ABSTRACT
Purpose To explore the diagnostic value of dif-ferent fluorescence in situ hybridization(FISH)signal types and chromosomal karyotyping analysis in ETV6/RUNX1-positive B-cell acute lymphoblastic leukemia(B-ALL).Methods Clini-cal data of 164 newly diagnosed ETV6/RUNX1-positive B-ALL patients were collected for retrospective analysis of chromosomal karyotyping and FISH.Results Among the 164 patients,163 positive cases were detected by FISH,among them the classic 2F1R1G signal type was found in 61 cases,and 102 cases showed non-classic signal types,with 2F1G and 1F1R2G signal types being the most common,indicating ETV6 deletion.Among them,the classic 2F1R1G signal type was found in 61 cases,and 102 cases showed non-classic signal types,with 2F1G and 1F1R2G signal types being the most common,indicating ETV6 deletion.Among the 125 children who could undergo karyoty-ping analysis,106 had a normal karyotype and 19 had an abnor-mal karyotype,with no detection of t(12;21)translocation.Conclusion FISH technology has high sensitivity in detecting ETV6/RUNX1 fusion genes,and it often manifests as non-clas-sic signal types,including ETV6 deletions.Chromosomal karyo-typing analysis helps to identify complex karyotypes and polyploidy but is not conducive to detecting t(12;21)fusion.Therefore,both FISH signal types and karyotyping analysis play indispensable roles in ETV6/RUNX1-positive B-ALL.
ABSTRACT
Venous thromboembolism (VTE) is a complication in children with acute lymphoblastic leukemia (ALL). The Chinese Children's Cancer Group-ALL-2015 protocol was carried out in China, and epidemiology, clinical characteristics, and risk factors associated with VTE were analyzed. We collected data on VTE in a multi-institutional clinical study of 7640 patients with ALL diagnosed in 20 hospitals from January 2015 to December 2019. First, VTE occurred in 159 (2.08%) patients, including 90 (56.6%) during induction therapy and 108 (67.92%) in the upper extremities. T-ALL had a 1.74-fold increased risk of VTE (95% CI 1.08-2.8, P = 0.022). Septicemia, as an adverse event of ALL treatment, can significantly promote the occurrence of VTE (P < 0.001). Catheter-related thrombosis (CRT) accounted for 75.47% (n = 120); and, symptomatic VTE, 58.49% (n = 93), which was more common in patients aged 12-18 years (P = 0.023), non-CRT patients (P < 0.001), or patients with cerebral thrombosis (P < 0.001). Of the patients with VTE treated with anticoagulation therapy (n = 147), 4.08% (n = 6) had bleeding. The VTE recurrence rate was 5.03% (n = 8). Patients with VTE treated by non-ultrasound-guided venous cannulation (P = 0.02), with residual thrombus (P = 0.006), or with short anticoagulation period (P = 0.026) had high recurrence rates. Thus, preventing repeated venous puncture and appropriately prolonged anticoagulation time can reduce the risk of VTE recurrence.
Subject(s)
Humans , Child , Venous Thromboembolism/etiology , East Asian People , Precursor Cell Lymphoblastic Leukemia-Lymphoma/epidemiology , Risk Factors , Thrombosis/chemically induced , China/epidemiology , Anticoagulants/adverse effects , RecurrenceABSTRACT
Objective:To explore the application of scenario simulation teaching based on PBL in communication skills training of hematology students in Children's Hospital.Methods:The training of doctor-patient communication skills was conducted among trainees who had the standardized residency training at the Department of Hematology of the Children's Hospital of Soochow University. All the residents were randomized into the control group and observation group by lottery, with 24 residents in each group. The control group adopted the traditional narrative teaching method, and the observation group adopted PBL combined with scenario simulation teaching method. The Liverpool communication skills assessment scale (LCSAS) was used to compare the differences between the two groups before and after training, and the differences between the two groups after training. Then the degree of residents' recognition of these two teaching methods was investigated. Finally, the examination results were used to evaluate knowledge mastery of doctors in department of hematology. SPSS 20.0 was used for Chi-square test and t-test. Results:LCSAS scores of the two groups before training were respectively (11.61±2.21) and (11.95±2.22), with no statistically significant difference ( P >0.05). After PBL-based scenario simulation teaching and training in the observation group, the LCSAS score of the observation group (27.41±2.53) was higher than that of the control group (23.30±1.81), and the difference between the two groups was statistically significant ( P<0.05). Questionnaire survey results showed that the favorable rating rate of PBL-based scenario simulation teaching was 91.67% (22/24), higher than that of the traditional narrative teaching method [62.50% (15/24)], and the difference was statistically significant ( P<0.05). The examination of students' mastery of professional knowledge showed that after the PBL-based scenario simulation teaching and training, the trainees had a better grasp of knowledge and a higher score, with excellence rate of 91.67% (22/24）, which was higher than 66.67% (16/24) of the control group, with a statistically significant difference ( P<0.05). Conclusion:The scenario simulation teaching based on PBL could improve the communication ability and professional knowledge of trainees taking standardized residency training in the department of hematology, and the trainees are highly satisfied with this teaching method.
ABSTRACT
Methotrexate(MTX)is one of the main drugs used to prevent graft-versus-host disease(GVHD)after hematopoietic stem cell transplantation, but it can cause a variety of adverse reactions, including severe mucositis, bone marrow suppression and hepatotoxicity.Studies on MTX gene polymorphisms mainly focused on the efficacy and complications of high-dose MTX therapy for various cancers, with relatively few studies on hematopoietic stem cell transplantation.From the perspective of allogeneic hematopoietic stem cell transplantation(allo-HSCT), this article provided a comprehensive review on the pharmacokinetics, complications, and prognosis with MTX gene polymorphisms in allo-HSCT patients, in order to provide clinical reference.
ABSTRACT
Objective:To evaluate the clinical efficacy and safety of Blinatumomab on the treatment of refractory or relapsed precursor B-cell acute lymphoblastic leukemia (R/R BCP-ALL) in children.Methods:Clinical data of children with R/R BCP-ALL treated with Blinatumomab in the Department of Hematology, Children′s Hospital of Soochow University, from August 2021 to June 2022 were retrospectively analyzed.Children were divided into<45 kg group and ≥45 kg group according to their weight at admission.They were treated with different dosages of Blinatumomab, and bone marrow remission was assessed at about 15 days.Clinical indicators and adverse events during the treatment period were recorded.The rank sum test of two independent samples were used to compare the differences between groups.The Fisher′ s test was used for comparing categorical variables. Results:Among the 16 children with R/R BCP-ALL, 12 cases (75%) achieved complete response (CR) and minimal residual lesion (MRD) turned negative at about 14 days.Among them, 5 out of 9 children with bone marrow primitive naive cell ratio≥0.5 achieved CR, and 7/7 children with bone marrow primitive naive cell ratio<0.5 achieved CR.The peak value of interleukin-6 (IL-6) in children with CR was significantly higher than those without CR ( Z=2.50, P=0.012). Twelve cases achieved CR on bone marrow assessment around day 15, and 3 cases who did not achieve CR remained in remission on day 28, with an efficacy prediction accuracy of 93.8%(15/16). Adverse events included fever, neutropenia, hypokalemia, abnormal liver function, hypocalcemia, edema, rash, hypertension, myocardial damage, abdominal pain, hypotension, and cytokine release syndrome, which were all grade 1.Neurotoxicity and death were not reported. Conclusions:The remission rate of R/R BCP-ALL in children treated with Blinatumomab was high, especially in patients with a low tumor load.The toxicity and adverse events of Blinatumomab treatment are minor and controllable.Day 15 is the optimal time point to evaluate the efficacy of Blinatumomab on children with R/R BCP-ALL, and a higher IL-6 peak can be served as a predictor of its efficacy.
ABSTRACT
Abnormal transcription of oncogenes driven by super enhancers was found to be critical for maintaining tumor cell identity.The expression of oncogenes can be effectively suppressed by inhibiting the key regulator that super enhancers regulate oncogene transcription.Bromodomain protein 4(BRD4)is a key protein to recognize the super enhancer regulatory elements, which can bind to acetylated histones or non-histones to regulate gene transcription.The abnormal expression of BRD4 is closely related to the malignant development of a variety of hematologic oncology.Targeting BRD4 can effectively control the malignant development of hematologic tumors.In recent years, BRD4-targeted drugs in hematologic oncology have received extensive attention, and they showed good antitumor effects either as a single drug or in combination with other drugs.In this paper, in order to provide a new understanding of the occurrence of leukemia and treatment of hematologic oncology, the biological functions of BRD4 as well as the molecular drugs targeting BRD4 are reviewed.
ABSTRACT
This study reports the diagnosis and treatment of 2 children with isolated testicular recurrence (ITR) of acute B lymphoblastic leukemia (B-ALL) treated with CD 19 targeted chimeric antigen receptor T (CD 19 CAR-T) cells in May and December 2019 in the Department of Hematology and Oncology, Children′s Hospital of Soochow University, and explores the efficacy of CD 19 CAR-T cells therapy versus conventional radiotherapy and chemotherapy through literature review.Both cases were diagnosed as B-ALL by the morphologic, immunologic, cytogenetic and molecular biology methods.ITR was diagnosed by testicular biopsy at 60 months and 38 months after initial diagnosis in 2 cases, respectively.After infusion of CD 19 CAR-T cells at 7.0×10 6/kg and 1.5×10 7/kg, respectively for 7-10 days, testicular leukemia cell infiltration disappeared and complete remission was obtained.Among them, case 2 developed cytokine release syndrome and immune effector cell-related neurotoxicity syndrome after treatment, which was improved after drug intervention.It is suggested that CD 19 CAR-T cells are effective in the treatment of ITR in children, which may be an alternative to orchiectomy or local radiotherapy for ITR in children with B-ALL.
ABSTRACT
【Objective】 To determine the therapeutic efficacy of cladribine combined with BuCy conditioning regimen for childhood acute leukemia, and compare it with fludarabine. 【Methods】 The clinical data of 70 children with acute leukemias who underwent all-HSCT from August 2018 to June 2020 were collected. The data of pretreatment-related toxicity, hematopoietic reconstitution, graft-versus-host disease, virus infection, relapsed and survival between CLAG and FLAG group were statistically analyzed. 【Results】 EBV infection in CLAG group was significantly more than that in FLAG group(P0.05). Multivariate analysis showed that there was no significant difference in the effect of conditioning regimen on relapsed and survival. Among other risk factors, types of diseases were significantly correlated with OS (P<0.05, HR: 0.088). Relapse was significantly correlated with bone marrow morphological remission before transplantation and the matching degree of donor and recipient HLA(P<0.05, HR: 34.678; P<0.05, HR: 0.024). 【Conclusion】 There was no significant difference in RRTs, hematopoietic reconstitution, GVHD occurrence, OS and relapsed between CLAG group and FLAG group. The overall efficacy of CLAG group was not inferior to FLAG group.
ABSTRACT
Objective:To explore the risk factors for hemophagocytic syndrome (HPS) in childhood Epstein-Barr virus (EBV)-associated infectious mononucleosis (IM).Methods:From January 2013 to December 2017, the medical charts of all children who were diagnosed with EBV-associated IM and HPS in Children′s Hospital of Soochow University were analyzed retrospectively. Statistical analyses were performed using SPSS version 22.0.Results:A total of 316 IM and 59 HPS were enrolled. The age was (4.26 ± 2.95) years old with a male-to-female ratio of 1.2∶1. In addition to the diagnostic criteria of HPS, there were significantly lower rates of fever >10 d, hepatomegaly, jaundice, alanine aminotransferase >500 U/L, aspartate aminotransferase >500 U/L, LDH >1 000 U/L, C-reactive protein >50 mg/L and hypoalbuminemia in children with EBV-associated IM compared to those with HPS, and the differences were statistically significant ( P<0.05). Multivariate Logistic regression analysis showed that fever >10 d, eyelid edema, lymphadenopathy and purulent tonsils were independent predictors of HPS in children with EBV-associated IM ( P<0.05). Hepatomegaly and fever >10 d were risk factors ( OR = 16.079 and 12.138, 95% CI 2.788 to 92.744 and 2.878 to 51.180). Eyelid edema, lymphadenopathy and purulent tonsils were protective factors ( OR = 0.087, 0.006 and 0.031; 95% CI 0.010 to 0.723, 0.001 to 0.058 and 0.007 to 0.146). Conclusions:Hepatomegaly and fever >10 d are the risk factors for hemophagocytic syndrome in childhood EBV-associated infectious mononucleosis.
ABSTRACT
Griscelli syndrome type Ⅱ (GS2) is a rare disease, and patients with GS2 are susceptible to secondary haemophagocytic lymphohistiocytosis (HLH). GS2 accompanied by secondary HLH has a dangerous clinical course, high mortality, and a high miss-diagnosis rate.In this paper, the pathogenesis and prognosis of a case confirmed as GS2 with secondary HLH by gene screening were reported, so as to improve diagnosis and treatment of this disease.The patient had clinical manifestations of silver hair and eye lashes, recurrent pulmonary infection, contiuning high fever, significantly increased ferroprotein levels and decreased fibrinogen levels.Besides, RAB27A gene homozygous mutations were found in the patient, originating from her parents (p.P126Qf3*3 frameshift mutation). This finding confirmed the diagnosis of GS2.The patient underwent transplantation of marrow stem cells from her father since the father-daughter HLA was 7/10.The follow-up results showed that the patient was still alive and healthy 2 years after transplantation.
ABSTRACT
Objective:To investigate the effect of different collection procedures of COBE Spectra blood cell separator on collection of autologous peripheral blood hematopoietic stem cells in children.Methods:The clinical data of 10 children who were collected autologous peripheral blood hematopoietic stem cells with the monocytes (MNC) or fully automatic peripheral blood stem cells (AutoPBSC) programs of COBE Spectra blood cell separator in the Children's Hospital of Soochow University from July 2018 to January 2020 were retrospectively analyzed, and the children were 3-10 years old with the body weight of 15-31kg. There were 5 cases in MNC group and 5 cases in AutoPBSC group.Results:Autologous peripheral blood hematopoietic stem cells were collected for 25 times, with an average of 2.5 times (1-4 times), 10 times in MNC group and 15 times in AutoPBSC group. The number of stem cells [the median (the range)] before collection was 19.3/μl (3.5-129.0/μl) in MNC group and 9.4/μl (2.2-38.7/μl) in AutoPBSC group. The number of CD34 + cells of single collection was 1.22×10 6/kg (0.18×10 6/kg-6.30×10 6/kg) in MCN group and 0.85×10 6/kg (0.13×10 6/kg-2.64×10 6/kg) in AutoPBSC group. Correlation analysis showed that there was a positive correlation between the number of collected CD34 + cells and the number of stem cells before collection (AutoPBSC group: r=0.921, P < 0.01; MNC group: r=0.833, P=0.003). The collection efficiency was 5.4% (3.4%-11.2%) in MNC group and 10.4% (4.7%-13.9%) in AutoPBSC group, and the difference was statistically significant ( Z=2.163, P = 0.031). Conclusion:The collection effect of children's autologous peripheral blood hematopoietic stem cells with COBE Spectra blood cell separator AutoPBSC program is better than that with MNC program.
ABSTRACT
Programmkd ckll dkath 1( PD-1),locatkd on thk ckll mkmbrank,rkstricts lemphocetk activation and down-rkgulatks ckll growth and cetocink skcrktion through its ligands -1(PD-F1)or PD-F2. Thk main rolk of PD-1╱PD-F1 pathwae in canckr is thk immunosupprkssion in tumor microknvironmknt,which macks thk tumor cklls kscapk from thk immunk survkillanck in patiknts. Thk bloccing drugs targkting thk PD-1╱PD-F1 pathwae,which is thk spkcific antibode against PD-1╱PD-F1 pathwae,can bk uskd in thk trkatmknt of tumor and thk combinkd applica-tion is kxpkctkd to improvk thk thkrapkutic kffkct.
ABSTRACT
Objective@#To evaluate the efficiency and safety of low intensity conditional regimen for children with Fanconi anemia (FA) receiving allogenic hematopoietic stem cells transplantation (allo-HSCT).@*Methods@#Four patients diagnosed as Fanconi anemia were enrolled in this study. One patient received HLA-identical sibling donor hematopoietic stem cell transplantation, two patients underwent unrelated donor matched (UD) HSCT, and one patient received unrelated cord blood transplantation. The conditional regimen consisted of Busulfan with low dose of cyclophosphamide.@*Results@#All 4 cases succeeded in allo-HSCT. The median time for neutrophils engraftment was 11(9-15) day, median time to platelets (PLT) engraftment was 12 (8-28) day. One case occurred with grade I of aGVHD, 1 case with hemorrhagic cystitis. No patient happened with hepatic veno-occlusive disease (VOD).@*Conclusion@#Low intensity of conditional regimen is efficient and safe which should be recommended for FA patients with HSCT.
ABSTRACT
Objective To compare the efficacy and safety of induction therapy in 3+7 protocol and 3+10 protocol in children with acute myeloid leukemia (AML). Methods Two protocols were carried out in our hospital during January 2010 to January 2015, namely 3+7 protocol(AML-06,A group) and 3+10 protocol (modified AML protocol, B group). A total of 56 cases aged from 1 year-old to 13 year-old were enrolled in A group with male to female ratio at 31:25. Five of them were classified as FAB M1, 25 as M2, 11 as M4, 10 as M5, 2 as M6 and 3 as M7. Another 44 cases aged from 1 year to 12 years were enrolled in B group with a male to female ratio at 26:18, and 17 cases were classified as FAB M2, 14 as M4, 9 as M5, 2 as M6, and 2 as M7. Efficacy and adverse events were compared between the two groups. Results The complete remission rate (CR) of B group was 70.4%, while CR in A group was 48.2%. Considering the CR, 3+10 protocol showed higher efficacy than 3+7 protocol (P< 0.05). The major adverse event was bone marrow suppression. Treatment-related mortality (TRD) in A group was 1.8%, which was lower than that in B group (2.3%). The overall survival rate in A group was 75.0%, which was lower than that in B group (86.4%, P< 0.05). Conclusions The induction therapy of 3+10 protocol and 3+7 protocol showed effectiveness for AML treatment. The 3+10 protocol showed a higher CR than 3+7 protocol with no TRD increase, indicating that the 3+10 protocol should be recommended for AML treatment in children.
ABSTRACT
The safety of decitabine as bridging treatment before allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children with refractory hematological malignancies was evaluated.All 11 cases succeeded in hematopoietic reconstitution.The main adverse reaction was hematological toxicity.Neither did infections occur,nor drug-induced liver damage and renal impairment during decitabine administration.Most cases showed grade Ⅰ-Ⅱ gastrointestinal adverse events.One case was diagnosed as severe acute graft versus host disease and died of intracranial hemorrhage on day 61 after allo-HSCT.The other 10 patients survived.Decitabine bridge is a safe regimen before allo-HSCT in children with refractory hematological malignancies.
ABSTRACT
Objective To explore the effect of parvovirus B19 (VB19) infection on pediatric leukemia patients. Methods The pediatric leukemia patients were enrolled in the study in the Children's Hospital of Soochow University. Expression levels of VB19-DNA-PCR were detected using the polymerase chain reaction. Positive patients would be monitored and treated by conventional treatment as well until VB19 gene became negative. The data was compared according to the VB19 clearance time, clinical symptoms and blood counts to evaluate the effect. Results In the 3009 samples from 824 pediatric leukemia patients, there were 36 samples (1.2%) from 12 cases (1.5%) of pediatric leukemia paients with positive VB19 infection. Among the positive patients, 11 cases (1.9%) were from 582 with acute lymphoblastic leukemia (ALL) patients and 1 (0.45%) was from 212 with acute myeloid leukemia (AML). According to the treatment stage, 3 cases were in initially diagnosed period, 2 cases in early stage of consolidation chemotherapy, 4 cases in delayed enhanced chemotherapy period, and 3 cases in maintenance chemotherapy period. According to the treatment response, 4 cases were in continuous treatment, 2 cases were sensitive to treatment, and 3 cases were drug resistant. In the drug resistance group, 2 cases developed into the pure red cell aplastic anemia (PRCA). After treatment, one was recovered from PRCA with VB19 cleared, the other one remained PRCA with continuously positive VB19. Conclusions More VB19 virus infection in pediatric ALL happened in delayed enhanced chemotherapy period. The persistent presence of VB19 infection on pediatric leukemia patients is closely related with PRCA.
ABSTRACT
Objective To evaluate the predictive role of TEL/AML1 fusion gene in protocol CCLG-ALL-2008 and to identify relevant factors influencing the outcome of ALL with TEL/AML1 fusion gene. Methods Ninety-nine patients with ALL harboring TEL/AML1 fusion gene (positive) and 329 cases without any specific fusion genes (negative) at diagnosis of B-lineage ALL from June 2008 to December 2014 were enrolled and their clinical and biological features were analyzed. Following-up ended in October 2015, the survival status was calculated by K-M curve and prognostic factors were analyzed by COX model. Results There were no differences between the two groups in age, white blood cell at the diagnostic stage, and treatment responses at 4 time points, namely, prednisone good response on day 8, M3 status of BM on D15, and the minimal residual disease (MRD) more than 1.0×10-3 on day 33 and 12th week. During the follow-up period, the relapse rate was lower in the positive group than that in the negative group (14/99 vs 69/329), the mortality rate of the negative group was twice of that in the positive group (55/329 vs 8/99). The five-year overall survival (OS) rate, relapse-free survival (RFS) rate and event-free survival (EFS) rate of the positive group were (86.1 ± 4.9)%, (80.7 ± 5.1)% and (78.9 ± 5.1)%, respectively, and (79 ±2.8)%, (72± 3.1)%, and (69.6+ 3.1)% for the negative group as well. COX regression analysis indicated that relapse and MRD level at the 12th week were independent prognostic factors on OS, RFS, and EFS (P<0.05) for the two groups. Conclusions TEL/AML1 fusion gene could be regarded as a relatively good indicator of risks in ALL children treated by CCLG-ALL-2008 protocol. ALL patients with TEL/AML1 are recommended to receive more intensive therapy including hematopoietic stem cell transplantation when the patients were high level of MRD on the 12th week after treatment.
ABSTRACT
Objective To explore the gene sequencing and prenatal diagnosis of Glanzmann thrombasthenia (GT). Methods The blood samples were drawn from one case of phenotype GT pediatric patient, patient’s parents, and one normal control. The amniotic lfuid and cord blood from the fetus of patient’s mother were collected. When the fetus was born 2 days, the blood was drawn. The coagulation routine test and platelet aggregation test were performed. The expression of platelet membrane glycoprotein (GP) IIb and GPIIIa were tested by lfow cytometry. Microsatellite technology is used to determine whether fetal cord blood is contaminated with maternal cells. The expressed region and the junctional zone between exon and introns of GPIIb and GPIIIa were ampliifed by PCR technology from blood sample of patient, patient’s parents, and fetus’s cord and 2 days after birth. The PCR products were then subjected to DNA sequencing. Results Adenosine diphosphate (ADP) cannot induce the platelet aggregation in the patient. The max rate of the platelet aggregation in the fetus’s cord blood was half of the normal. However, the max aggregation rate induced by ADP in the blood sample of parents and fetus 2 days after birth were equal to normal. The mean lfuorescence intensity (MnX) of platelet membrane GPIIb and GPIIIa in the patient were 10%and nearly zero of the normal control, respectively, while those in the parents, the fetus’s cord blood and 2 days after birth were more than 90%and 30%to 50%of the normal control. The cast-off cells in amniotic lfuid and the DNA in cord blood analysis by microsatellite technology conifrmed that the amniotic lfuid and cord blood not contaminated by maternal cells. Gene analysis showed the heterozygosis mutation in exon6 A3829→C and exon9 G42186→A of the patient’s GPIIIa led to the amino acid heterozygosis mutation in GPIIIaHis281→Tyr and Cys400→Pro. These two mutations came from the father and the mother separately. However, there was only one heterozygosis mutation in exon9 G42186→A in the cast-off cells in amniotic lfuid, the fetus’s cord and blood 2 days after birth. Conclusion This GT patient have double heterozygosis mutation. The fetus has heterozygosis mutation conifrmed after birth.
ABSTRACT
Objective To evaluate the prognostic factors in predicting relapse of childhood acute lymphoblastic leukemia (ALL) treated with CCLG-2008 protocol. Methods From December 1st 2008 and December 31st 2012, 358 patients diagnosed with ALL and treated with the CCLG-ALL 2008 protocol were enrolled in this study. All patients were followed up until September 1st, 2015. Prognostic impact of clinical features, response to treatment, biological features were analyzed and multivariate analysis of predicted value was performed by Cox-regression analysis. Results After treatment of CCLG-ALL 2008 protocol, 79 patients suffered from relapse. The relapse rate in the standard-risk, intermediate-risk and the high-risk groups were 13.3%, 17.6%, and 41.3%, respectively (P?100×109/L, non-remission in 15th day of induction (M3), the level of minimal residual disease (MRD) on 12w (12w-MRD)?>10-4 were signiifcantly higher, their corresponding hazard ratio were 3.17 (1.58?~?6.36), 1.87 (1.07?~?3.30), and 1.90 (1.12?~?3.20), respectively (P?10-4 were the independent prognostic factors for childhood B-ALL.
ABSTRACT
<p><b>OBJECTIVE</b>To compare the differences between hematopoietic reconstitution and longterm prognosis of patients with severe aplastic anemia （SAA） after HLA- matched sibling donor hematopoietic stem cell transplantation（MSD-HSCT）, Haploidentical HSCT（Haplo-HSCT）, unrelated donor allogeneic HSCT（UD-HSCT）and umbilical cord blood HSCT（UCB-HSCT）.</p><p><b>METHODS</b>In this retrospective study, 63 patients with SAA who received HSCT in the First Affiliated Hospital of Soochow University between May 2008 and December 2013 were enrolled. The subjects were divided into 4 groups according to the transplantation types. The hematopoietic reconstitution, the incidence of acute graft-versushost disease（aGVHD）and 5- year survival rate after transplantation were compared.</p><p><b>RESULTS</b>All 53 subjects who received MSD-HSCT, Haplo-HSCT and UD-HSCT achieved hematopoietic reconstitution. Of them, the recovery of neutrophil and platelet were not significantly different（P<0.05）. Patients receiving UCB-HSCT had delayed recovery of hematopoiesis, and a significantly reduced reconstruction rate, when compared with those in the other 3 groups （P<0.01）. However, 4 patients undergoing UCB- HSCT presented with autologous hematopoiesis, a period of time after the failure of hematopoietic reconstitution. The expected 5- year survival rates after MSD- HSCT, Haplo- HSCT, UD- HSCT and UCB- HSCT were 70.0%, 81.0%, 88.9% and 77.8%, respectively（P>0.05）.</p><p><b>CONCLUSION</b>MSD-HSCT, Haplo-HSCT and UD-HSCT had no statistically significance in terms of hematopoietic reconstitution or prognosis. Although hematopoietic reconstitution of UCB-HSCT was lower than other transplantation types, but no significant difference in overall prognosis. So if HLA-matched sibling donor is not available, SAA patients can choose Haplo- HSCT, UD - HSCT or UCB- HSCT with comparable efficacy to MSD- HSCT, as an alternative therapy.</p>