .013), anemia at diagnosis (p = .010), and donor relationship (parent-to-child, p = .013) were independently associated with worse EFS in the HID group. Our data suggested that HID was comparable to MRD in CMML. However, under certain conditions, such as CPSS lower-risk ones, HID was preferred.
Background: Inotuzumab ozogamicin(INO) is an antibody-drug conjugate for CD22-positive acute B-cell lymphoblastic leukemia(B-ALL). INO has been available in China since June 2022, however, it lacks published data of INO from China. Herein, we present the clinical outcomes of INO-based treatment for adult B-ALL patients (pts) in our real-world study from Shanghai Acute Lymphoblastic Leukemia (SH-ALL) Research Group. Methods: In this retrospective real-world multi-center study, B-ALL pts treated by INO-based therapy within 8 centers of SH-ALL Research Group were analyzed. Pts received INO monotherapy or INO-based combined therapy. All Ph positive (Phpos) ALL pts received TKI. CD22 positivity is defined as CD22 expression ≥20% by multiparameter flow cytometry (MFC). Efficacy evaluation was assessed after 1 cycle of INO-based therapy. Minimal residual disease (MRD) is routinely monitored by MFC with a sensibility of 10-4. Adverse events (AEs) were assessed according to CTCAE v5.0. Results: From June 2022 to July 2025, a total of 102 pts with B-ALL received INO-based therapy in 8 centers in Shanghai, among whom 93 pts with available data were analyzed. The median age was 57 years old (range, 17-77). There were 23 (24.7%) Phpos pts and 70(75.3%) Ph negative (Phneg) pts. Before INO initiation, 3 pts were newly diagnosed (ND), 9 pts were on CR (3 MRD negative, 6 MRD positive) and 81(87.1%) pts were in relapsed/refractory (R/R) status. Among 70 Phneg pts, 21 pts received INO+venetoclax (VEN). Among 23 Phpos pts, besides TKI+INO combination, 12 pts received VEN additionally. Once CR or MRD negativity reached, eligible pts received allogeneic hematopoietic stem cell transplantation (allo-HSCT). After 1 cycle, CR rate and MRD negative rate were both 100% in 3 ND Phneg pts treated by INO+VEN. Among 6 MRD positive CR pts (3 Phneg and 3 Phpos), all pts but 1 Phneg pt with TP53 mutation reached MRD negativity (83.3%). For 81 R/R pts, 65(80.2%) pts reached CR. In 65 R/R pts who achieved CR, 63 pts had evaluable MRD, the MRD negative rate was 77.8% (49/63). Among all R/R pts, there were 61 Phneg and 20 Phpos pts. There was no significant difference neither in CR rate (82.0% vs 75.0%, p=0.526) nor in MRD negative rate (75.0% vs 86.7%, p=0.486) between Phneg and Phpos pts. In 81 R/R pts, 26 received INO+VEN regimen, in whom the CR rate was higher than that of pts received INO + other combination (92.3% vs 74.5%, p=0.07). The MRD negative rate of INO+VEN was also higher than INO + other combination (86.7% vs 75.0%, p=0.215). Up to August 2025, the median follow-up time was 6 months, median Overall Survival (OS) was not reached. The 6-months OS was 83.6% (95%CI 75.8%~92.3%). All 3 ND pts are alive in persistent remission. As for 9 CR pts, only 1 pt with persistent MRD after INO treatment died of disease progression. Among 81 R/R pts, the 6-months OS was 83.2% (95%CI 74.9%~92.4%), 60 Phneg pts had significant better survival than 21 Phpos pts (87.2% vs 69.4%, p=0.0473). The safety profiles of the INO-based therapy were acceptable. Non-hematological AEs were primarily G1-2, reversible rapidly after symptomatic management. Veno-occlusive disease (VOD) occurred in 4 pts: 2 pts during INO treatment, and 2 pts after allo-HSCT. Most of the G3-4 AEs were hematological AEs, especially thrombocytopenia. No treatment-related mortality was observed. Conclusion Here, we first reported the real-world multi-center outcomes of INO-based treatment from SH-ALL research group. In this large cohort, we confirmed the efficacy and safety of INO in B-ALL pts with different tumor burdens. The combination of INO+VEN showed higher CR rate and MRD negative rate. These findings will be further validated through a prospective study.
Acute myeloid leukaemia(AML)is characterized mainly by an increase in the number of myeloid cells in the bone marrow and a decrease in the number of mature cells;AML accounts for 28%of leukaemia cases,and it has a five-year survival rate of only 30.5%[1].
Hematologic adverse events (AEs) are common and serious toxicities in patients with hematologic malignancies undergoing blinatumomab therapy. However, restrictive selection criteria in pivotal clinical trials can lead to an underestimation of rare but fatal toxicities. In this study, we systematically analyzed hematologic AEs associated with blinatumomab using the Food and Drug Administration Adverse Event Reporting System (FAERS) from October 2014 to December 2023. Disproportionate analysis was performed to identify overreported AEs, with a reporting odds ratio (ROR), and a lower bound of the 95% confidence interval (ROR025) exceeding one considered significant. Additionally, adjusted mortality rates and risk ratios (RR) of the top 10 reported hematologic AEs were calculated using a logistic regression model. Among 4745 blinatumomab-related cases, 418 (8.81%) involved hematologic AEs. We identified 22 significantly overreporting hematologic AEs compared to the full database, with myelosuppression (n = 39 [9.33%], ROR025 = 8.04), disseminated intravascular coagulation (DIC, n = 31 [7.42%], ROR025 = 15.14), and bone marrow failure (n = 14 [3.35%], ROR025 = 3.41) notably underestimated in clinical trials. DIC resulted in a substantial mortality rate of 45.16%. Finally, DIC was found to be independently associated with death in a multivariable logistic regression analysis (RR = 2.47 [95% CI: 1.11-3.83]). These findings could aid clinicians in the early detection of these rarely reported but fatal hematologic AEs, thereby reducing the risk of severe toxicities in blinatumomab recipients.
AbstractBackgroundTo explore the effects of monitoring measurable residual disease and post‐remission treatment selection on the clinical outcomes of B‐cell acute lymphoblastic leukemia (B‐ALL) in adults.MethodsBetween September 2010 and January 2022, adult patients with B‐ALL who received combination chemotherapy, with or without allogeneic hematopoietic stem cell transplantation (allo‐HSCT), were included in the retrospective study, which was approved by the Ethics Committee and the observation of Declaration of Helsinki conditions.ResultsOne hundred and forty‐three B‐ALL patients achieved complete remission (CR) were included in the study, of whom 94 patients (65.7%) received allo‐HSCT in first complete remission (CR1).Multivariate analysis showed that the most powerful factors affecting OS were transplantation (hazard ratio [HR] = 0.540, p = 0.037) and sustained measurable residue disease (MRD) negativity (HR = 0.508, p = 0.037). The subgroup analysis showed that the prognosis of the allo‐HSCT group was better than that of the chemotherapy group, regardless of whether MRD was negative or positive after two courses of consolidation therapy. After consolidation therapy, the prognosis of patients with positive MRD remained significantly better in the allo‐HSCT group than in the chemotherapy group. However, no significant difference was observed in the prognosis between the allo‐HSCT and chemotherapy groups with negative MRD after consolidation therapy.ConclusionsB‐ALL patients who achieve sustained MRD negativity during consolidation therapy have excellent long‐term outcomes even without allo‐HSCT. Allo‐HSCT is associated with a significant benefit in terms of OS and DFS for patients who were with positive MRD during consolidation therapy.
The preferred donor (haploidentical donor [HID] versus matched unrelated donor [URD]) choice in patients with acquired severe aplastic anemia (SAA) who lack an HLA-matched sibling donor (MSD) and fail upfront immunosuppressive treatment (IST) therapy is unknown. We retrospectively investigated SAA patients (n = 58) who underwent allogeneic stem cell transplantation (allo-SCT) between January 2012 and October 2022. The 5-year overall survival (OS) and 5-year failure-free survival (FFS) were comparable among the URD (n = 8), HID (n = 25), and MSD (n = 25) cohorts (OS: mean, 87.5 ± 11.7% versus 98.0 ± 6.5% versus 83.3 ± 7.6% [P = .926]; FFS: mean, 60.0 ± 18.2% versus 87.0 ± 7.0% versus 78.3 ± 8.6% [P = .222]). Multivariate analysis revealed that primary engraftment failure independently predicted OS and secondary graft failure predicted FFS among SAA patients who underwent allo-SCT, but donor type and age were not predictive of these outcomes. An urgent second SCT for patients with engraftment failure may be an effective salvage treatment. Our findings show that an alternative donor SCT is indicated for eligible SAA patients without an MSD even if age ≥40 years.
Autologous stem cell transplantation (ASCT) is a salvage therapy for relapsed or refractory diffuse large B-cell lymphoma (DLBCL). We have developed a novel conditioning regimen called CEAC (oral semustine 250 mg/m2 d-6, etoposide 300 mg/m2 d-5 d-2, cytarabine 500 mg/m2 d-5 d-2, and cyclophosphamide 1200 mg/m2 d-5 d-2) In lymphoma patients in China. Here, we conducted a study to compare the conventional BEAM regimen with the CEAC regimen in 110 DLBCL patients. Propensity-score matching was performed in a 1:4 ratio (22 patients received BEAM and 88 received CEAC). Our results showed no significant difference in the overall response rate (95
Pediatric-inspired chemotherapy significantly improves survival for adolescent and adult patients with acute lymphoblastic leukemia (ALL). However, the benefits over allogeneic hematopoietic stem cell transplantation (allo-HSCT) remain unclear. To compare clinical outcomes between pediatric-inspired chemotherapy and allo-HSCT in consolidation therapy of adolescent and adult Philadelphia chromosome-negative (Ph-neg) ALL in first complete remission (CR1), related studies from MEDLINE, Embase, and Cochrane Controlled Register of Trials updated to July 2022 were searched. A total of 13 relevant trials including 3161 patients were included in the meta-analysis. Compared with allo-HSCT, pediatric-inspired chemotherapy achieved better OS (hazard risk (HR), 0.53; 95% confidence interval (CI), 0.41 to 0.68) and DFS (HR, 0.64; 95% CI, 0.48 to 0.86), with a significant reduction in NRM (risk ratio (RR), 0.30; 95% CI, 0.18 to 0.51), but no difference in the relapse rate (RR, 1.13; 95% CI, 0.93 to 1.39). When only studies based on intention-to-treat analysis were included, pediatric-inspired chemotherapy consistently conferred a survival advantage. In subgroup analyses, patients with baseline high-risk features demonstrated similar OS and DFS between pediatric-style chemotherapy and allo-HSCT, while pediatric-style chemotherapy had an OS and DFS advantage in standard-risk subgroup. Particularly, patients with positive minimal residual disease (MRD) achieved better OS and DFS if proceeded to allo-HSCT.
Chronic myelomonocytic leukemia (CMML) is a clonal hematopoietic stem cell malignancy, and allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only curable treatment. The outcomes after transplant are influenced by both disease characteristics and patient comorbidities. To develop a novel prognostic model to predict the post-transplant survival of CMML patients, we identified risk factors by applying univariable and multivariable Cox proportional hazards regression to a derivation cohort. In multivariable analysis, advanced age (hazard ratio [HR] 3.583), leukocyte count (HR 3.499), anemia (HR 3.439), bone marrow blast cell count (HR 2.095), and no chronic graft versus host disease (cGVHD; HR 4.799) were independently associated with worse survival. A novel prognostic model termed ABLAG (Age, Blast, Leukocyte, Anemia, cGVHD) was developed and the points were assigned according to the regression equation. The patients were categorized into low risk (0-1), intermediate risk (2, 3), and high risk (4-6) three groups and the 3-year overall survival (OS) were 93.3% (95%CI, 61%-99%), 78.9% (95%CI, 60%-90%), and 51.6% (95%CI, 32%-68%; p < .001), respectively. In internal and external validation cohort, the area under the receiver operating characteristic (ROC) curves of the ABLAG model were 0.829 (95% CI, 0.776-0.902) and 0.749 (95% CI, 0.684-0.854). Compared with existing models designed for the nontransplant setting, calibration plots, and decision curve analysis showed that the ABLAG model revealed a high consistency between predicted and observed outcomes and patients could benefit from this model. In conclusion, combining disease and patient characteristic, the ABLAG model provides better survival stratification for CMML patients receiving allo-HSCT.
Background: Extranodal involvement is recognized as a poor prognostic factor for diffuse large B-cell lymphoma (DLBCL). However, the prognostic differences of patients with refractory/relapsed (R/R) nodal and extranodal DLBCL in the chimeric antigen receptor T cell (CART) therapy era are still unclear. Materials and methods: In this study, 18 R/R nodal DLBCL (R/R N-DLBCL) and 19 R/R extranodal DLBCL (R/R EN-DLBCL) were enrolled to compare clinical outcomes. Results: The median follow-up time was 13 (range, 1-47) months and one-year progression-free survival (PFS; 83.3% vs. 42.1%, P = 0.008) and one-year overall survival (OS; 94.4% vs. 63.2%, P = 0.020) were significantly different between nodal and extranodal patients. In the multivariable Cox regression analysis, R/R EN-DLBCL was associated with worse PFS (hazard ratio [HR] = 4.263, P = 0.018) and OS (HR = 9.589, P = 0.034) compared to R/R N-DLBCL. Additionally, autologous hematopoietic stem cell transplantation (ASCT) combined with CART therapy (ASCT + CART) was correlated with better PFS (HR = 0.164, P = 0.003) compared to CART treatment alone. Conclusions: The clinical outcomes of R/R EN-DLBCL were worse than R/R N-DLBCL in patients receiving CART therapy and ASCT + CART therapy is a promising alternative treatment for patients with R/R EN-DLBCL.
Hematotoxicity is the most common long-term adverse event after chimeric antigen receptor T cell (CAR-T) therapy. Here, a total of 71 patients with relapsed or refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL) or large B-cell lymphoma (LBCL) were used to develop an early hematotoxicity predictive model and verify the accuracy of this model. The incidences of early hematotoxicity at 3 month following CAR-T infusion in B-ALL and LBCL were 45.5% and 38.5%, respectively. Multivariate analyses revealed that the severity of cytokine release syndrome (CRS) was an independent risk factor affecting early hematotoxicity. The analysis between the peak cytokine levels and early hematotoxicity suggested that tumor necrosis factor-α (TNF-α) and C-reactive protein (CRP) were closely associated with early hematotoxicity. Then, an early predictive model of hematotoxicity was constructed based on the peak contents of TNF-α and CRP. This model could diagnose early hematotoxicity with positive predictive values of 87.7% and 85.0% in training and validation cohorts, respectively. Lastly, we constructed the nomogram for clinical practice to predict the risk of early hematotoxicity, which performed well compared with the observed probability. This early predictive model is instrumental in the risk stratification of CAR-T recipients with hematotoxicity and early intervention for high-risk patients.
Background Acute myeloid leukemia (AML) is the most common acute leukemia in adults, with a median age of 68 in clinical diagnosis. About 60% patients are over 60 years old. There are various treatment options for AML patients. But for elderly patients, the complete remission rates are disappointing due to genetic, molecular, and age-related factors. Development of next-generation sequencing technologies makes it possible to seek individual strategies for patients in different ages. This study analyzed transcriptome profiles in platelets of AML patients in different ages for the first time. Methods Platelet RNA sequencing in AML of ten elderly and seven young patients were performed with Illumina TruSeq Stranded mRNA library Prep Kit and Illumina HiSeq4000 sequencing instrument. With the FASTQ sequencing data obtained, statistical analyses between elderly with young AML patients were analyzed by R program. GO and KEGG enrichment analyses were performed via R package clusterProfiler. TOP 10 down-regulated/up-regulated genes in elderly patients compared to young patients were selected with the threshold of |L2FC| > 2 and padj ≤ 0.0001. The down-regulated gene ATF4 was chosen by GSEA analysis and ROC analysis with AUC > 0.95. Results We found 3059 genes with differential transcript levels (GDTLs) in AML patients of different age. Among them, 2048 genes are down-regulated and 651 genes are up-regulated in elderly patients. We found that gene transcript profiles in elderly patients is obviously different from those in young patients, including a collection of down-regulated genes related to proteins processing in endoplasmic reticulum and immunity. We further identified that genes of pathway in cancer and mitogen activated protein kinase (MAPK) pathway, involved in natural immunity and metabolism, are significantly down-regulated in elderly patients. Among all screened genes with decreased transcript levels, we believe that activating transcription factor 4 (ATF4) is a biomarker indicating different chemotherapy strategies for elderly patients. Conclusions In summary, gene transcript profiles are different in platelets of elderly and young AML patients. And ATF4 can be a useful biomarker indicating different chemotherapy strategies for AML patients with different ages.
Abstract Adult patients with relapsed or refractory T‐cell acute lymphoblastic leukemia (R/R‐T‐ALL) have extremely poor prognosis, representing an urgent unmet medical need. Finding an optimal salvage regimen to bridge transplantation is a priority. The CAG (cytarabine, aclarubicin, and G‐CSF) regimen was initially used by one group in China, showing unexpectedly promising results in 11 R/R‐T‐ALL patients. Here, we report the multicenter results of 41 patients who received the CAG regimen as salvage therapy. After one cycle of the CAG regimen, complete remission and partial remission were achieved in 33 (80.5%) and two (4.9%) patients, respectively. Failure to respond was observed in six patients (14.6%). Early T‐cell precursor (ETP) (n = 26) and non‐ETP (n = 15) patients had a similar CR rate (80.8% vs 80.0%, P = .95). Among 41 patients, allo‐HSCT was successfully performed in 27 (66%) patients (22 in CR and 5 in non‐CR). With a median follow‐up time of 12 months, the estimated 2‐year overall survival and event‐free survival were 68.8% (95% CI, 47.3%‐83.0%) and 56.5% (95% CI, 37.1%‐71.9%), respectively. The CAG regimen was well‐tolerated, and no early death occurred. Our multicenter results show that the CAG regimen is highly effective and safe, representing a novel choice for adult patients with R/R‐T‐ALL and providing a better bridge to transplantation.
In this retrospective study we assessed the efficacy and safety of tocilizumab in patients with critical or severe coronavirus disease 2019 (COVID-19). We enrolled 181 patients admitted to Huoshenshan Hospital (Wuhan, China) with confirmed COVID-19 between January 2020 and February 2020. Ninety-two patients were treated with tocilizumab, and 89 patients were treated conventionally. We analyzed the clinical manifestations, changes in CT scan images, and laboratory tests before and after tocilizumab treatment, and compared these results with the conventionally treated group. A significant reduction in the level of C-reactive protein was observed 1 week after tocilizumab administration. In some cases this meant the end of the IL-6-related cytokine storm. In addition, tocilizumab relieved fever, cough, and shortness of breath with no reported adverse drug reactions. These findings suggest tocilizumab improves clinical outcomes and is effective for treatment of patients with critical or severe COVID-19. However, future clinical trials are needed to better understand the impact of tocilizumab interference with IL-6 and provide a therapeutic strategy for treatment of COVID-19.
目的:提高对急性未分化型白血病(AUL)的认识。方法:分析海军军医大学附属长海医院收治的1例AUL患者诊治经过,并复习国内外相关文献。结果:该患者骨髓流式细胞术免疫表型结果为缺乏T系和髓系特异性标志,无B系特异性标志,同时也缺乏巨核细胞和浆细胞样树突细胞等系列特异性标志,诊断为AUL,经DA+VP方案诱导化疗后血象恢复,后失访。结论:目前对AUL仍没有标准的诊断和治疗指导,有必要收集更多病例,以便提高对该病的认识,指导临床治疗。
Objective: To compare the difference of efficacy between traditional Hyper-CVAD/MA regimen and the adolescents inspired chemotherapy regimen, CH ALL-01, in treatment of adult Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph(+) ALL) . Methods: In this study we retrospectively analyzed 158 Ph(+) ALL patients receiving Hyper-CVAD/MA regimen (n=63) or CHALL-01 regimen (n=95) in our center and Changzheng hospital from January 2007 to December 2017, excluding patients with chronic myeloid leukemia in blast crisis. Tyrosine kinase inhibitor (TKI) was administered during induction and consolidation chemotherapy. Patients who underwent hematopoietic stem cell transplantation received TKI as maintenance therapy. Results: Of them, 91.1% (144/158) patients achieved complete remission (CR) after 1-2 courses of induction. CR rate was 90.5% (57/63) for patients in Hyper-CVAD/MA group and 91.6% (87/95) for patients in CHALL-01 group. There was no difference in CR rates between the two groups (χ(2)=0.057, P=0.811) . The last follow-up was June 2018. A cohort of 134 CR patients could be used for further analysis, among them, 53 patients received Hyper-CVAD/MA regimen and other 81 patients received CHALL-01 regimen. The molecular remission rates were significantly higher in CHALL-01 group (complete molecular response: 44.4%vs 22.6%; major molecular response: 9.9% vs 18.9%) (χ(2)=7.216, P=0.027) . For the patients in Hyper-CVAD/MA group, the 4-year overall survival (OS) was 44.81% (95%CI: 30.80%-57.86%) and the 4-year disease free survival (DFS) was 37.95% (95%CI: 24.87%-50.93%) . For patients received CHALL-01 regimen, the 4-year OS was 55.63% (95%CI: 39.07%-69.36%) (P=0.037) and 4 year DFS was 49.06% (95%CI: 34.24%-62.29%) (P=0.015) , while there was no significant difference in 4 year cumulative incidence of relapse (CIR) (P=0.328) or cumulative incidence of nonrelapse mortality (CI-NRM) (P=0.138) . The rate of pulmonary infection was lower in patients received CHALL-01 regimen compared with patients received Hyper-CVAD regimen (43.4% vs 67.9%, χ(2)=7.908, P=0.005) . Conclusions: Outcome with CHALL-01 regimen appeared better than that with the Hyper-CVAD/MA regimen in Ph(+) ALL, which has lower incidence of pulmonary infection, higher molecular remission rate and better OS and DFS.