scholarly journals Choroid plexus carcinoma in children

2021 ◽  
Vol 20 (1) ◽  
pp. 66-74
Author(s):  
A. F. Valiakhmetova ◽  
L. I. Papusha ◽  
A. V. Sanakoeva ◽  
L. V. Shishkina ◽  
O. I. Budanov ◽  
...  
Keyword(s):  
Choroid Plexus ◽  
Ethics Committee ◽  
Partial Resection ◽  
Event Free Survival ◽  
Total Resection ◽  
Free Survival ◽  
Pediatric Hematology ◽  
National Medical ◽  
Choroid Plexus Carcinomas ◽  
Optimal Treatment Strategy

Choroid plexus carcinomas (CPCs) are rare pediatric tumors with a generally poor prognosis. Currently there is no definite optimal treatment strategy for this neoplasm. This study is supported by the Independent Ethics Committee and approved by the Academic Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology. This study included patients with a verified diagnosis of CPC at the age 0 to 18 years in the period from 01.01.2009 to 31.12.2019. A total of 32 patients were registered. The median age was 2.65 years; 93.5% of CPCs were localized in the lateral ventricles. Initial metastases were found in 21.8% of cases; 5-year event-free survival (EFS) in children with metastases was lower than in those who did not have metastases 29 ± 17% and 49 ± 12%. In our cohort, gross total resection (GTR) was performed in 65.6% of patients. The five-year EFS in patients with complete CPC removal was higher than in patients who underwent subtotal and partial resection (63 ± 13%, 12 ± 11%, and 0%, respectively). In addition, overall survival (OS) was slightly higher in those who underwent GTR compared with subtotal and partial resection (74 ± 12%, 67 ± 16%, and 0%, respectively). Of the 32 children with CPCs, 15 children received programm chemotherapy, 17 non-programm chemotherapy, 5-year EFS in patients who received programm and non-programm chemotherapy was 79 ± 11% and 0%, respectively (p = 0.0006), 5-year OS in patients who received programm and non-programm chemotherapy was 93 ± 7% and 36 ± 14% (p = 0.0054). 

scholarly journals Results of the use of cladribine in children with acute myeloid leukemia in the treatment according to the AML-MM-2006 protocol

2021 ◽  
Vol 20 (1) ◽  
pp. 40-45
Author(s):  
D. A. Venyov ◽  
I. I. Kalinina ◽  
T. Yu. Salimova ◽  
D. A. Evseev ◽  
V. E. Matveev ◽  
...  
Keyword(s):  
Myeloid Leukemia ◽  
Risk Group ◽  
Research Center ◽  
Control Group ◽  
Consolidation Therapy ◽  
Event Free Survival ◽  
Free Survival ◽  
Pediatric Hematology ◽  
National Medical ◽  
Significant Therapeutic Effect

The aim of this work was to evaluate the results of the use of cladribine in the treatment according to the AML-MM-2006 protocol as post-remission therapy in children. This study is supported by the Independent Ethics Committee and approved by the Academic Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology. The article presents the experience of treating children with AML at the Russian Children's Clinical Hospital, and later at the Dmitry Rogachev National Research Center within the framework of the AML-MM-2006 protocol. For the period from 2006 to 2018, 25 children were included in the study. As a comparison, to assess the effectiveness of therapy, the remaining cohort of patients from the intermediate risk group, which consisted of 83 children, was selected. Ultimately, the addition of cladribine in consolidation therapy did not show a significant therapeutic effect (event-free survival 0.47 ± 0.1 for the cladribine group, 0.52 ± 0.06 for the control group), including the development of relapse (56% patients in the cladribine group had a relapse, in the control group – in 34.5%). Thus, the study proved that further inclusion of cladribine in consolidation therapy for primary AML is inappropriate. 

scholarly journals Rhabdomyosarcoma in the first year of life. Experience of Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology

2021 ◽  
Vol 20 (1) ◽  
pp. 76-90
Author(s):  
T. V. Stradomskaya ◽  
D. Yu. Kachanov ◽  
N. S. Grachev ◽  
A. V. Akhaladze ◽  
M. V. Teleshova ◽  
...  
Keyword(s):  
High Risk ◽  
Confidence Interval ◽  
Local Control ◽  
Stage Iv ◽  
First Year ◽  
Event Free Survival ◽  
Free Survival ◽  
Pediatric Hematology ◽  
National Medical ◽  
First Year Of Life

Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in childhood, but is very rare in infants. RMS diagnosed during the first year of life is reported to have poor outcome. The aim of the study was to analyze the results of therapy of RMS in the first year of life treated in federal center in Russian Federation. This study is supported by the Independent Ethics Committee and approved by the Academic Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology. All prospectively registered patients with RMS in infants treated during the period 02.2012–05.2018 (75 month) were included. Diagnosis was confirmed by histology. All patients were examined and stratified according to the GPOH Cooperative Weichteilsarkom Study (CWS) Group guidance 2009. 13 prospectively registered patients with RMS in infants treated during the period were included. Median age at initial diagnosis was 6.7 (range 0.23–11.9) months. The tumor was detected prenatally at 32 and 33 weeks of gestation in 2 (15%) patients. The age of these patients was ≤ 1 month in 2 of 13 patients. Tumor size was ≤ 5 cm in 8 (61,5%) of 13 patients. Median volume tumor was 24 (range 0.001–150) ml. The primary site was head and neck (n = 4; 31.5%), pelvis (n = 3; 23%), extremities (n = 3; 23%), genitourinary system (n = 2; 15%), other (n = 1; 7.5%). IRS stage distribution was stage III in 12 (92.5%) patients, stage IV – 1 (7.5%). Regional nodal metastasis (N1) occurred in 1 (7.5%) patient with tumor of the head and neck nonparameningeal. Distant metastasis occurred in 1 (7.5%) patient and the site of spread was subcutaneous fat (blueberry muffin syndrome), pleura, roots of both lungs, pancreas, bone marrow. Histology of these infants was RMA (n = 6; 46%), RME (n = 6; 46%) and spindle-cell RMS (n = 1; 7.5%). The FOXO1-fusion positive status was found in 4/6 patients RMA by fluorescence in situ hybridization (FISH). 11/13 (85%) patients were categorized as high risk, 1/13 (7.5%) as very high risk, 1/13 (7.5%) – treatment for stage IV patients with metastatic disease. All patients (n = 13; 100%) received chemotherapy according to CWS guidance 2009 protocol. All patients were administered at a reduced dose according to body weight. The distribution of treatment regimens was as follows: 11/13 (85%) – IVA, 1/13 (7.5%) – IVADO, 1/13 (7.5%) – CEVAIE. 3/13 patients aged less than 1 month was administered chemotherapy according to VAC. Initial surgery included biopsy in 8/13, surgery – 5/13 (R2 – resection). Local control was provided in 11/13 (85%) patients: only surgery in 7/13 (54%) patients, only radiotherapy in 1/13 (7.5%), radiotherapy and surgery 1/13 (7.5%), only brachytherapy 1/13 (7.5%), brachytherapy and surgery in 1/13 (7.5%). 2/13 patients were not local control: one patient with favorable site (vagina) and one patient with stage IV that showed complete response after chemotherapy in both cases. Radiotherapy including brachytherapy was administered of the all patients at the over 1 year. Radiotherapy was used in 2 of 13 patients (dose range 50.4 Gy and 51.2 Gy), brachytherapy – 36 Gy. At the start of the radiotherapy the age was 21.1 and 13.37 months, brachytherapy – 12,4 and 14,5 months. Second-look surgery was performed in 9/13 (69%) patients: R0 (n = 7), R1 (n = 1), R2 (n = 1). 2/9 patients were performed mutilating surgery: orbital exenteration and cystectomy. Induction therapy was completed in 12 (92.5%) of 13 patients. Median follow-up time was 42.7 (range 3.7–90) months. 8 patients were alive, 5 died. 8/13 patients whom alive were observation: 6/8 patients no relapse/progressive, 2/8 patients – remission after local relapse and progression. 3-year overall survival was 68,4% (95% confidence interval 42,6–94,1. 3-year event-free survival was 46,2% (95% confidence interval 19,1–73,3). Patients aged less than 1 year are particularly problematic. The relatively low event-free survival rate in this age group is associated with the impossibility of carrying out the entire volume of multimodal therapy and required a tailored therapeutic approach. 

scholarly journals Clinical and molecular characteristics of WNT medulloblastomas

2019 ◽  
Vol 18 (4) ◽  
pp. 11-18
Author(s):  
L. I. Papusha ◽  
L. A. Yasko ◽  
A. P. Ektova ◽  
K. A. Voronin ◽  
A. A. Merishavyan ◽  
...  
Keyword(s):  
Gene Mutations ◽  
Favorable Outcome ◽  
Molecular Characteristics ◽  
Total Resection ◽  
Pediatric Hematology ◽  
National Medical ◽  
Molecular Group ◽  
International Data ◽  
Disease Free

WNTMBaccounts for about 10% of all medulloblastomas and have a favorable outcome in patients under 16 years according international data. We analyzed clinical and molecular characteristics of 20 patients with WNTMBpatients. The study was approved by the Independent Ethics Committee of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology. The molecular group verification was performed by Nanostring gene expression profiling. The detection of CTNNB1 and TP53 gene mutations was carried out with Sanger sequencing. WNT Medulloblastoma are mainly classic (18/20 patients), non-metastatic (19/20 patients). Gross total resection was performed in 18 patients. Somatic CTNNB1 mutations were found in 17 patients, somatic TP53 were identified in 2 patients. 19/20 assessable patients are disease-free with a median follow-up of 23 months. One patient died from the progression of the disease. WNT MB patients have an overall favorable outcome, even for metastatic and TP53 positive tumors. The reduction in the intensity of therapy is indicated forWNTMB in case of reliable identification ofWNTMB.

scholarly journals Anthracycline Type during Induction Associated with Outcome in Pediatric t(8;21) and Inv(16) AML

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 11-11 ◽  
Author(s):  
Henrik Hasle ◽  
Jonas Abrahamsson ◽  
Evelina S De Bont ◽  
Valerie de Haas ◽  
Barbara De Moerloose ◽  
...  
Keyword(s):  
Conflicts Of Interest ◽  
Secondary Malignancy ◽  
Event Free Survival ◽  
Core Binding Factor ◽  
Hematopoietic Stem ◽  
Liposomal Daunorubicin ◽  
Free Survival ◽  
Pediatric Hematology ◽  
Binding Factor ◽  
Salvage Rate

Abstract Background. The core-binding factor AMLs, t(8;21)(q22;q22), AML1-ETO or RUNX1-RUNX1T1 and inv(16)(p13q22), CBFB–MYH11, are considered as favorable risk factors in AML with superior event-free survival (EFS) and overall survival (OS). However, within the AML protocol from the Nordic society of pediatric hematology/oncology (NOPHO) from 2004 we observed a decrease in EFS for t(8;21) AML. Material. We analyzed all children with t(8;21) or inv(16) treated on NOPHO-AML 93 and NOPHO-AML 2004 from Denmark, Finland, Iceland, Norway, and Sweden from 1993 to 2013 and patients from countries later joining the NOPHO-AML 2004 protocol; Estonia (2004), Hong Kong (2007), Belgium and the Netherlands (2010). The choice of anthracycline during induction changed from two courses including doxorubicin (75 mg/m2 in each course), Dox-Dox, in the NOPHO-AML 93 to idarubicin (36 mg/m2) and mitoxantrone (30 mg/m2), Ida-Mitox, in NOPHO-AML 04. After the observation of a decrease in EFS in patients with t(8;21) in late 2010 it was recommended to give fludarabine, cytarabine, and DaunoXome (liposomal daunorubicin 180 mg/m2) as second induction to patients with t(8;21); Ida-DNX. Results. From 1993 to 2013 a total of 90 children, median age 9.1 years range 1-16, with t(8;21) AML were treated with Dox-Dox (n=25), Ida-Mitox (n=41), and Ida-DNX (n=24). Hematopoietic stem cell transplantation was given to 10 patients (11%), mostly with sibling donors in the NOPHO-AML 93 protocol (n=7). A reduction of the 3-year EFS was noted in patients treated with Ida-Mitox (41%) compared with Dox-Dox (64%). Those treated with Ida-DNX had significantly higher 3-year EFS of 87% (Figure). The events in t(8;21) AML on Ida-Mitox were relapses (n=20), death in CR1 (n=2), and secondary malignancy (n=2). The salvage rate for relapsed t(8;21) patients remained high and the overall favorable OS did not change significantly (Figure). From 1993 to 2013 a total of 58 children with inv(16) AML were treated with Dox-Dox (n=18) and Ida-Mitox (n=40). No significant changes in EFS (72% vs. 65%) or OS (89% vs. 79%) were observed for patients with inv(16). Conclusion. Despite the overall favorable survival in t(8;21) the present study suggests that the choice of anthracycline during induction may be of major importance for the EFS in t(8;21) AML. Idarubicin and mitoxantrone during induction was associated with an inferior EFS compared with doxorubicin in t(8;21) AML. DaunoXome combined with fludarabine and cytarabine seemed to be particular effective during induction therapy in t(8;21) AML. Figure 1 Figure 1. Figure 2 Figure 2. Disclosures No relevant conflicts of interest to declare.

scholarly journals Use of romiplostim for newly diagnosed immune thrombocytopenia in children

2020 ◽  
Vol 19 (1) ◽  
pp. 18-26
Author(s):  
E. V. Suntsova ◽  
I. I. Chikvina ◽  
M. N. Sadovskaya ◽  
N. N. Kotskaya ◽  
L. A. Hachatryan ◽  
...  
Keyword(s):  
Adverse Events ◽  
Immune Thrombocytopenia ◽  
Ethics Committee ◽  
Clinical Use ◽  
Newly Diagnosed ◽  
Pediatric Hematology ◽  
Number Of Children ◽  
National Medical ◽  
Clinical Onset ◽  
Life Threatening

Immune thrombocytopenia (ITP) is a disease with a heterogeneous clinical manifestation. In the majority of children newly diagnosed ITP is a self-limited benign disorder, while chronic ITP develops rarely. The clinical onset of ITP can occur in very different ways: from nearly invisible skin hemorrhage to severe life-threatening bleeding. Conventional treatments promote a response in most patients, but in a small number of children thrombocytopenia is unresponsive. In this article, we describe our experience of the clinical use of romiplostim in children with severe unresponsive newly diagnosed ITP. The study was approved by the Independent Ethics Committee and the Scientific Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology. The severity of bleeding decreased significantly after the start of romiplostim therapy in all cases. Durable complete (platelets > 100 × 109 /l) response was achieved in five out of six patients 4 to 8 weeks after starting therapy. Three children have remained in lasting remission for 1 to 3 years after the discontinuation of romiplostim. There were no adverse events associated with romiplostim.

Factors affecting event free survival (EFS) after high dose chemotherapy with stem cell rescue (HDC/SCR) in children with brain tumors

2006 ◽  
Vol 24 (18_suppl) ◽  
pp. 9057-9057
Author(s):  
J. Aguayo ◽  
A. Teplick ◽  
A. Butturini ◽  
A. Erdreich-Epstein ◽  
R. Jubran ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Stem Cell ◽  
Brain Tumors ◽  
Conditioning Regimen ◽  
Gross Total Resection ◽  
High Dose ◽  
Event Free Survival ◽  
Total Resection ◽  
Free Survival ◽  
Localized Disease

9057 Background: HDC/SCR is a novel approach for treatment of children with brain tumors. Despite several studies suggesting results are better in children with primitive neural ectodermal tumors (PNET) or medulloblastoma (MB), it is controversial whether other factors can predict outcome. Methods: We retrospectively analyzed data from 53 patients who underwent HDC/SCR for brain tumors at Children Hospital Los Angeles between June 1992 and June 2005. Patients were aged 4 months to 15.8 years at diagnosis and 9 months to 18.1 years at transplant. In all cases the conditioning regimen included thiotepa and/or etoposide and/or carboplatin. The variables considered were age at diagnosis, histology, extent of disease, conditioning regimen, radiation therapy, stem cell type and timing of transplant. Results: Diagnoses included 33 PNET/MB, 8 high-grade gliomas, 5 ependymomas, 3 germinomas, 2 choroid plexus carcinomas and 2 rhabdoid tumors. Overall event free survival at 36 months was 53±9.1% in PNET/MB and 40±15.5% in tumors with other histology. Variables associated with better EFS in PNET/MB were no prior progression (p=0.00094), no spread into bone marrow (p=1.2 E-10) and age less than 3 years at diagnosis (p=0.046). There was a trend toward improved outcome if there was gross total resection of the tumor (p=0.11). Patients with PNET transplanted before progression had better EFS if they had localized disease at diagnosis (p=0.00024), and if gross total surgical resection was accomplished (p=0.11). Patients with PNET/MB transplanted after progression had better EFS if there was no spread of disease into blood or bone marrow (p=0.00023), if they were male (p=0.053), had localized disease at relapse (p=0.12) and if radiotherapy was given after transplant (p=0.053). In patients with tumors of other histology, the only variable associated with prolonged EFS was gross total resection (p=0.026). Conclusions: Children most likely to benefit from HDC/SCR for brain tumors are those less than 3 years old who are newly diagnosed with PNET that is locally confined, and those with a non-PNET diagnosis who have gross total resection of tumor. No significant financial relationships to disclose.

scholarly journals Surgical management of complex spinal cord lipomas: how, why, and when to operate. A review

2019 ◽  
Vol 23 (5) ◽  
pp. 537-556 ◽  
Author(s):  
Dachling Pang
Keyword(s):  
Spinal Cord ◽  
Survival Data ◽  
Progression Free Survival ◽  
Partial Resection ◽  
Total Resection ◽  
Multiple Series ◽  
Free Survival ◽  
Anatomical Relationship ◽  
Aggressive Surgical Approach

This review summarizes the classification, anatomy, and embryogenesis of complex spinal cord lipomas, and it describes in some detail the new technique of total lipoma resection and radical reconstruction of the affected neural placode. Its specific mission is to tackle two main issues surrounding the management of complex dysraphic lipomas: whether total resection confers better long-term benefits than partial resection and whether total resection fares better than conservative treatment—i.e., no surgery—for asymptomatic lipomas. Accordingly, the 24-year progression-free survival data of the author and colleagues’ series of over 300 cases of total resection are compared with historical data from multiple series (including the author and colleagues’ own) of partial resection, and total resection data specifically for asymptomatic lesions are compared with the two known series of nonsurgical treatment of equivalent numbers of patients. These comparisons amply support the author’s recommendation of total resection for most complex lipomas, with or without symptoms. The notable exception is the asymptomatic chaotic lipoma, whose peculiar anatomical relationship with the neural tissue defies even this aggressive surgical approach and consequently projects worse results (admittedly of a small number of cases) than for the other two lipoma subtypes of dorsal and transitional lesions. Prophylactic resection of asymptomatic chaotic lipomas is therefore not currently endorsed.

scholarly journals Terapi Leukemia Mieloblastik Akut Anak: ProtokolAra-C, Doxorubycinedan Etoposide $'(YV0RGLÀNDVLNordic Society of Pediatric Hematology and Oncology(m-NOPHO)

Sari Pediatri ◽  
2016 ◽  
Vol 14 (6) ◽  
pp. 345
Author(s):  
Eddy Supriyadi ◽  
Ignatius Purwanto ◽  
Pudjo Hagung Widjajanto
Keyword(s):  
Event Free Survival ◽  
Free Survival ◽  
Pediatric Hematology

Latar belakang. Pengobatan leukemia mieloblastik akut (LMA) telah banyak mengalami kemajuan. Di negara maju, keberhasilan pengobatan sudah mencapai 65%, sementara di Indonesia diperkirakan masih di bawah 10%.Tujuan.Mengetahui profil LMA mengevaluasi keberhasilan pengobatan. Metode.Dilakukan analisis terhadap catatan medis pasien LMA yang dirawat di bangsal anak Rumah Sakit Sardjito Jogjakarta pada tahun 1999-2011. Diagnosis ditetapkan berdasarkan analisis morfologi dan sitokimia terhadap apus sumsum tulang dan atau darah tepi. Analisis survivaldilakukan terhadap pasien yang diberi pengobatan dengan protokol ADE atau m-NOPHO. Hasil.didapatkan 210 pasien berumur 0–17 tahun yang didiagnosis LMA. Enampuluh delapan mendapat kemoterapi, 46 (32%) dengan protokol ADE, 91 (64%) dengan protokol m-NOPHO, dan 5 (4%) pasien diobati dengan protokol lain. Event Free Survival (EFS)setelah lima tahun pada pasien yang mendapat kemoterapi adalah 2,4%, pasien sesudah 4 tahun dengan protokol m-NOPHO (3,7% ± 2,3%), sedangkan semua pasien yang menggunakan protokol ADE meninggal sebelum 1,5 tahun pengamatan (p=0,005).Kesimpulan.Tingkat keberhasilan pengobatan LMA di RS Dr. Sardjito masih sangat rendah. Pengobatan dengan protokol m-NOPHO mempunyai tingkat keberhasilan yang lebih baik dibandingkan protokol ADE.

scholarly journals Platelet phenotype in children with ANKRD26-related thrombocytopenia

2021 ◽  
Vol 20 (2) ◽  
pp. 65-73
Author(s):  
D. M. Polokhov ◽  
D. V. Fedorova ◽  
A. V. Pshonkin ◽  
A. A. Ignatova ◽  
E. A. Ponomarenko ◽  
...  
Keyword(s):  
Flow Cytometry ◽  
Ethics Committee ◽  
Control Group ◽  
Healthy Children ◽  
Functional Disorders ◽  
Pediatric Hematology ◽  
National Medical ◽  
Platelet Size ◽  
Shape Changes ◽  
Apparently Healthy

The mechanisms of hemorrhagic manifestations in patients with ANKRD26associated thrombocytopenia (ANKRD26-AT) are poorly understood. The aim of this work is to detect possible morpho-functional disorders of platelets in patients with mutations in the ANKRD26gene by flow cytometry with activation. The study was approved by the Independent Ethics Committee of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology. 8 children aged from 1.5 to 15 years were examined. The platelet count ranged from 29 to 172 thousand/μl, with a median of 60 thousand/μl. The severity of hemorrhagic manifestations was assessed on a standardized scale (Pediatric Bleeding Questionnaire, PBQ) and it ranged from 0 to 5 points, with a median of 3.5 points. Platelet activation was performed with a CRP + TRAP mixture. Comparison was carried out with the results of examination of 26 apparently healthy children (control group, CG) aged 2 to 15 years. When compared with CG, patients showed an increase in platelet size (FSC; p= 0.018) and granularity (SSC; p< 0.001) after activation. In contrast to the CG, the correlation between FSC and SSC of platelets in patients was not significant (cor. = 0.55; p= 0.15). Patients showed a high, significant relationship between the number and FSC of platelets (cor. = –0.93; p< 0.001), as well as an increased density of CD42b (p < 0.001) and a decrease in the proportion of procoagulant platelets (p= 0.01) after activation. The revealed changes indicate violations of the mechanisms of activation and shape changes of platelets in patients with ANKRD26-AT.

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