National Medical Journal of China
Volume 102 · Issue 38 · 2022
Natl Med J China
Lower urinary tract dysfunction (LUTD) is the dysfunction of bladder and urethra during bladder filling and voiding phase. There are two general categories: neurogenic and non-neurogenic. It can cause upper urinary tract damage if left untreated. Urodynamic study (UDS) can objectively detect the function of detrusor and sphincter of urethra as well as predict the damage of LUTD to upper urinary tract. However, the development of UDS for children in China has not been popularized, which affects the accurate diagnosis and the treatment of LUTD. This article reviewed the importance of paying attention to LUTD and promoting the clinical application of UDS in children, so as to improve the diagnosis and treatment of LUTD in children.
Hepatic disease-related pulmonary vascular disease refers to the changes in the structure or function of pulmonary circulation caused by liver parenchymal or hepatic vascular diseases, mainly including hepatopulmonary syndrome characterized by hypoxemia, portopulmonary hypertension characterized by pulmonary hypertension, and hepatic vascular shunt characterized by increased cardiac output and pulmonary hypertension. Early diagnosis and treatment of liver disease and early evaluation of liver transplantation may significantly improve the prognosis for patients with hepatic disease-related pulmonary vascular disease. Moreover, it is necessary to improve clinicians′ understanding of this disease and strengthen multidisciplinary cooperation, improve the level of diagnosis and treatment.
To establish a predictive model for upper urinary tract damage in children with neurogenic bladder and verify its efficacy.
From January 2011 to December 2021, 143 children with NB in the Children′s Hospital of Chongqing Medical University and 84 children with NB in the First Affiliated Hospital of Zhengzhou University were selected as the research objects. The former is set as the training set and the latter is set as the validation set, and the general parameters of the two are compared. The independent risk factors of upper urinary tract damage in children with NB were screened out by Lasso regression, and multivariate logistic regression analysis and a nomogram prediction model was established. The models were validated internally and externally on the training set and validation set, respectively, and the area under the receiver operating curve (ROC) was used to verify the accuracy of the model.
A total of 227 children with NB were included in this study, including 121 males and 106 females, aged (10.2±3.8) years. There was no significant difference in other parameters except age between the training set and validation set (all P>0.05); Lasso regression and multivariate logistic regression analysis showed that detrusor leakage point pressure (DLPP) ≥ 40 cmH2O (OR=4.76, 95%CI: 2.01-11.26, 1 cmH2O=0.098 kPa), overactive bladder (OAB) (OR=3.08, 95%CI: 1.34-7.04), bladder compliance (BC)<20 ml/cm H2O (OR=3.65, 95%CI: 1.41-9.47), history of previous urinary tract infection (OR=2.73, 95%CI: 1.09-6.81), and abdominal pressure/other voiding patterns (OR=2.86, 95%CI: 1.20-6.82) were risk factors for upper urinary tract damage in children with NB (all P<0.05). The above parameters were used to establish a nomogram model of upper urinary tract damage in children with NB. The internal and external validation results show that the AUC values for the training and validation sets were 0.84 (95%CI: 0.77-0.91) and 0.86 (95%CI: 0.79-0.94), respectively.
The prediction model of upper urinary tract damage in children with NB constructed in this study has high discrimination, accuracy and clinical applicability, which can help clinicians identify high-risk patients and make individualized treatment design for these patients.
To evaluate the bladder function and sleep pattern in the children with primary mono-symptomatic nocturnal enuresis (PMNE) by the polysomnography (PSG) and ambulatory urodynamic monitoring (AUM).
From October 2019 to October 2021, forty-three patients with PMNE were selected as PMNE group from the First Affiliated Hospital of Zhengzhou University and further subdivided into the severe PMNE group (enuresis>4 times/week) and the non-severe PMNE group (enuresis times 4 times/week) according to the severity. The conventional urodynamics (CUD), AUM, and PSG examinations and bladder diary were completed in the PMNE group. The control group consisted of 23 children with normal PSG findings and without the lower urinary tract symptoms.
The severe PMNE group included 9 males and 14 females, aged(12.1±3.2)years, and nocturnal enuresis number per week is 6.7±1.7. The non-severe PMNE group included 9 males and 11 females, aged(12.0±3.4)years, and nocturnal enuresis number per week is 2.3±1.0. The incidences of nocturnal polyuria and the reduction in maximum bladder capacity in the PMNE group was 34.9% and 11.6%, respectively. The incidence and frequency of detrusor overactivity (DO) in the severe PMNE group were significantly higher than those in the non-severe PMNE group [78.3% vs 45.0%, (5.5±1.8) times/h vs (3.4±1.0) times/h, respectively, all P<0.05]. It was found by the PSG that the severe PMNE group had significantly higher cortical arousal index, apnea hypopnea index (AHI), and percentage of N1+N2 phase in total sleep time, compared with the control group[(58.6±9.8)% vs (49.3±9.5)%, (9.4±4.4) times/h vs (3.1±1.5) times/h, (2.7±0.9) times/h vs (0.9±0.7) times/h] (all P<0.05). While the sleep efficiency of the severe PMNE group was substantially lower than that of the non-severe PMNE group [(86.4±4.3)% vs (91.0±3.9)%], the cortical arousal index and AHI were significantly greater than those of the non-severe PMNE group[(9.4±4.4) times/h vs (5.7±3.2) times/h, (2.7±0.9) times/h vs (1.9±0.7) times/h] (all P<0.05). In the PMNE group, there were positive correlations between cortical arousal index and nocturnal DO frequency or AHI (r=0.705, 0.765, P=0.001).
Children with PMNE have nocturnal bladder dysfunction and abnormal sleep pattern, and there is a certain correlation between them. PSG and AUM are necessary for the evaluation and treatment of children with PMNE.
To analyze the video-urodynamic(VUDS) and clinical features of non-neuropathic lower urinary tract dysfunction (NNLUTD) in children.
Children diagnosed with NNLUTD in the First Affiliated Hospital of Zhengzhou University from January 2016 to December 2020 were included. Children with neurological, rectal dysfunction and anatomical abnormalities were excluded. VUDS and urinalysis were performed in all children who were divided into 4 groups accordingly: normal group (Normal group), detrusor overactivity group(DO group), detrusor sphincter dyssynergia group (DSD group) and detrusor underactivity group (DU group). VUDS and clinical features, vesicoureteral reflux (VUR) and urinary tract infections (UTI) were analyzed.
A total of 173 children were included in this study, including 103 males and 70 females, aged (7.2±3.3) years. VUDS showed that 46 cases (26.6%) were in Normal group, 63 cases (36.4%) in DO group, 39 cases (22.5%) in DSD group and 25 cases (14.5%) in DU group. Compared with Normal group and DO group, the proportion of VUR in DSD group and DU group was significantly higher [18(46.2%) and 11(44.0%) vs 7(15.0%) and 14(22.2%), all P<0.05],and the proportion of male children was significantly higher than that of female children with VUR only in DO group [12(32.4%) vs 2(7.7%), P=0.020 ]; Compared with DO group, the proportion of UTI in DSD group and DU group was significantly higher [16(41.0%) and 12(48.0%) vs 12(19.0%), all P<0.05], and the proportion of female children was significantly higher than that of male children with UTI in normal group, DO group and DU group [9(45.0%) vs 4(15.4%), 8(30.8%) vs 4(10.8%)and 7(87.5%) vs 5(29.4%), all P<0.05]. The maximum detrusor pressure in DSD group was significantly higher than that in Normal group, DO group and DU group [(95±47) vs (43±18), (56±18) and (12±9)cmH2O, all P<0.05, 1 cmH2O=0.098 kPa).Compared with Normal group and DO group, post void residual in DSD group and DU group was significantly increased [(58±38) and (70±62) vs (8±8) and (8±7)ml, all P<0.05], and the proportion of lower bladder compliance was significantly increased [(15(38.5%) and 11(44%) vs 1(2.2%) and 10(15.9%), all P<0.05]. Compared with normal group, the maximum bladder capacity of DO, DSD and DU group were all significantly decreased [(178±61), (184±81) and (194±93) vs (256±92)ml, all P<0.05]. The proportion of urgency had significant difference in the four groups [13(28.3%) in Normal group, 41(65.1%) in DO group, 22(56.4%) in DSD group and 11(44.0%) in DU group, P=0.001], and the proportion of dysuria had significant difference too [5(10.9%) in Normal group, 18(28.6%) in DO group, 20(51.3%) in DSD group and 15(60.0%) in DU group, P<0.001].
Children with 4 different conditions of NNLUTD have distinct video-urodynamic features. The higher ratio of VUR and UTI in DSD and DU children may be associated with reduced bladder compliance and increased post void residual. VUDS is useful for the diagnosis and treatment of refractory children with NNLUTD.
To evaluate the effect of topical glucocorticoid in treating phimosis on urinarytract infection(UTI) of vesicoureteral reflux(VUR) in infants.
Clinical data of infants with UTI diagnosed as primary VUR admitted to our hospital from January 2016 to January 2021 were retrospectively analyzed. The children were divided into three groups:the effective group (topical glucocorticoid was effective in the treatment of phimosis), the ineffective group(topical glucocorticoid was ineffective in the treatment of phimosis), and the untreated group(phimosis was not treated). Age of onset, degree of reflux, side and other indicators were compared to understand the effectiveness of topical glucocorticoid in treating phimosis, and the clinical characteristics of repeated UTI with VUR in treated phimosis and untreated phimosis.
A total of 544 children were included. Among them, 59 cases were treated with topical glucocorticoid for phimosis, 48 cases in the effective group, and their age was (12.5±8.4) months;11 cases in the ineffective group,and their age was (11.2±8.9) months. There were 485 cases in the untreated group, and their age was (13.1±9.3) months.The effective rate of topical glucocorticoid in the treatment of phimosis was 81.36%. There were 12 cases(12/48) of recurrent UTI in the effective group and 213 cases (213/485)of recurrent UTI in the untreated group, and the difference between the two groups was statistically significant (P=0.008).
Treatment of phimosis with topical glucocorticoid is an effective, easy to perform, and cost-effective method, and can effectively reduce the risk of recurrent UTI in infants with primary VUR.
To explore the characteristics and prognostic value of pulmonary hemodynamics in patients with acute coronary syndrome (ACS).
From a prospective multicenter registry study of pulmonary hypertension due to left heart disease, consecutive ACS patients who underwent coronary angiography in combination with left and right heart catheterization during hospitalization between January 2013 and November 2016 were involved. The primary endpoint was all-cause mortality. The prognostic variables identified by the Lasso analysis were included in the Global Registry of Acute Coronary Events (GRACE) score. Model performance was evaluated before and after the addition of hemodynamic parameters.
A total of 251 patients were enrolled, with age of (63.7±11.5) years. A total of 198 males (78.9%) and 53 females (21.1%) were recruited, and the median follow-up time was 34.7 months. Right heart catheterization-assessed mean pulmonary arterial pressure (mPAP), systolic pulmonary arterial pressure (sPAP) and diastolic pressure gradient (DPG) were found to be significant predictors for survival in ACS. Adjusted for age and sex, the adjusted HR (95%CI) of mPAP, sPAP and DPG were 1.068 (1.015-1.123), 1.033 (1.002-1.065) and 1.094 (1.008-1.187), respectively (P<0.05). Applied to the present cohort of 251 patients, the median of the GRACE score was 123 points, with a C-index of 0.703 (95%CI: 0.615-0.791) for predicting mortality. After the addition of mPAP or DPG to the GRACE score, the C-index increased to 0.715 (95%CI: 0.629-0.801) or 0.711 (95%CI: 0.625-0.797), respectively. When comparing two models before and after the addition of mPAP or DPG, the integrated discriminatory index (IDI) was 4.3% (95%CI: 0.2%-13.5%, P=0.030) and 3.0% (95%CI: 0.2%-11.1%, P=0.020), respectively.
Pulmonary hemodynamics can be predictive for survival in ACS patients, providing incremental prognostic value to risk assessment in ACS.
To construct a diagnostic and predictive model for chronic obstructive pulmonary disease complicated with pulmonary hypertension (COPD-PH) and evaluate its effect.
A total of 1 514 COPD patients treated in 5 hospitals from January 1, 2014 to December 31, 2019 were retrospectively collected and divided into training cohort (1 072 cases) and validation cohort (442 cases) according to the ratio of 7∶3 according to the inclusion time. Data including demographic data, smoking status, history of disease, and clinical examination were collected through patient medical records and electronic medical record systems. Multivariate logistic regression models were used to explore the related factors of COPD-PH, and the nomogram model was constructed using the "rms" program package. The calibration curve was used to evaluate the consistency between the prediction probability of the model and the actual results. The C index and the area under the receiver operating characteristic curve (ROC) were used to evaluate the discrimination of the model. The decision curve analysis (DCA) was used to evaluate the clinical practicability of the model.
In the training cohort, 3.7%, 15.2% and 81.1% were aged 50-59, 60-69 and ≥70 years, respectively, which were significantly different from the age composition of the validation cohort (7.9%, 27.8% and 64.3%, respectively) (P=0.041). There was no significant difference between the training cohort (79.4%) and the validation cohort (84.6%) (P=0.243). Multivariate logistic regression analysis of the training cohort showed that age ≥70 years [OR (95%CI): 3.32 (1.49-7.36)] and smoking status [former (current) smoking, OR (95%CI)] were 3.67 (2.51-5.37) and 2.04 (1.44-2.90), respectively], NT-probNP≥1 400 ng/L[OR (95%CI): 9.88 (6.23-15.66)], right atrial diameter [OR (95%CI): 1.11 (1.07-1.15)] was COPD-related factors of PH, based on the above factors-PH nomogram COPD model was set up and develop for online tools (https://ph-666.shinyapps.io/zhonghua-PH/). The calibrated C index (95%CI) of the training cohort and the validation cohort were 0.82 (0.77-0.87) and 0.77 (0.68-0.86), respectively. The calibration curve was close to the diagonal in both the training cohort and the validation cohort. The AUC (95%CI) of the nomogram model was 0.82 (0.80-0.85) in the training cohort and 0.77 (0.73-0.82) in the validation cohort. ROC curve showed that the optimal threshold in the training cohort was 0.60, and the sensitivity and specificity under this value were 0.74 and 0.78, respectively; the optimal threshold for the validation cohort was 0.70, and the sensitivity and specificity under this value were 0.76 and 0.65, respectively. DCA analysis showed that the nomogram model provided better net benefits than the all-variable selection and no-variable selection strategies with threshold probabilities greater than 15.0% and 13.0% in the training and validation cohorts, respectively.
The nomogram model for the diagnosis and prediction of COPD-PH is simple and accurate, which has a good clinical application prospect.
To investigate the relationship between KCNE family gene polymorphisms of potassium channel gene and the susceptibility of atrial fibrillation (AF).
In the case-control study, a total of 648 subjects were studied, of which 338 patients with atrial fibrillation were selected from the Department of Cardiovascular Medicine, Putuo Hospital Affiliated to Shanghai University of Traditional Chinese Medicine from January 2019 to December 2019, and 310 healthy people were selected from the physical examination population during the same period. DNA sequencing technology and polymerase chain reaction (PCR) were used to detect the genotype and allele frequency of rs1805127 of KCNE1, rs9984281 of KCNE2, rs9516, rs626930 of KCNE3 and rs12621643 of KCNE4.
The ages of subjects in atrial fibrillation group and control group were (69±13) and (73±8) years, respectively (P=0.077). Men subjects accounted for 57.70% (195 men) and 40.00% (124 men) in the two groups, respectively (P=0.092). The distribution frequencies of the allele C at rs1805127 of gene KCNE1, the allele A at rs9984281 of gene KCNE2 and the allele G at rs12621643 of gene KCNE4 were significantly different between groups (P<0.05). After adjustment for sex, smoking, hypertension, cardiac insufficiency and other factors, it was found that the increase in the frequency of the above three loci would increase the risk of atrial fibrillation (rs1805127 OR=7.064, 95%CI:1.559-31.997; rs9984281 OR=4.210, 95%CI:1.118-15.850; rs12621643 OR=2.679, 95%CI:1.025-6.998).
The rs1805127 of KCNE1, the rs9984281 of KCNE2,the rs12621643 of KCNE4 were significantly associated with the susceptibility to atrial fibrillation.
This study analyzed the clinical data of children with anorectal malformation (ARM)who underwent surgery from 2006 to 2021, and explored the related factors of postoperative voiding dysfunction (VD).A total of 60 children including 51 males and 9 females, aged 4-15 years, were enrolled. During follow up, normal voiding function were found in 43 cases, VD in 17 cases. It was found that middle to high clinical classification (OR=6.732, 95%CI:1.854-24.443), multiple surgeries (OR=3.712, 95%CI:1.133-12.160), associated spinal deformity (OR=3.297, 95%CI:1.029-10.566) and abnormal postoperative defecation (OR=4.971, 95%CI:1.387-17.816) were the risk factors of VD after ARM (all P<0.05). Urodynamic study and early intervention should be carried out in children with VD after ARM surgery.
This study is to investigate the effect of valve ablation on bladder function in patients with posterior urethral valves. The clinical data of patients with posterior urethral valves who received urodynamic examination before and after valve ablation were retrospectively analyzed.The bladder compliance improved during urine storage after operation, and the maximum detrusor pressure decreased during micturition. The postoperative urinary system ultrasound showed that the residual urine volume of the group with significantly improved upper urinary tract hydrocephalus was significantly less than that of the group with no improvement. The bladder compliance was significantly higher than that of the group with no improvement, and the maximum urine flow rate was significantly higher than that of the group with no improvement (all P<0.05). Valve ablation has limited effect on improving bladder function in patients with PUV. Valve incision can help improve the maximum bladder volume, residual urine volume and maximum urinary flow rate. It has a certain effect on bladder compliance and maximum detrusor pressure.
Neurogenic bladder (NB) is a disease caused by neuropathy or nerve damage resulting in vesicourethral dysfunction and abnormal urine storage and urination. Clean intermittent catheterization (CIC) is the best treatment for NB at present, but transurethral CIC has many complications. This paper introduces a case of NB treated by CIC through urachal cyst. The establishment of new catheterization channel can not only effectively reduce related complications, alleviate renal function damage, but also significantly improve the child's quality of life. It is worthy of promotion for children with suitable conditions.
Lower urinary tract dysfunction (LUTD) in children is more common clinically, and can be manifested as various lower urinary tract symptoms, urinary tract infection and vesicoureteral reflux. Neurogenic LUTD is mostly caused by congenital spinal cord agenesis; Non-neurogenic LUTD is mainly caused by various factors such as delayed maturation of lower urinary tract function and poor urinary habits. The diagnosis of LUTD relies on medical history, physical examination, urine routine, urine flow rate-residual urine and urinary system B-ultrasound. Severe cases require assessments such as bladder pressure/flow rate or imaging uromotility. Urodynamic examination is an important basis for diagnosing and formulating the treatment of LUTD. In this paper, the mechanism, diagnosis and treatment of LUTD symptoms were reviewed to provide clinical reference.
Glioma is a kind of malignant tumor of central nervous system that originates from glial cells, and has the characteristics of high recurrence rate and poor prognosis. Most glioma patients still have recurrence after standardized treatment, often accompanied by biological malignant progression, and more effective treatment methods need to be explored. Pyroptosis is a programmed cell death mode, which can promote and inhibit the development of glioma: on the one hand, pyroptosis inhibits the development of glioma by promoting the inflammatory death of tumor cells; On the other hand, inflammatory mediators released by pyroptosis promote the formation of tumor microenvironment adapted to tumor cell growth and thus promote glioma cell proliferation. In order to further understand the effect of pyroptosis on the development of glioma, this paper discusses the molecular mechanism of pyroptosis and the related research of pyroptosis and glioma, in order to provide new ideas and insights for the diagnosis and treatment of glioma.
Triple negative breast cancer (TNBC) is a type of breast cancer with high aggressiveness, easy recurrence and metastasis, strong heterogeneity, and lack of therapeutic targets. At present, chemotherapy is still the standard treatment for TNBC. However, with the progress of molecular typing and gene detection, immune checkpoint inhibitors (ICIs) have made considerable progress in the neoadjuvant, adjuvant and advanced treatment of TNBC, providing more choices for the treatment of TNBC. This article will review the progress of immunotherapy efficacy predictors, efficacy, and ICIs combined with other targeted therapies in TNBC.
Pulmonary hypertension (PH) is a group of clinical syndromes caused by thickening and remodeling of pulmonary blood vessels, which lead to pulmonary hemodynamic disorders. Its clinical characteristics are increased pulmonary circulatory resistance and increased pulmonary artery pressure, which can lead to right heart failure and death. Due to the etiology or trigger of unique developmental or maternal-related factors in children with PH, as well as the differences in children's own drug metabolism ability, the dosage of pH-targeting drugs and response to drug therapy are different from those of adults. In recent years, more and more clinical evidence-based studies have begun to pay attention to the targeted drug therapy of children's PH. This paper reviews the latest research progress of prostacyclin analogs and prostacyclin receptor agonists, endothelin receptor antagonists, phosphodiesterase type 5 inhibitors and soluble guanylate cyclase agonists.
Endovascular thoracic aortic repair (TEVAR) has become the first-line treatment for Stanford type B aortic dissection due to its advantages of less trauma, fewer perioperative complications, and fast postoperative recovery. However, with the widespread application of TEVAR, the incidence of new rupture in the proximal end of the stent is gradually increasing, and the mortality rate related to reverse tear type A aortic dissection (RTAD) is as high as 37.1%, which has attracted close attention of clinicians. This review focuses on the triggers and treatment of RTAD, and provides some reference value for the study of RTAD by understanding RTAD from multiple angles.
Human monkeypox infection (HMPX) can affect people of all ages, usually with systemic symptoms such as fever, fatigue, muscle pain, headache, back pain, and local or systemic lymph node swelling; After 1 to 3 days of prodromal symptoms, the patient develops a rash, which often begins from the face and spreads to other parts of the body, which can involve the soles of the feet and palms; The rash goes through the stages of maculation, papules, blisters, pustules, umbilication, crusting and shedding; The whole course of the disease usually lasts 2 to 4 weeks. However, the clinical manifestations of HMPX are affected by factors such as genetic background of the infected strain, host immune history, infection route, mixed infection and complications. Patients may have rare symptoms such as oral mucosal ulcer, dysphagia, diarrhea, etc., and may also have atypical manifestations such as no prodromal symptoms such as fever, rash before fever, only local single lesions and no rash, and asymptomatic infection has been confirmed. Only by understanding and being alert to various clinical manifestations of HMPX can we diagnose and treat patients in time and control the epidemic.
Pediatric vesicorectal dysfunction (BBD) refers to a type of excretory dysfunction of lower urinary tract symptoms (LUTS) and functional constipation (FC) caused by unknown causes. Approximately half of patients were seen for LUTS or FC. Multiple studies have demonstrated that there is an association between mother and child LUTS, however, whether a similar relationship exists for BBD has yet to be determined. To assess the family association of BBD, LUTS alone, and FC alone between mothers and children, the study was conducted in a population-based cross-sectional study in a Brazilian city according to the international diagnostic criteria for BBD, collecting relevant information about the family history of BBD, LUTS alone (LUTS without FC), and FC alone (FC without LUTS) in a total of 526 mothers and children. Among them, 441 pairs of mother-child information met the design criteria. Subjects were divided into BBD group, LUTS group alone (LUTS without FC), LUTS group (FC may be present at the same time), FC group alone (FC without LUTS), and FC group (LUTS may be present at the same time) according to the presence or absence of LUTS and FC. The surveyed children were all frequently living with their mothers, aged from 5 to 14 (9.1±2.7) years, of which 249 (56.5%) were females. The age of the mother was (35.7 ± 6.1) years.
Nocturnal enuresis, commonly known as bedwetting, is more common in children, and the prevalence rate of 7-year-old children is about 10% to 16%. It was found that nocturnal enuresis is highly heritable, but its genetically related gene loci are still not fully understood. The existing treatment methods for enuresis have a low cure rate and are prone to recurrence. By identifying genetic variants associated with nocturnal enuresis, it is conducive to exploring its genetic changes and potential biological concentration, and provides a reference for finding new therapeutic targets.
With the advent of drugs such as second-generation proteasome inhibitors (carfilzomib and ithazomib), new immunomodulatory drugs (pomalidomide), and monoclonal antibodies (daratumumab), overall survival of patients with multiple myeloma (MM) has improved over the past 10 years. But despite advances in the treatment of MM, the treatment of relapsed/refractory MM (RRMM) remains challenging and requires the development of drugs with different mechanisms of action. One marker of tumor cell survival in MM is the overexpression of anti-apoptotic proteins, including BCL-2, BCL-XL, BCL-W, and myeloid leukemia cell sequence-1 (MCL-1), among others. Veneclax, an inhibitor of the anti-apoptotic protein BCL-2, is a promising drug, especially in patients containing t (11; 14). Approximately 20% of patients with MM have t (11; 14), which leads to overexpression of BCL-2, and previous studies have shown strong monotherapy activity of veneclax in these patients. To explore the efficacy and safety of veneclax-based regimen treatment in patients with t (11; 14), the study retrospectively analyzed clinical data from 10 patients with t (11; 14) RRMM based on veneclax regimen treatment, with the primary endpoint being the objective response rate (ORR). Prior to the use of veneclax, 10 patients had received an average of 6 lines of therapy and were all resistant to bortezomib. Among the 9 patients with assessable response, the ORR was 78% (7 of 9 patients). One had a complete response (CR), one had a very good partial response (VGPR), four had a partial response (PR), and one had a therapeutically minimal response (MR). One patient with concomitant myocardial amyloidosis achieved organ remission with a 45% decrease in N-terminal B-type pronatriuretic peptide. Six patients survived the most recent follow-up, with a median follow-up of 5 months (range 3.1 to 13.5 months), a 6-month overall survival rate of 77% and a 6-month progression-free survival rate of 28%. The causes of death were 3 recurrences of MM and 1 septic shock/acute hypoxic respiratory failure due to invasive aspergillosis (in the case of disease progression). Non-hematological toxicities include fatigue, weight loss, loss of appetite, and gastrointestinal toxicities (nausea, diarrhea, and abdominal cramps). Four of the 10 patients required transfusion of red blood cells or platelets due to hematologic toxicity.
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