OBJECTIVETo study the treatment and outcome of childhood endodermal sinus tumor.

METHODSThe clinical data of twelve children with endodermal sinus tumor between April 2000 and July 2013 were reviewed. The basic demographics, stages of the lesion and the treatment outcome were analyzed. Of the twelve patients, seven were boys and five were girls. The age of the disease onset was between 1 and 3.3 years, except one in 11 years. Two patients were in Brodeur Stage I, four in Stage II, two in Stage III, and four in Stage IV. One patient underwent surgery alone, one underwent surgery plus a combination therapy with vincristine, actinomycin and cyclophosphamide (VAC), and the other ten were treated by surgery with the use of cisplatin, etoposide and bleomycin (PEB) before or after the operation.

RESULTSEleven patients were successfully followed up and ten were alive. The length of survival was 4.5 to 66 months in the 10 patients. In the 10 patients treated with PEB before or after surgery, 8 achieved complete remission, one achieved partial remission and one was not followed up. The major complications associated with the PEB regimen included myelosuppression and gastrointestinal upset symptoms and no late toxicity was observed.

CONCLUSIONSPreoperative or postoperative administration of PEB may be an effective and safe management modality for childhood endodermal sinus tumor. Nevertheless, further validation is warranted in prospective studies involving a larger sample size.

Antineoplastic Combined Chemotherapy Protocols ; therapeutic use ; Child ; Child, Preschool ; Combined Modality Therapy ; Endodermal Sinus Tumor ; mortality ; pathology ; therapy ; Female ; Humans ; Infant ; Male ; Neoplasm Staging ; alpha-Fetoproteins ; analysis

So far treatment of advanced neuroblastoma is still difficult, due to its high malignancy. Currently comprehensive therapies, including high-dose multi-drug chemotherapy, surgery, stem cell transplantation, radiation, biological therapy and immune therapy as well as target therapy dominant the treatment of this disease, and we hereby introduce the latest development of treatment protocols for this disease.
Combined Modality Therapy ; Female ; Humans ; Male ; Neoplasm Recurrence, Local ; therapy ; Neuroblastoma ; therapy

OBJECTIVETo investigate the clinical manifestations, diagnosis, and treatment of peripheral primitive neuroectodermal tumor (pPNET) in children and the survival of patients treated with the CCG7942/POG9354 protocol.

METHODSA retrospective analysis was performed on the clinical data of 10 patients with pPNET admitted from October 2008 to October 2013. Of the 10 patients, 3 had metastasis, while others had no metastasis. The 7 patients without metastasis were treated with the Children's Cancer Study Group 7942 (CCG7942) protocol, and the other 3 patients with metastasis with the Pediatric Oncology Group 9354 (POG9354) protocol. The therapeutic response and chemotherapy-related toxicities were evaluated by WHO criteria and Common Terminology Criteria for Adverse Events (version 4.0).

RESULTSIn the 7 patients treated with the CCG7942 protocol, 4 achieved a complete remission (CR), 1 had stable disease, 2 developed progressive disease (PD), and 2 had recurrence. In the 3 patients treated with the POG9354 protocol, 1 achieved a CR, 2 developed PD, 2 had recurrence, and 2 died. For the 7 patients without metastasis, the survival time was 5-60 months, and the event-free survival rate was 71%. For the 3 patients with metastasis, the survival time was 13-25 months, and the event-free survival rate was 33%. All patients developed grade 4 bone marrow suppression, and other grade 1-2 toxicities, including gastrointestinal reactions, liver function impairment, and renal function impairment, were also seen.

CONCLUSIONSCCG7942 protocol is effective and safe for children with non-metastatic pPNET. However, POG9354 protocol has unsatisfactory efficacy in children with metastatic pPNET, so further studies are needed to improve the therapy for this disease.

Antineoplastic Combined Chemotherapy Protocols ; adverse effects ; therapeutic use ; Child ; Child, Preschool ; Clinical Protocols ; Female ; Humans ; Infant ; Male ; Neuroectodermal Tumors, Primitive, Peripheral ; drug therapy ; Retrospective Studies

This study was purposed to assess the effectiveness of haploidentical hematopoietic stem cell transplantation (HSCT) without in vitro T cell depletion for the treatment of severe aplastic anemia (SAA) in children. Two children with SAA/very SAA (VSAA) received T cell-depleted HSCT from their fathers with 2 loci mismatched in our center between October 2010 and March 2013. During 4 months after onset, both failed in treatment of cyclosporine (CsA)+ granulocyte colony stimulating factor (G-CSF), had active or serious infections, were transfusion dependent and lacked HLA-identical sibling donors and unrelated donors. The conditioning regimen before HSCT included fludarabine, cyclophosphamide and thymoglobulin. The source of grafts was a combination of G-CSF mobilized peripheral blood hematopoietic stem cells and BM. The recipients received CsA, mycophenolate mofetil (MMF) and short-term MTX for GVHD prophylaxis. Both children with SAA achieved 100% donor myeloid engraftment. Neutrophil engraftment occurred at day 12 and day 18 after transplant respectively. Platelet engraftment occurred at day 17 and day 26 after transplantation respectively. Two patients all developed grade I acute graft versus host disease (GVHD), one of which evolved into chronic limited GVHD well-controlled. Both have survived for two years after transplantation with 100% donor myeloid engraftment and effective lymphoid reconstitution. In conclusion, these limited cases suggest that haploidentical HSCT for children with SAA without a HLA-identical sibling donor and unrelated donor may be feasible. Further prospective clinical study is required to increase the overall survival (OS) by decreasing GVHD while maintaining stable engraftment.
Anemia, Aplastic ; therapy ; Child ; Child, Preschool ; Female ; Haploidy ; Hematopoietic Stem Cell Transplantation ; Humans ; Male ; Transplantation, Homologous

OBJECTIVETo evaluate the clinical efficacy of the ARAR0331 protocol for treatment of childhood nasopharyngeal carcinoma.

METHODSThe clinical data of eight children with nasopharyngeal carcinoma between May 2004 and May 2012 were retrospectively studied. The eight patients included six boys and two girls, and the onset age was between 3 and 13 years. Six patients were in AJCC Stage Ⅲ, one was in StageⅡA and one was in Stage ⅣA. One patient had been treated with combined radiotherapy and chemotherapy which mainly included EAP, BEP and EA. The other seven patients had been treated with the ARAR0331 protocol provided by the America Children's Oncology Group (COG).

RESULTSThe patient who had been treated with combined radiotherapy and chemotherapy developed multiple bony metastasis during the chemotherapeutic period. Four out of seven patients who had been treated with ARAR0331 protocol achieved complete remission, and two achieved partial remission. The seven patients were followed-up from 8 to 75 months and the survival rate was 100%. The ARAR0331 protocol treatment-related complications included radiodermatitis, mucocitis and nausea. Late toxicity was not found.

CONCLUSIONSBased on the limited cases, ARAR0331 protocol appears to be effective and safe for childhood nasopharyngeal carcinoma.

Adolescent ; Carcinoma ; Chemoradiotherapy ; adverse effects ; Child ; Child, Preschool ; Female ; Humans ; Male ; Nasopharyngeal Neoplasms ; pathology ; therapy ; Neoplasm Staging ; Retrospective Studies

Objective To investigate whether knockingdown Foxp3 affects the immunological function of neuroblastoma cells.Methods Different siRNAs of Foxp3 were designed and the one that effectively inhibited Foxp3 gene expression was selected by real-time polymerase chain reaction and by fluorescence activated cell sorter analysis.Effects of Foxp3 knocking down on the expression of co-stimulatory molecule CD86 were also examined.Finally,neuroblastoma cells silenced of Foxp3 expression were co-cultured with human peripheral blood monouclear cells and the activities of T cells were examined.Results Expression of Foxp3 in SK-N-BE2 cells could be efficiently knocking down in both mRNA and protein levels.Knocking down Foxp3 increased the expression of CD86 and moderately increased IFN-γand IL-17 expressions,and significantly inhibited the expressions of TGF-β and IL-10.Conclusion Silencing Foxp3 expression in neuroblastoma SK-N-BE2 cells reverses its immune evasion mechanisms,which suggests that this is a new strategy for treating this kind of diseases.

Child, Preschool ; Chromosomes, Human, Pair 11 ; Female ; Humans ; Kidney Neoplasms ; genetics ; Ring Chromosomes ; Wilms Tumor ; genetics

OBJECTIVETo study the clinical characteristics, treatment and outcome of childhood rhabdomyosarcoma.

METHODSThe clinical data of 23 children with rhabdomyosarcoma from January, 1998 to October, 2008 were retrospectively reviewed.

RESULTSOf the 23 cases, 15 were male and 8 were female, with a mean age of 5 years old (7 months to 12 years old). Based on the American IRS staging system, 2 cases were in stage I, 4 cases in stage II, 8 cases in stage III, and 9 were in stage IV. The primary sites were found in head and neck (14 cases), extremities (4 cases), bladder (2 cases), kidney (1 case), post-peritoneum (1 case) and bile duct (1 case). All of the children were confirmed with rhabdomyosarcoma by biopsy and immunohistochemistry. The clinical manifestations were related to the tumor tissues-induced space occupying, compression and erosion and were aspecific. The patients in different IRS stages were given different treatment regimens. The chemotherapy regimens VDCA, VAC or VadrC were used before 2002. After 2002, the Children's Oncology Group (COG) protocol was employed. The two-year survival rate was 63% in 19 patients who received a combination of surgery, chemotherapy and radiotherapy, but none of 4 patients who received a surgery alone or a combination of surgery and chemotherapy or radiotherapy survived more than two years.

CONCLUSIONSThe clinical manifestations of childhood rhabdomyosarcoma are not specific. A combination therapy including surgery, chemotherapy and radiation is effective to the improvement of the survival rate in children with rhabdomyosarcoma.

Child ; Child, Preschool ; Combined Modality Therapy ; Female ; Humans ; Infant ; Male ; Retrospective Studies ; Rhabdomyosarcoma ; mortality ; therapy

OBJECTIVETo investigate whether neuroblastoma cells LA-N-6 express Foxp3 and whether the expression of Foxp3 is sensitive to chemotherapy by cyclophosvnamide (CTX)and pirarubicin (THP).

METHODSExpression of Foxp3 on LA-N-6 cells was examined by flow cytometry analysis. The dose-effects of chemotherapy drugs including CTX and THP on LA-N-6 cells were investigated by MTT assay. The effects of CTX and THP on Foxp3 expression were examined by flow cytometry and real-time PCR assays.

RESULTSFlow cytometry analysis showed that LA-N-6 cells expressed Foxp3 at a high level. At sub-optimal concentration, chemotherapy drugs CTX and THP significantly down-regulated expression of Foxp3 on LA-N-6 cells at protein level (P<0.05). CTX also decreased the expression of Foxp3 at mRNA level (P<0.05). CONCLSUSIONS: Neuroblastoma cells LA-N-6 express Foxp3 at a high level, which can be suppressed by chemotherapy drugs CTX and THP. These data suggest that chemotherapy might suppress the growth and metastasis of tumor cells partially through inhibiting the expression of Foxp3.

Antineoplastic Agents ; pharmacology ; Cell Line, Tumor ; Cyclophosphamide ; pharmacology ; Doxorubicin ; analogs & derivatives ; pharmacology ; Flow Cytometry ; Forkhead Transcription Factors ; analysis ; antagonists & inhibitors ; genetics ; Humans ; Neuroblastoma ; drug therapy ; immunology ; pathology ; Reverse Transcriptase Polymerase Chain Reaction

Child ; Follow-Up Studies ; Humans ; Neoplasm Recurrence, Local ; Neuroblastoma ; Recurrence ; Treatment Outcome