Xiaofeng Luo

Thrombotic thrombocytopenic purpura (TTP) is a rare but fatal disease requiring urgent diagnosis. The PLASMIC scoring model has been reported to be a valuable model for identifying TTP. This study aimed to investigate the diagnostic accuracy of this model and the diagnostic utility of lactate dehydrogenase (LDH)-to-serum creatinine (sCr) ratio in identifying TTP in Chinese patients. The records of 61 patients with suspected TTP tested for ADAMTS13 activity in our hospital between June 2016 and April 2024 were retrospectively analyzed. Receiver operating characteristic (ROC) analysis was performed to evaluate the diagnostic accuracy of LDH, sCr, and the LDH-to-sCr (LDH/sCr) in predicting TTP. Multivariate logistic regression analysis was used to screen for independent risk factors of TTP, and a combined predictive algorithm was established. The AUC for the PLASMIC scoring model distinguishing TTP from non-TTP was 0.850 (optimal cutoff: 5), with 92.8% sensitivity, 76.5% specificity, 75.8% positive predictive value (PPV), and 92.9% negative predictive value (NPV). The AUC derived from the LDH/sCr ratio was higher than that derived from LDH or sCr (P < 0.05). A combined predictive algorithm, termed the LCRP algorithm (including the LDH/sCr ratio, reticulocyte percentage, and platelet count), was developed for TTP. The AUC (0.955) derived from the LCRP algorithm, with 96.3% sensitivity, 88.2% specificity, 86.7% PPV, and 96.8% NPV, was higher than that of the PLASMIC scoring model (P < 0.05). The LDH/sCr ratio is more effective than isolated LDH or sCr measurements for predicting TTP. The LCRP algorithm involving the LDH/sCr ratio showed superior predictive performance and may hold significant potential for early identification of Chinese patients with TTP.

Two of the main toxicities linked with chimeric antigen receptor T (CAR-T) cell therapy are cytokine release syndrome (CRS) and immune effector cell associated neurotoxicity syndrome (ICANS). It is believed that an inflammatory factor storm resulting in organ dysfunction is the fundamental pathophysiology, and this has a significant impact on the survival rate following CART cell therapy. While steroids and other IL-6R antagonists like Tocilizumab are useful treatment approaches, not all patients benefit from them. Following CD19-targeted CART cell infusion, a 3-year-old kid with relapsed refractory B-cell acute lymphoblastic leukemia (ALL) started to have CRS on Day 1. Despite receiving steroids and tocilizumab early on, his illness worsened and eventually led to multiple organ failure, particularly heart failure. After receiving mesenchymal stem cells (MSCs) as a rescue treatment on Days 17 and 20, the patient's clinical symptoms were stabilized by Day 25. Negative microscopic residual disease (MRD) was achieved with CART therapy, and CAR expression was maintained in peripheral blood until Day 75. The case implies that MSC may be a useful treatment for multi-organ dysfunction that results from severe CRS and ICANS brought on by CART cell therapy.

The relapse rate remains high after chemotherapy for adult patients with acute lymphoblastic leukemia (ALL). With better molecular diagnosis and classification as well as better assessment for minimal residual disease, major progress in the treatment for refractory and/or relapsed ALL is being made. In addition to the tyrosine kinase inhibitors (TKIs) for Philadelphia chromosome-positive ALL, immunotherapeutic agents, blinatumomab, inotuzumab ozogamicin (INO), and chimeric antigen receptor (CAR) T cells, are changing the treatment paradigm for ALL. Blinatumomab and INO are being incorporated into induction chemotherapy regimens and combined with TKIs for ALL therapy. A novel low-intensity regimen, miniHCVD-INO-blinatumomab, appears to be less toxic and more effective than conventional intensive chemotherapy regimens. This review summarized new therapeutic researches of ALL and updated latest progress in clinical trials on bispecific antibodies, antibody-drug conjugates, and new regimens incorporating these novel antibodies.

A multicenter retrospective study in 131 patients (44 females/87 males) with hematological disorders who underwent G-CSF-primed/haplo-identical (Haplo-ID) (n = 76) or HLA-identical (HLA-ID) HSCT (n = 55) from February 2013 to February 2016 was conducted to compare the incidence and risk factors for pre-engraftment bloodstream infection (PE-BSI). In the Haplo-ID group, 71/76 patients with high-risk (n = 28) or relapsed/refractory hematological malignancies (n = 43) received FA5-BUCY conditioning (NCT02328950). All received trimethoprim–sulfamethoxazole (TMP–SMX) prophylaxis. Blood cultures and catheter tip cultures were obtained to confirm the BSI. PE-BSI was detected in 24/131 HSCT patients (18/76 in Haplo-ID and 6/55 in HLA-ID) after 28 febrile neutropenic episodes. Among 28 isolates for the 24 patients, 21 (75%) were G^neg bacteria, 6 (21.4%) G^pos and 1 (3.6%) fungi. Bacteria sources were central venous line infection (7/29.2%), gastroenteritis (6/25%), lower respiratory tract infection (LRTI; 5/20.8%), perianal skin infection (4/16.7%), and unknown (2/8.3%). The duration of neutropenia (P = 0.046) and previous G^neg bacteremia (P = 0.037) were important risk factors by univariate analysis, while the type of HSCT was not. A trend of TMP–SMX-resistant BSI in both groups may be due to routine antibacterial prophylaxis strategies. Our data show that G-CSF-primed Haplo-ID HSCT did not increase the risk of PE-BSI, even with intensive immunosuppressive treatments.