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The clinical data of one child with metanephric stromal tumor (MST) and BRAF V600E gene mutation admitted to the First Affiliated Hospital of Zhengzhou University in June 2022 was analyzed retrospectively.Literature was reviewed.The patient, a 2-year-old girl, was diagnosed with a tumor in the left abdomen.The maximum diameter of the tumor was 10.5 cm.A radical nephrectomy was performed on the left kidney, and postoperative pathology revealed MST.Microscopically, the tumor had no envelope and exhibited expansive growth.The tumor cells were fusiform or stellate, and nuclear division was visible in the cell-rich region.Dysplastic blood vessels were seen inside the tumor.The tumor cells around the blood vessels and invaginated renal tubules were arranged like onion skin.CD34 was detected positive by immunohistochemical staining, and BRAF V600E mutation was also detected positive by fluorescent polymerase chain reaction.A total of 21 relevant case reports were retrieved, including 16 in English and 5 in Chinese.Fifty-eight MST patients, including the one in this report were analyzed.These patients were aged 2 days to 15 years, with a median age of 2 years.Except for 2 patients with unknown sex, the ratio of male to female was about 1.4∶1.0.Most MST patients were asymptomatic, with an average tumor size of 5.3 cm.The tumor cell CD34 showed positive expression in different degrees.Eight patients received the BRAF V600E mutation detection, and the results were all positive.Fifty-eight patients underwent nephrectomy and were followed up for 0-156 months, of which 7 patients were assisted with radiotherapy and chemotherapy.During the follow-up, 1 patient died, and 1 patient had a relapse.MST is a rare benign renal stromal tumor. BRAF V600E mutations are detected in a variety of malignancies.This paper is the first to report MST with BRAF V600E mutation in China and points out the importance of molecular detection of BRAF mutation for accurate diagnosis of MST.
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Objective:To investigate the safety and efficacy of Belintoumab on the treatment of children with acute B-lymphoblastic leukemia (B-ALL).Methods:The clinical data of 10 children with CD 19+ B-ALL who were admitted to the Department of Pediatrics, the First Affiliated Hospital of Zhengzhou University from September 2021 to May 2022 and treated with Belintoumab were analyzed retrospectively. Results:Among the 10 cases, there were 6 recurrent cases, 3 cases with persistent minimal residual disease (MRD) positive after an initial treatment, and 1 case complicated with invasive candidiasis.Before treatment, bone marrow blasts ≥0.25, and that ranged 0.05-<0.25 were detected in 2 cases and 1 case, respectively.Seven cases had a complete remission (CR) of bone marrow, 6 of which were MRD positive and 1 case was MRD negative.After treatment with Belintoumab, the CR rate was 66.7% (2/3). The overall MRD negative rate was 88.9% (8/9), and the negative rate in previously MRD positive children was 100% (6/6). The median follow-up time was 4.1 (1.6-10.0) months after the application of Belintoumab.The overall survival (OS) rate was 70.0% (7/10). Eight MRD negative children received hematopoietic stem cell transplantation, and the OS rate was 75% (6/8). Survived children did not relapse until the last follow-up visit.Fever (90%, 9/10) was the most common adverse events, followed by neutropenia (90%, 9/10). One case (10%, 1/10) of neurotoxicity was seizures (grade 2) and one case (10%, 1/10) suffered cytokine release syndrome (grade 2), which did not influence the therapeutic efficacy of Belintoumab after symptomatic treatment.Conclusions:Belintoumab is safe and effective on the treatment of children with recurrent/refractory CD 19+ B-ALL, and those with MRD positive who have achieved CR in bone marrow have a higher rate of turning negative.Belintoumab can also be used as a bridge scheme for CD 19+ B-ALL children who cannot tolerate chemotherapy.
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Objective:To examine the clinical experience and efficacy of unrelated cord blood transplantation (UCBT) in the treatment of recurrent refractory Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis (EBV-HLH) in children.Methods:The clinical data of a patient with recurrent refractory EBV-HLH and intestinal perforation who was treated by UCBT in Department of Pediatrics, the First Affiliated Hospital of Zhengzhou University in September 2015 and finally cured were retrospectively analyzed.Meanwhile, literature was reviewed.Results:The patient, male, 1 year and 6 months, was admitted to the hospital with " fever for 15 days, rash for 9 days" as the main complaint, mainly manifested as high fever, large liver, spleen, lymph nodes, rapidly progressing pancytopenia, liver function damage, phagocytic blood cells on bone marrow smear, diagnosed as EBV-HLH in September 2015.The patient received chemotherapy according to the HLH-2004 protocol developed by the International Association of Cell Societies.During the treatment, he suffered two recurrence during the maintenance period, and a second-line rescue treatment was adopted, namely, " Pegaspargase, Doxorubicin liposome, Etoposide and Methylprednisolone" (L-DEP regimen) chemotherapy.The complete relief of diagnostic indexes for hemophagocytic lymphohistiocytosis was evaluated after chemotherapy.The patient developed sudden intestinal perforation and underwent emergency surgical surgery, enteroenterostomy.After the condition was stabilized, the patient was pretreated with the " Fludarabine+ Busulfan+ Cyclophosphamide" (Flu+ BU+ CY) therapy and then treated with UCBT, with intravenous nutritional support provided during the entire process.Neutrophil and platelet implantation was implemented on day 13 and day 35 after transplantation, respectively.The chimeric rate was 100%, and the implantation was a success.Hepatic veno-occlusive disease, fungal pneumonia and skin graft-versus-host disease (GVHD) Ⅱ occurred on the 15 th day, 22 nd day and 26 th day after transplantation, respectively.The corresponding symptoms improved after treatment.On day 49 after transplantation, phase Ⅱ " enterostomy fistula" was performed.The patient was followed up to 70 months after transplantation, and generally in good condition.His symptoms relieved, and no chronic GVHD and other comorbidities occurred. Conclusions:Allogeneic hematopoietic stem cell transplantation is the only possible effective means of treating relapsed refractory EBV-HLH in children.In the absence of a suitable sibling or unrelated donor, unrelated cord blood stem cells can be used as a graft source.Enterostomy after intestinal perforation is not contraindicated for transplantation.
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Objective:To investigate the clinical features and long-term prognosis of pediatric acute lymphoblastic leukemia (ALL) with renal involvement as the initial manifestation, thus enhancing the diagnostic and therapeutic efficacy.Methods:Twenty-four cases of pediatric ALL with renal involvement as the initial manifestation treated in the First Affiliated Hospital of Zhengzhou University from March 2013 to March 2019 were analyzed retrospectively, and their clinical characteristics were analyzed.According to renal imaging examination findings, they were divided into abnormal group and normal group.The differences in clinical features between the two groups were compared, and the cumulative survival rate was evaluated by Kaplan-Meier method.Results:Among 1 030 newly treated cases of pediatric ALL, 24 cases(2.33%) had renal involvement as the initial manifestation, involving 20 males and 4 females, with a male/female ratio of 5∶1 and the median age of 4.3 years (1.3-14.0 years). There were 16 cases of superficial lymph node enlargement and 21 cases of hepatosplenomegaly.Immature cells in peripheral blood were found in 15 cases.Nine cases were examined with abnormal renal imaging, involving 8 cases returned normal after chemotherapy, and 1 died of renal failure.At the end of follow-up on August 1, 2020, there were 9 cases of bone marrow relapse, 11 survival cases, 10 death cases and 3 cases of loss to follow-up.There were no significant differences in the sex, age, immunophenotype, organ infiltration and urinary protein between the two groups (all P>0.05). The proportion of high creatinine level and intramedullary recurrence rate in the abnormal group were significantly higher than those in the normal group [55.6%(5/9 cases) vs.0(0/15 cases), P=0.003; 66.7%(6/9 cases) vs.20.0%(3/15 cases), P=0.036]. The survival analysis indicated that the 3-year cumulative survival in the abnormal group was significantly lower than that of normal group (17.3% vs.72.7%, χ2=4.047, P< 0.05). Conclusions:For children with unexplained renal involvement as the initial manifestation, clinicians should consider the possibility of leukemic renal infiltration or nephrogenic lymphoma.Physical examinations of the liver, spleen and lymph nodes, morphological analysis of peripheral blood cells, bone marrow examination and renal biopsy are important to make a definite diagnosis in time.Children with imaging abnormalities caused by leukemic renal infiltration are more likely to relapse and have a lower survival rate, which may be a poor prognostic factor for ALL.
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Objective:To discuss whether platelet distribution width (PDW) can effectively predict the prognosis of neuroblastoma (NB).Methods:The clinical data of 67 NB patients in the First Affiliated Hospital of Zhengzhou University between January 2014 and January 2018 were retrospectively analyzed.They were divided into low PDW group and high PDW group according to the PDW level, and the differences in clinical indicators between the 2 groups were compared.The prognostic effects of PDW were assessed by using the Kaplan- Meier method and Cox regression model. Results:Among the 67 patients, 41 cases were male, 26 cases were female, with the ratio of male to female being 1.58∶1.00, and the average age was 44 months (2-156 months). Five cases were in stage Ⅰ, 1 case in stage Ⅱ, 15 cases in stage Ⅲ and 46 cases in stage Ⅳ.At the first time of diagnosis, there were 14 cases with age ≤ 18 months, 53 cases with age > 18 months, 47 cases with neuron specific enolase (NSE) level ≥ 100 μg/L, 20 cases with NSE level<100 μg/L.The median follow-up time was 20.4 months.At the end of follow-up, 35 cases died and 32 cases survived.There was no statistical difference in age, gender, primary site of tumor, tumor stage and mean platelet volume between the low PDW group and the high PDW group (all P>0.05). The proportion of high-risk patients, the level of NSE, bone marrow metastasis rate, MYCN gene amplification rate and the red blood cell distribution width in the high PDW group were significantly higher than those in the low PDW group, but the high PDW group had a lower level of thrombocytocrit than the low PDW group, and the differences were statistically significant(all P<0.05). Survival analysis revealed that the 2-year overall survival of the low PDW group was significantly higher than that of the high PDW group (69.8% vs.25.3%, χ2=15.761, P<0.05). Univariate analysis showed that NSE ( HR=6.606, 95% CI: 2.018-21.620), MYCN gene ( HR=1.977, 95% CI: 0.794-4.919), tumor risk stratification ( HR=5.926, 95% CI: 1.416-24.794), PDW ( HR=4.036, 95% CI: 1.957-8.322), and red blood cell distribution width ( HR=1.120, 95% CI: 1.005-1.249) were the adverse factors affecting the overall survival, and thrombocytocrit was a protective factor for the prognosis of NB.Multivariate analysis indicated that PDW was an independent risk factor of NB ( HR=2.524, 95% CI: 1.017-6.264, P=0.046). Conclusions:There is a good consistency between the increase of PDW and the known prognostic risk factors, elevated tumor markers and bone marrow metastasis.Increased PDW is associated with poor prognosis in NB patients, and PDW is an independent risk factor for the poor prognosis of NB.
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Objective:To analyze the efficacy and relevant indicators of efficacy prediction of immunosuppre-ssive therapy (IST) that was composed of rabbit anti-human thymocyte immunoglobulin (rATG) and cyclosporine A (CsA) to treat acquired aplastic anemia (AA) in children.Methods:Retrospective analysis of the clinical data from 89 cases of children who were diagnosed with acquired AA and applied IST in Children′s Hospital of the First Affiliated Hospital of Zhengzhou University from January 1, 2012 to November 30, 2016 were collected.Patients were followed up for 2 years.Clinical features, curative effect and relevant indicators of efficacy prediction were analyzed retrospectively.Results:Among the 89 children with acquired AA, there were 27 cases of very severe AA(vSAA), 48 cases of severe AA(SAA) and 14 cases of transfusion-dependent non-severe AA(NSAA), with the median age of 7 years old.There was no significant difference in the curative effect among vSAA, SAA and transfusion dependent NSAA at different follow-up time nodes ( P>0.05). The recurrence rate of acquired AA was 4.49%(4/89 cases) and the median recurrence time was 18 months.The clonal evolution of acquired AA was 2.24%(2/89 cases). In multi-factor analysis, at the 6 th month of IST treatment, the newly diagnosed children displayed increased CD4 +/CD8 + ratio and early treatment response to granulocyte colony stimulating factor (G-CSF), indicating that the children responded well to the treatment of IST. Conclusions:Combined with CsA, the IST of rATG is a safe and effective method for the treatment of acquired AA in children.The higher CD4 +/CD8 + ratio and early treatment response of G-CSF are good predictors of treatment response to immunosuppressive therapy at 6 months, respectively.
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Objective To analezk thk charactkristics of drug-induckd livkr injure( DIFI)in childrkn with acutk lemphoblastic lkuckmia(LFF),so as to improvk thk phesician＇s undkrstanding of chkmothkrape DIFI,and to guidk clinical rational drug usk. Methods Onk hundrkd and forte-thrkk casks with LFF diagnoskd in thk Dkpartmknt of Hk-matologe and Oncologe in thk Pirst Lffiliatkd Hospital of Yhkngzhou Rnivkrsite from Januare 2012 to Dkckmbkr 2016 wkrk analezkd rktrospkctivkle. Baskd on DIFI diagnostic critkria and thk ARCLM scalk,thk casks with a scork of ≥3 points wkrk considkrkd to havk chkmothkrape DIFI. Groupkd be gkndkr,agk,immunoteping,risc and stagk of chkmo-thkrape,thk incidknck of DIFI was comparkd. Thk situation aftkr DIFI prkvkntion was comparkd bktwkkn two groups which was groupkd according to whkthkr thk application of hkpatoprotkctivk drugs. ResuIts Onk hundrkd and kight ca-sks(75. 52﹪)had DIFI,66 casks(61. 11﹪)showkd clinical manifkstations of livkr injure,and 42 casks(38. 89﹪) had no clinical semptoms. Lmong all thk casks 57. 41﹪(62 casks)wkrk mild livkr damagk,25﹪(27 casks)wkrk modkratk livkr injure and 17. 59﹪(19 casks)wkrk skvkrk livkr damagk. Thk clinical tepks which wkrk hkpatockllular accounting for 79. 63﹪(86 casks),cholkstatic 7. 41﹪(8 casks)and mixkd 12. 96﹪(14 casks). Malk wkrk 80 casks (79. 21﹪)and fkmalk 28 casks(66. 67﹪),but thk incidknck of DIFI bktwkkn diffkrknt gkndkr group had no statistical diffkrknck(χ2 ﹦2. 524,P﹦0. 112). Skvknte-fivk casks(77. 32﹪)wkrk ＜7 ekars agk and 33 casks(71. 74﹪)≥7 ekars agk,and thk incidknck of DIFI bktwkkn 2 groups was not statisticalle diffkrknt(χ2 ﹦0. 526,P﹦0. 468). Thkrk was no significant diffkrknck in T-LFF(8 casks,61. 54﹪)and B-LFF(100 casks,76. 92﹪)( χ2 ﹦0. 795,P﹦0. 372). Thk incidknck had significant diffkrknck in diffkrknt risc(P﹦0. 002). Thk incidknck of DIFI bktwkkn thk middlk risc group(60 casks,88. 24﹪)and standard risc(21 casks,58. 33﹪)had statistical diffkrknck( P ＜0. 05 ). Thk incidknck of DIFI bktwkkn thk middlk risc group and skvkrk risc(27 casks,69. 23﹪)had statistical diffkrknck( P﹦0. 015). Thk incidknck was diffkrknt in diffkrknt stagks of chkmothkrape(P＜0. 05). Thk incidknck of DIFI in induckd stagk was diffkrknt comparkd to othkr stagks(P＜0. 05). ARCLM scork ＞8 points accountkd for 21 casks(19. 45﹪), 6-8 points accountkd for 59 casks(54. 63﹪)and 3 -5 points accountkd for 28 casks(25. 92﹪). Eighte -nink patiknts(92. 71﹪)wkrk kffkctivk in thk hkpatoprotkctivk group and 8 patiknts(66. 67﹪)in thk no hkpatoprotkctivk thkrape group. Thk diffkrknck bktwkkn thk 2 groups was statisticalle significant(χ2 ﹦5. 317,P﹦0. 021). ConcIusions Thk clinical semptoms of drug-induckd livkr injure in childrkn with LFF chkmothkrape ark lacc of spkcificite. Thke ark mainle charactkrizkd be mild livkr injure. Thk clinical tepk of hkpatic injure is common in hkpatockllular. Thk ARCLM scork was mostle 6 to 8. Thkrk is no rklationship bktwkkn thk incidknck in LFF and gkndkr,agk,tepk of lkuck-mia. Thk incidknck with modkratk risc tepk is highkr than that of thk standard and high-risc tepk. Thk incidknck in induction rkmission stagk is highkst. Lpplication of hkpatoprotkctivk drugs is bknkficial to DIFI prognosis.
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Objective To explore the clinical and pathological features, diagnosis and treatment of eosinophilic cystitis in children. Method The clinical data of 7 patients with eosinophilic cystitis admitted from 2012 to 2016 were retrospectively analyzed, and the related literature were reviewed. Results The median age of the 7 patients was 9 years, and clinical manifestations were urgent urination, frequent micturition, odynuria, hematuria, abdominal pain and nocturnal enuresis. Ultrasonography and CT examination showed thickened bladder wall and space occupying lesions.All the 7 children received bladder biopsy, and pathology was consistent with eosinophilic cystitis. Six of them were cured after 2 months of drug therapy, and the other one was cured by repeated drug treatment for 1 year.All patients were followed up for 3 months to 4 years until the abnormal symptoms of voiding disappeared and the abnormal changes of bladder disappeared by imaging examination. Conclusion Eosinophilic cystitis in children is a benign lesion, having extremely similar clinical manifestations to bladder tumor. Without biopsy, the diagnosis of eosinophilic cystitis can also be made according to the clinical manifestation, laboratory examination and treatment effect. The treatment for this disease mainly includes hormone, antihistamine and anti-inflammatory drugs.
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Objective To evaluate the bioequivalence of domestic and imported terazosin hydrochloride tablets after single oral dose .Methods It was a single center ,randomized ,open ,cross-over trail design ,21 subjects were fasting oral adminis-tered of 2 mg domestic and imported terazosin hydrochloride tablets in different periods ,venous blood 4 ml were collected in different time points before and 60 h after administration ,plasma concentration of terazosin was determined by LC-MS/MS . Results The main pharmacokinetic parameters of domestic and imported terazosin hydrochloride tablets were as follows :t1/2 :(13.2± 2.39)hvs(12.5±1.93)h,tmax :(1.01±0.83)hvs(1.08±0.69)h,Cmax :(40.1±10.6)ng/mlvs(37.3± 9 .57) ng/ml;AUC0- ∞ :(428 ± 82 .1) ng · h/ml vs (426 ± 85 .2) ng · h/ml .The relative bioavailability of domestic terazosin hydrochloride tablets was (101 .2 ± 14 .7)% .90% CI of domestic and imported terazosin hydrochloride tablets AUC0-t and Cmax geometric mean ratio fell between 80% -125% .Conclusion The domestic tablets are bioequivalent to the imported tablets .
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Objective To detect the expression of autophagy-related gene Atg3 and Atg5 in bone marrow mononuclear cells (BMMNCs) from children with acute leukemia(AL),so as to explore the relationship between autophagy and the pathogenesis of AL in children.Methods Seventy-four bone marrow specimens were obtained from children with AL in the First Affiliated Hospital of Zhengzhou University Pediatrics Hematology Ward,including 37 cases of initially diagnosed AL without any treatment,28 cases of AL in complete remission,9 cases of refractory or relapse AL and 28 bone marrow specimens from children without tumor were also collected as the control group.BMMNCs were separated by Lydroxypropylmethyl Cellulose.After BMMNCs were stained by Monodansylcadaverine,the autophagy phenomenon was observed by using fluorescence microscope,and the ratio of autophagy was detected by using flow cytometry.Reverse transcription polymerase chain reaction(RT-PCR) was used to detect the expression of Atg3 mRNA and Atg5 mRNA in each group.Results It was found that autophagy phenomenon was more common in the initially diagnosed group and the refractory/relapse group,and the autophagy ratio in both groups was respectively (17.07 ±2.31) ％,(15.37 ± 1.59) ％,respectively,which were obviously higher than that of the control group (2.71 ± 1.57) ％ and that of the complete remission group.The differences were statistically significant (t =28.29,20.96,all P ＜ 0.01).The autophagy ratio in complete remission group was (3.48 ± 1.94) ％,and compared with the control group,the difference was of no statistical significance(t =1.634,P ＞ 0.05).The autophagy ratio in the refractory/relapse group higher than that in the complete remission group (t =16.61,P ＜ 0.05).The expressions of Atg3 mRNA and Atg5 mRNA in initially diagnosed group and refractory/relapse group were higher than those of the complete remission group and control group,and the differences were statistically significant (F =67.592,106.160,all P ＜ 0.008) ; the difference between complete remission group and control group was of no statistical significance (P ＞ 0.008).Conclusions The autophagy ratio and the expressions of Atg3 mRNA and Atg5 mRNA in initially diagnosed group and the refractory/ relapse group were both obviously higher.It was revealed that higher autophagy activity,which was caused by upregulated expressions of Atg3 mRNA and Atg5 mRNA,had a closely connection with the mechanism of occurrence,development and resistance of AL in children.