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Objective:To analyze the clinical efficacy and prognostic factors of intracranial primary diffuse large B-cell lymphoma (DLBCL).Methods:Clinical data of 205 patients pathologically diagnosed with intracranial primary DLBCL at Sun Yat-sen University Cancer center from March 2001 to September 2020 were retrospectively analyzed. Among them, 101 patients were male and 104 female, the median age was 54 years old. Non-germinal center B cell (GCB) subtype accounted for 74.1%(126/170). A total of 177 patients received high-dose methotrexate (HD-MTX) and 91 patients received rituximab. After induction chemotherapy, 59 patients (30.4%) achieved complete response (CR), 112 patients (57.7%) achieved partial response (PR) or stable disease (SD). A total of 83 patients received consolidation or salvage radiotherapy, and only 14 patients received autologous stem cell transplantation (ASCT). The influence of pathological type, chemotherapy, rituximab treatment, radiotherapy and radiotherapy mode, ASCT and other factors on the overall survival (OS) and progression free survival (PFS) was evaluated. The survival rate was calculated by Kaplan-Meier method. Univariate prognostic analysis was performed by log-rank test. Multivariate prognostic analysis was conducted by COX model.Results:The median follow-up time was 34 months. The 5-year OS and PFS rates were 55.6% and 44.2%, respectively. GCB subtype, chemotherapy with HD-MTX, rituximab treatment, remission status after induction chemotherapy, and radiotherapy were favorable prognostic factors for OS or PFS, in which the last three were the independent prognostic factors. Consolidation radiotherapy in patients who obtained CR after induction chemotherapy did not significantly improve survival, while salvage radiotherapy in patients who achieved PR/SD after induction chemotherapy significantly improved both OS and PFS(both P<0.01). Consolidation radiotherapy showed no significant survival difference compared with consolidation ASCT. Conclusions:The non-GCB subtype of intracranial primary DLBCL is related to poor prognosis. The addition of rituximab to HD-MTX based induction chemotherapy can improve survival. Radiotherapy is still an important treatment for intracranial primary DLBCL, and there are limitations of ASCT in practical clinical application.
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Objective:To investigate the efficacy and safety of lenalidomide combined with bortezomib and dexamethasone (RVD) in patients with newly diagnosed multiple myeloma (NDMM).Methods:A total of 100 consecutive NDMM patients treated with RVD from August 2016 to September 2020 at Peking University People′s Hospital were retrospectively analyzed, including response, drug toxicity, follow-up and survival, and subgroup analysis.Results:The median follow-up time was 19.5 (2.0-57.0) months. For patients undergoing autologous stem cell transplantation (ASCT) after RVD regimen, the objective response rate (ORR)/complete response+stringent complete response (CR+sCR)/≥very good partial response (VGPR) rates were 100%, 73.3% (33/45), 95.6% (43/45) respectively. For 54 patients not receiving transplantation, the ORR/CR+sCR/≥VGPR rates were 79.6% (43/54), 18.5% (10/54), 51.9% (28/54) respectively. As to the survival analysis, 2-year progression free survival (PFS) rates were 84.5% and 70.9% in transplant and non-transplant patients respectively ( P=0.102). Two-year overall survival (OS) rates were 100% and 80.8% in transplant and non-transplant patients respectively ( P=0.003). The common hematologic adverse events (AEs) were thrombocytopenia (33%) and neutropenia (25%). Abnormal liver function (43%) and peripheral neuropathy (24%) were recognized more as non-hematologic AEs. Conclusion:RVD as front-line regimen has high efficient response rate and acceptable safety in Chinese NDMM patients.
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Objective:To investigate the efficacy and safety of sequential therapy of targeting CD19 and CD22 chimeric antigen receptor T-cell (CAR-T) following autologous stem cell transplantation (ASCT) in treatment of renal diffuse large B-cell lymphoma (DLBCL) with central nervous system (CNS) recurrence.Methods:The clinical data of 1 renal DLBCL patient with CNS recurrence admitted to Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology in May 2019 were retrospectively analyzed. The patient received sequential therapy of targeting CD19 and CD22 CAR-T following ASCT. The relative indicators of the primary disease remission at 1, 3, 6, 12, 18 and 24 months after therapy were analyzed, and relevant literature was reviewed.Results:The male patient aged 23 years had CNS recurrence after 8 courses of R-CHOP chemotherapy and then he received sequential therapy of targeting CD19 and CD22 CAR-T following ASCT. During the process of treatment, this patient developed grade 1 cytokine release syndrome and his condition was well controlled after active treatment. The white blood cell and platetes were successfully implanted. CNS symptoms along with immature cells in cerebrospinal fluid disappeared completely. Liquid biopsy was used to dynamically monitor the residue disease of the patient and the duration of remission period lasted 26 months. This patient developed tuberculosis one year after treatment and recovered from anti-tuberculosis agents.Conclusions:Sequential therapy of targeting CD19 and CD22 CAR-T following ASCT provides a novel therapeutic approach for renal DLBCL with CNS recurrence. Especially for patients who are neither sensitive to conventional chemotherapy nor CAR-T therapy alone, this regimen may improve remission rate and survival.
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Background & objectives: Survival of patients with multiple myeloma (MM) has improved in the past two decades following use of novel agents and autologous stem cell transplantation. To determine predictors of long-term outcome, data of MM patients who underwent autologous stem cell transplantation (ASCT) at a tertiary care centre in north India were retrospectively analyzed. Methods: Between 1995 and 2016, 349 MM patients underwent ASCT. Patients' median age was 52 yr, ranging from 29 to 68 yr, 68.2 per cent were males. Thirty three per cent patients had international staging system (ISS) Stage III and 68.5 per cent had received novel agents-based induction. High-dose melphalan (200 mg/m2) was used for conditioning; patients with renal insufficiency (estimated glomerular filtration rate <40 ml/min) received melphalan 140-150 mg/m2. Results: Post-transplant, 317 of 349 (90.8%) patients responded; complete [complete response (CR)] ?213 (61%)], very good partial response (VGPR) ?62 (17.8%) and PR in 42 (12%)]. Induction with novel agents, pre-transplant chemosensitive disease, transplant in first remission and serum albumin (?3.5 g/dl) were predictors of significant response. At a median follow up of 73 months, median overall survival (OS) was 90 months [95% confidence interval (CI) 70.8-109.2], and progression-free survival (PFS) was 41 months (95% CI 33.0-49.0). On multivariate analysis, achievement of CR post-transplant, transplant in first remission, ISS Stages I and II (vs. III), absence of extramedullary disease and serum albumin ?3.5 g/dl were predictors of prolonged OS. For PFS, achievement of post-transplant CR and transplant in first remission were predictors of superior outcome. Interpretation & conclusions: Treatment with novel agents, achievement of complete remission post-transplant, ISS Stages I and II, absence of extramedullary disease and transplant in first remission were predictors of long-term survival for patients with MM.
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Objective@#To study the efficacy, safety and long-term outcomes of integrated strategy of bortezomib-based induction regimens followed by autologous hematopoietic stem cell (ASCT) and maintenance therapy in Chinese multiple myeloma (MM) patients.@*Methods@#200 MM patients receiving integrated strategy of bortezomib--based induction regimens followed by ASCT and maintenance therapy were retrospectively and prospectively analyzed from December 1. 2006 to April 30. 2018.@*Results@#The complete remission rates (CR) and better than very good partial remission rates (VGPR) after induction therapy, transplantation and maintenance therapy were respectively 31% and 75.5%, 51.8% and 87.7%,73.6% and 93.4%. There was no difference between 4 cycles and more than 5 cycles induction chemotherapy. The negative rate of MRD detection by flow cytometry was 17.6% and 38.2% respectively after induction and 3 months after transplantation. The negative rate of MRD gradually increased during the maintenance therapy. The success rate of high dose CTX combined with G-CSF mobilization was 95.5% and transplantation related mortality (TRM) was zero. The median time to progress (TTP) was 75.3 months and the median overall survival (OS) was 99.5 months. TTP of patients obtaining CR and negative MRD after induction were longer that those of no CR and positive MRD. TTP and OS of patients receiving triple-drug induction and ASCT in early stage were longer than those of double-drug induction and ASCT in late stage. LDH≥240 U/L, high risk cytogenetics, ISS II+III stage and HBsAg positive were prognostic factors at diagnosis. However, only MRD and high risk cytogenetics were independent prognostic factors after transplantation and maintenance therapy. The clinical characteristics of patients of TTP ≥6 years were listed below: light-chain type M protein, ISS I stage, normal level of hemoglobin and platelet, normal LDH, HBsAg negative, chromosome 17p-negative, good response and sustained good response.@*Conclusions@#Integrated strategy of bortezomib-based induction regimens followed by ASCT and maintenance therapy can significantly improve the short-term and long-term efficacy. The prognostic factors of TTP in different disease stages were different. Response to treatment, especially MRD, played a more important role in prognostic factors.
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Objective@#To compare the efficacy, response and survival between high-dose melphalan (HDM) and cyclophosphamide+ etoposide+ busulfan (CVB) as the conditioning regimen in autologous stem cell transplantation (ASCT) for newly diagnosed multiple myeloma (NDMM) .@*Methods@#Retrospectively enrolled 123 consecutive NDMM patients who had received PAD induction with subsequent ASCT from Jan 2011 to Aug 2017. The CVB group and HDM group had 82 and 41 patients respectively.@*Results@#①No differences existed between these 2 groups in non-hematological side effects. ②Patients of CVB group had faster neutrophil and platelet engraftment time, with the median neutrophil engraftment time of 10 (9-35) day vs 11 (9-12) day for patients of HDM group (z=-3.433, P=0.001) , and with median platelet engraftment time of 11 (7-55) day vs 13 (10-35) day for patients of HDM group (z=-3.506, P<0.001) . CVB group entered neutropenia and severe thrombocytopenia more earlier than the HDM group, resulting similar neutropenia duration and severe thrombocytopenia duration between the CVB group and HDM group. However, patients of CVB group had significantly longer fever persistent time and antibiotic administration time. ③The response rate was significantly lower in patients of CVB group vs. patients of HDM group (9/46 vs 14/28, P=0.021) . Further, the minimal residual disease (MRD) negative rate at 3rd month post-transplantation seemed to be lower in CVB group than that in HDM group (31.7%vs 48.8%, P=0.065) . ④Both the univariate and multivariate analysis showed that HDM and CVB groups had similar duration to progression (TTP) (P=0.619) and overall survival (OS) (P=0.295) .@*Conclusion@#HDM conditioning regimen is superior to CVB regimen in hematological side effects, tumor burden reduction and administration convenience. However, these two regimen had similar TTP and OS in MM patients receiving ASCT.
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BACKGROUND/AIMS: Data on dexamethasone, cytarabine, and cisplatin (DHAP) as a mobilization regimen, compared to high-dose cyclophosphamide (HDC), for up-front autologous stem cell transplantation (ASCT) in non-Hodgkin’s lymphoma (NHL) is limited. METHODS: Consecutive patients with aggressive NHL treated with cyclophosphamide, doxorubicin, vincristine, and prednisone (CHOP) or rituximab-CHOP who underwent chemomobilization using HDC or DHAP plus granulocyte-colony stimulating factor (G-CSF) for up-front ASCT were enrolled from three institutions between 2004 and 2014. RESULTS: Ninety-six patients (57 men) were included. Sixty-five patients (67.7%) received HDC; and 31 (32.3%), DHAP. The total CD34+ cells mobilized were significantly higher in patients receiving DHAP (16.1 vs. 6.1 × 106/kg, p = 0.001). More patients achieved successful mobilization with DHAP (CD34+ cells ≥ 5.0 × 106/kg) compared to HDC (87.1% vs. 61.5%, respectively; p = 0.011), particularly within the first two sessions of apheresis (64.5% vs. 32.3%, respectively; p = 0.003). Mobilization failure rate (CD34+ cells < 2.0 × 106/kg) was significantly higher in patients receiving HDC (20.0% vs. 3.2%, p = 0.032). On multivariate analysis, the DHAP regimen (odds ratio, 4.12; 95% confidence interval, 1.12 to 15.17) was an independent predictor of successful mobilization. During chemomobilization, patients receiving HDC experienced more episodes of febrile neutropenia compared to patients receiving DHAP (32.3% vs. 12.9%, p = 0.043). CONCLUSIONS: The DHAP regimen was associated with a significantly higher efficacy for stem cell mobilization and lower frequency of febrile neutropenia. Therefore, DHAP plus G-CSF is an effective for mobilization in patients with aggressive NHL who were candidates for up-front ASCT.
Subject(s)
Humans , Blood Component Removal , Cisplatin , Cyclophosphamide , Cytarabine , Dexamethasone , Doxorubicin , Febrile Neutropenia , Granulocyte Colony-Stimulating Factor , Hematopoietic Stem Cell Mobilization , Lymphoma , Lymphoma, Non-Hodgkin , Multivariate Analysis , Prednisone , Stem Cell Transplantation , Stem Cells , VincristineABSTRACT
The prognosis of brain tumors in children has improved for last a few decades. However, the prognosis remains dismal in patients with recurrent brain tumors. The outcome for infants and young children in whom the use of radiotherapy (RT) is very limited because of unacceptable long-term adverse effect of RT remains poor. The prognosis is also not satisfactory when a large residual tumor remains after surgery or when leptomeningeal seeding is present at diagnosis. In this context, a strategy using high-dose chemotherapy and autologous stem cell transplantation (HDCT/auto-SCT) has been explored to improve the prognosis of recurrent or high-risk brain tumors. This strategy is based on the hypothesis that chemotherapy dose escalation might result in improvement in survival rates. Recently, the efficacy of tandem HDCT/auto-SCT has been evaluated in further improving the outcome. This strategy is based on the hypothesis that further dose escalation might result in further improvement in survival rates. At present, the number of studies employing tandem HDCT/auto-SCT for brain tumors is limited. However, results of these pilot studies suggest that tandem HDCT/auto-SCT may further improve the outcome. In this review, we will summarize our single center experience with tandem HDCT/auto-SCT for recurrent or high-risk brain tumors.
Subject(s)
Child , Humans , Infant , Brain Neoplasms , Brain , Diagnosis , Drug Therapy , Neoplasm, Residual , Pilot Projects , Prognosis , Radiotherapy , Stem Cell Transplantation , Stem Cells , Survival RateABSTRACT
The prognosis of brain tumors in children has improved for last a few decades. However, the prognosis remains dismal in patients with recurrent brain tumors. The outcome for infants and young children in whom the use of radiotherapy (RT) is very limited because of unacceptable long-term adverse effect of RT remains poor. The prognosis is also not satisfactory when a large residual tumor remains after surgery or when leptomeningeal seeding is present at diagnosis. In this context, a strategy using high-dose chemotherapy and autologous stem cell transplantation (HDCT/auto-SCT) has been explored to improve the prognosis of recurrent or high-risk brain tumors. This strategy is based on the hypothesis that chemotherapy dose escalation might result in improvement in survival rates. Recently, the efficacy of tandem HDCT/auto-SCT has been evaluated in further improving the outcome. This strategy is based on the hypothesis that further dose escalation might result in further improvement in survival rates. At present, the number of studies employing tandem HDCT/auto-SCT for brain tumors is limited. However, results of these pilot studies suggest that tandem HDCT/auto-SCT may further improve the outcome. In this review, we will summarize our single center experience with tandem HDCT/auto-SCT for recurrent or high-risk brain tumors.
Subject(s)
Child , Humans , Infant , Brain Neoplasms , Brain , Diagnosis , Drug Therapy , Neoplasm, Residual , Pilot Projects , Prognosis , Radiotherapy , Stem Cell Transplantation , Stem Cells , Survival RateABSTRACT
BACKGROUND: High-dose melphalan (HDMEL) represents the standard conditioning regimen before autologous stem cell transplant (ASCT) in multiple myeloma (MM), but recent updates have suggested combination of melphalan with bulsulfan (BUMEL) is also associated with favorable outcomes. We performed the current study to address the lack of comparative studies between the two conditioning regimens in Asian populations. METHODS: Using the Korean National Health Insurance and Korean Health Insurance Review and Assessment Service databases, 1,304 patients newly diagnosed with MM undergoing ASCT between January 2010 and December 2014 were identified. Patients were divided according to conditioning regimen (HDMEL vs. BUMEL), and after case matching, 428 patients undergoing HDMEL conditioning were compared to 107 patients undergoing BUMEL conditioning with respect to clinical course and treatment outcomes. RESULTS: The 3-year progression-free survival (PFS) was 52.5% for the HDMEL conditioning group versus 70.3% for the BUMEL conditioning group (P=0.043). The 3-year overall survival (OS) was 82.0% versus 83.5% (P=0.525), respectively. Although not statistically significant, BUMEL conditioning was associated with more platelet transfusion, while HDMEL was associated with more granulocyte colony stimulating factor support. In multivariate analysis, BUMEL conditioning was not inferior to HDMEL conditioning in regard to both PFS and OS. CONCLUSION: Our study confirmed that BUMEL is an effective and well-tolerated alternative to HDMEL conditioning, with better PFS.
Subject(s)
Humans , Asian People , Busulfan , Colony-Stimulating Factors , Disease-Free Survival , Granulocytes , Insurance, Health , Melphalan , Multiple Myeloma , Multivariate Analysis , National Health Programs , Platelet Transfusion , Stem Cell Transplantation , Stem CellsABSTRACT
With the aim to investigate the outcome of tandem high-dose chemotherapy and autologous stem cell transplantation (HDCT/auto-SCT) for high-grade gliomas (HGGs), we retrospectively reviewed the medical records of 30 patients with HGGs (16 glioblastomas, 7 anaplastic astrocytomas, and 7 other HGGs) between 2006 and 2015. Gross or near total resection was possible in 11 patients. Front-line treatment after surgery was radiotherapy (RT) in 14 patients and chemotherapy in the remaining 16 patients including 3 patients less than 3 years of age. Eight of 12 patients who remained progression free and 5 of the remaining 18 patients who experienced progression during induction treatment underwent the first HDCT/auto-SCT with carboplatin + thiotepa + etoposide (CTE) regimen and 11 of them proceeded to the second HDCT/auto-SCT with cyclophosphamide + melphalan (CyM) regimen. One patient died from hepatic veno-occlusive disease (VOD) during the second HDCT/auto-SCT; otherwise, toxicities were manageable. Four patients in complete response (CR) and 3 of 7 patients in partial response (PR) or second PR at the first HDCT/auto-SCT remained event free: however, 2 patients with progressive tumor experienced progression again. The probabilities of 3-year overall survival (OS) after the first HDCT/auto-SCT in 11 patients in CR, PR, or second PR was 58.2% ± 16.9%. Tumor status at the first HDCT/auto-SCT was the only significant factor for outcome after HDCT/auto-SCT. There was no difference in survival between glioblastoma and other HGGs. This study suggests that the outcome of HGGs in children and adolescents after HDCT/auto-SCT is encouraging if the patient could achieve CR or PR before HDCT/auto-SCT.
Subject(s)
Adolescent , Child , Humans , Astrocytoma , Brain Neoplasms , Carboplatin , Cyclophosphamide , Drug Therapy , Etoposide , Glioblastoma , Glioma , Hepatic Veno-Occlusive Disease , Medical Records , Melphalan , Radiotherapy , Retrospective Studies , Stem Cell Transplantation , Stem Cells , ThiotepaABSTRACT
El linfoma no hodgkiniano parecido al Burkitt es considerado una neoplasia de células B altamente agresiva, con un alto índice de proliferación. Las presentaciones en forma de masas tumorales abdominales no son muy frecuentes y están poco documentadas en la literatura. Se reporta un caso poco usual de un paciente adulto joven con linfoma no hodgkiniano parecido al Burkitt, con infiltración del colon derecho, tratado con poliquimioterapia asociada a la administración de anticuerpo monoclonal anti-CD20, con lo que se alcanzó la remisión completa de la enfermedad. Posteriormente esta respuesta se consolidó con esquemas intensivos de quimioterapia y trasplante de progenitores hematopoyético autólogo. Actualmente se mantiene asintomático y sin tratamiento(AU)
The Burkitt-like lymphoma is considered a highly aggressive B-cell lymphoma, with a high proliferative rate. Presentation with extensive mass intestinal involvement is rare and poorly documented in the literature. We report the case of a young adult with the Burkitt-like lymphoma, and presenting extensive infiltration of the right colon, who receives chemotherapy treatment associated with administration of anti-CD20 monoclonal antibody, achieves complete remission of the illness and consolidation with intensive chemotherapy with autologous stem cell transplantation. Currently the patient is asymptomatic and without treatment(AU)
Subject(s)
Humans , Male , Adult , Lymphoma, Non-Hodgkin/complications , Colonic Neoplasms/surgery , Hematopoietic Stem Cell Transplantation/methods , Antibodies, Monoclonal/therapeutic useABSTRACT
Despite increasing evidence that high-dose chemotherapy and autologous stem cell transplantation (HDCT/auto-SCT) might improve the survival of patients with high-risk or recurrent solid tumors, therapy effectiveness for bone and soft tissue sarcoma treatment remains unclear. This study retrospectively investigated the feasibility and effectiveness of HDCT/auto-SCT for high-risk or recurrent bone and soft tissue sarcoma. A total of 28 patients (18 high-risk and 10 recurrent) underwent single or tandem HDCT/auto-SCT between October 2004 and September 2014. During follow-up of a median 15.3 months, 18 patients exhibited disease progression and 2 died of treatment-related toxicities (1 veno-occlusive disease and 1 sepsis). Overall, 8 patients remained alive and progression-free. The 3-year overall survival (OS) and event-free survival (EFS) rates for all 28 patients were 28.7% and 26.3%, respectively. In the subgroup analysis, OS and EFS rates were higher in patients with complete or partial remission prior to HDCT/auto-SCT than in those with worse responses (OS, 39.1% vs. 0.0%, P = 0.002; EFS, 36.8% vs. 0.0%, P < 0.001). Therefore, careful selection of patients who can benefit from HDCT/auto-SCT and maximal effort to reduce tumor burden prior to treatment will be important to achieve favorable outcomes in patients with high-risk or recurrent bone and soft tissue sarcomas.
Subject(s)
Adolescent , Child , Child, Preschool , Female , Humans , Infant , Male , Young Adult , Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Bone Neoplasms/mortality , Disease-Free Survival , Follow-Up Studies , Retrospective Studies , Sarcoma/mortality , Soft Tissue Neoplasms/mortality , Stem Cell Transplantation , Survival Rate , Transplantation, Autologous , Treatment OutcomeABSTRACT
Autologous stem cell transplantation (ASCT) is the standard frontline therapy for newly diagnosed multiple myeloma (MM) in patients younger than 65 years in the era of conventional chemotherapy. The use of novel drug-based chemotherapy in the in-duction, consolidation, and maintenance phases of chemotherapy has significantly improved the response rates of patients. Thus, wheth-er or not ASCT is still necessary in the era of new drugs has become controversial. Currently available data supported that ASCT should be the frontline therapy for qualified newly diagnosed MM patients and that new drugs may be applied before and after ASCT to further improve the response rate and prolong the progression-free survival of patients. Further prospective clinical trials should be conducted to clarify the role of ASCT in MM and optimize the treatment strategies involving ASCT in the era of new drugsto cure myeiona.
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Multiple myeloma (MM) is malignant plasma cell clonal disease.Autologous stem cell transplantation (ASCT) strengthen the advantages of the high-dose chemotherapy in treatment of MM,which achieves long-term survival in some patients.Even in the era of targeted novel agents,ASCT can still obviously increase the response rate of treatment.To improve the therapeutic efficacy,targeted novel agents were administrated before and after ASCT.However,targeted novel agents still cannot replace ASCT therapy in the treatment of MM.The direction of future research is to find more reasonable,effective and low toxic treatment.
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To observe the effect of autologous stem cell transplantation in diabetic retinopathy. ●METHODS:Totally 58 cases (116 eyes) who underwent autologous stem cell transplantation were confirmed as no diabetic retinopathy (18 eyes), mild non-proliferative diabetic retinopathy (NPDR) (41 eyes), mid-level NPDR (51 eyes); severe NPDR (6 eyes) by ophthalmoscope directly or indirectly and fluorescence fundus angiography (FFA ). Follow - up was 6 - 12mo, the changes of retinopathy were observed. ●RESULTS: The total effective rate of vision and retinopathy was 84. 4%, 76. 7%. The results of severe NPDR was statistically worse than the other groups ( P ●CONCLUSlON: The stable blood glucose level and improved pancreatic function after autologous stem cell transplantation might be helpful in diabetic retinopathy, the long effects need to be researched further.
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Transplantation-associated thrombotic microangiopathy (TA-TMA) is an uncommon but devastating complication in patients who undergo hematopoietic stem cell transplantation (SCT). However, the optimal treatment strategy for TA-TMA is unclear. We report a rare case of TA-TMA in a 39-month-old boy who underwent tandem autologous SCT (autoSCT) for high-risk medulloblastoma. TA-TMA developed 64 days after the second autoSCT with microangiopathic hemolytic anemia, fever, renal impairment, acute respiratory distress syndrome and posterior reversible encephalopathy syndrome. The patient recovered after plasmapheresis and methylprednisolone therapy. He had mild to moderate deficiency of a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS-13). The patient's clinical course would suggest that plasmapheresis and methylprednisolone therapy could be a treatment option for TA-TMA. Early intervention is needed to aid the recovery of the patient who is suspected for TA-TMA.
Subject(s)
Child, Preschool , Humans , Male , Anemia, Hemolytic , Early Intervention, Educational , Fever , Hematopoietic Stem Cell Transplantation , Medulloblastoma , Methylprednisolone , Pediatrics , Plasmapheresis , Posterior Leukoencephalopathy Syndrome , Respiratory Distress Syndrome , Stem Cell Transplantation , Thrombospondins , Thrombotic MicroangiopathiesABSTRACT
BACKGROUND: Multiple myeloma (MM) is a heterogeneous and ultimately fatal disease. Risk stratification using prognostic biomarkers is crucial to individualize treatments. We sought to investigate the role of CD99, a transmembrane protein highly expressed in many hematopoietic cells including subpopulations of normal and neoplastic plasma cells, for MM risk stratification. METHODS: CD99 expression was measured in paraffin samples of bone marrow and extramedullary biopsies of 170 patients with MM. Patients were divided into those with high score (moderately and strongly positive) and low score (negative and weakly positive), with all staining being cytoplasmic and/or membranous. RESULTS: High anti-CD99 immunostaining was observed in 72 of 136 (52.9%) bone marrow biopsies and 24 of 87 (27.6%) extramedullary biopsies in MM. High CD99 expression of extramedullary specimens was associated with significantly longer overall survival (OS; p=.016). High CD99 expression of extramedullary specimens was also associated with better prognosis in the nonautologous stem cell transplantation group of MM patients (p=.044). In multivariate analysis, International Staging System stage was an independent prognostic factor, whereas CD99 expression was no longer statistically significant. CONCLUSIONS: Expression of CD99 in extramedullary specimens was correlated with longer OS, suggesting that CD99 may be a helpful immunohistochemical marker for risk stratification.
Subject(s)
Humans , Biomarkers , Biopsy , Bone Marrow , Cytoplasm , Multiple Myeloma , Multivariate Analysis , Paraffin , Plasma Cells , Prognosis , Stem Cell TransplantationABSTRACT
Background and purpose:The patients with aggressive lymphoma who have a poor prognosis and unlikely to be cured with conventional chemotherapy. This study was aimed to evaluate the effect of high-dose etoposide in mobilization followed auto-SCT in treating refractory lymphoma. Methods:40 patients [median age 33 (13-61) years] with refractory non-Hodgkin’s lymphoma (NHL, n=32) or Hodgkin’s lymphoma (HD, n=8) received high-dose etoposide [VP16 10-15 mg/(kg·d)×2 d] in mobilization in our center. Remission status prior to mobilization was PD (n=40). The use of such granulocyte colony-stimulating factor [G-CSF, 5-10μg/(kg·d)] mobilized peripheral blood stem cells (PBSC) after high-dose etoposide until the end of leukapheresis. Peripheral blood stem cell was collected and frozen in-80℃refrigerator. All these patients received auto peripheral blood stem cell transplantation (auto-PBSCT). Conditioning regimen was BEAM (n=19, 47.5%) or CBV (n=21, 52.5%). Results:Twenty-eight pa-tients (70%) were assessable for response after high-dose etoposide at a median pretreatment time of 39 days (range 17-172 days), 12 patients (30%) had no response. Median follow-up of 28 (4-66) months, 16 patients (40%) reached CR after auto-PBSCT. Fifteen of the 28 patients (53.6%) who had response to high-dose etoposide reached CR, 4 patients (14.3%) reached PR, 9 patients (32.1%) succumb to progression of disease. One of the 12 patients (8.3%) who had no response to high-dose etoposide reached CR, 1 patients (8.3%) reached PR, 10 patients (83.4%) succumb to progression of disease. The estimated 1-year OS and EFS were 69%and 56.7%respectively, 2-years OS and EFS were 63%and 52%respectively. The prognosis of the patients who had no response to etoposide was poor. The estimated 1-year OS and EFS were 25%and 16.7%respectively. Two group of comparison differences have statistics signiifcance (P<0.01). Conclusion: High-dose etoposide could be used in refractory lymphoma as rescue therapy in mobilization. It can increase the EFS and OS of patients who had response. The hematopoietic stem cells collection and hematopoietic reconstitution are not affected by etoposide.
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Background and purpose: High-dose chemotherapy followed by autologous stem cell transplantation (auto-HSCT) is considered as the ifrst line treatment for patients with relapse/refractory lymphoma after conventional chemotherapy. However, most of these patients still relapse the second time. The purpose of this study was to analyze the efifcacy of the consolidation chemotherapy after autologous stem cell transplantation (HSCT) refractory/relapse lymphoma in high risk. Methods:A total of 38 patients with relapsed/refractory lymphoma including Hodgkin lymphoma (HL) and non-Hodgkin lymphoma (NHL) were included, who were underwent auto-HSCT in our transplan-tation department from Jan. 2010 to Dec. 2013. In treatment group, 19 patients received 2 courses of consolidation che-motherapy after auto-HSCT every 2 to 3 months, with the regimen of mini-BEAM or modiifed mini-CBV. Another 19 patients had no chemotherapy after auto-HSCT as control group. Results:The median follow-up duration was 17.2 and 7.5 months in the treatment and control group respectively. The follow-up data demonstrated prolonged progression-free survival (PFS) in the treatment group than the control group [24.7 months vs 7.8 months, P=0.029 under intend-to-treat analysis ITT;24.7 months vs 5.2 months, P=0.01 under per protocol analysis(pp)]. There is also a trend of improved overall survival (OS) in the treatment group (P=0.055, ITT). Conclusion:Consolidation chemotherapy after auto-HSCT for refractory/relapsed lymphoma patients delay the relapse and tend to improve the overall relapse rate.