Molecular Identification of Extrapulmonary Vaccine Adverse Events after BCG in Paraffin-Embedded Specimens.

According to the World Health Organization (WHO), around 1 million children worldwide are diagnosed with tuberculosis each year. The Bacillus Calmette-Guérin (BCG) vaccine has been used around the world for over 100 years. The complications of the BCG vaccination can occur in about 0,06% of children and include local or systemic adverse reactions. Due to the close analogy between the vaccine strain and other species of the Mycobacterium tuberculosis complex (MTBC), molecular methods are recommended for differential diagnosis of Vaccine adverse events (VAE) after BCG. The ability to quickly and specifically identify BCG is important in view of different treatment regimens. The aim of the study was to assess the usefulness of genetic testing for Mycobacterium bovis BCG in the paraffin-embedded specimens' methods. We describe two cases of VAE in immune-compromised children presenting with osteoarticular changes that had been clinically suspected of tuberculosis and led to molecular identification through GeneXpert, GenoType MTBC, and Spoligotyping. Results: Mycobacterium bovis BCG was detected in osteoarticular changes embedded in paraffin block of two patients. Conclusion: Genetic tests using paraffin-embedded materials allow for quick identification and differential diagnosis of patients with Tuberculosis and VAE after BCG. This is an important issue, especially in cases where the tissue has only been submitted for histopathological examination without microbiological diagnostics for tuberculosis.

Exam stress and the metacognitive strategies of reading in students with dyslexia: The role of motivational mechanisms and educational support.

Despite numerous studies on dyslexia, there is still a lack of empirical data on the factors determining the functioning of students with dyslexia in the context of written exams. Therefore, the aim of the study is to identify the relationship between sources of stress in an exam situation and the reported use of reading strategies by dyslexic students in terms of motivation and therapeutic interventions in the educational environment. This descriptive-analytical study used data from a larger project focused on children with and without dyslexia. The research sample (n = 640) included girls (n = 280) and boys (n = 360) aged between 14 and 15 years (M = 14.40, SD = 0.55), attending the 7th or 8th grade in Polish mainstream primary schools. Random and intentional sampling was used. All students completed four questionnaires. The results were analyzed using regression analysis in Model templates for PROCESS v4 for SPSS by Hayes. The study showed significant weak and moderate positive correlations between the sources of exam stress and the reading strategies reported by students, ranging from 0.186 to 0.570, as well as significant moderate and strong correlations between reading strategies and experienced educational support, ranging from 0.229 to 0.505, and between reading strategies and motivation to read, ranging from 0.582 to 0.701. The type of stress source significantly influenced the selection of specific reading strategies. Motivation acted as a mediator, while educational support was a moderator in the relationship between exam stress and the reported use of reading strategies. Based on our results, the source of stress may be perceived as factors activating metacognitive mechanisms aimed at selecting appropriate strategies for working with texts. Researchers and teachers should be aware of the need to undertake activities to support students with dyslexia focused on developing the ability to recognize the sources of exam stress and select effective coping strategies.

Factors Modifying Outcome After MIBG Therapy in Children With Neuroblastoma-A National Retrospective Study.

BACKGROUND: Neuroblastoma is the most common pediatric extracranial tumor with varied prognoses, but the survival of treated refractory or relapsing patients remains poor. OBJECTIVE: This analysis presents the outcomes of children with neuroblastoma undergoing MIBG therapy in Poland in 2006-2019. STUDY DESIGN: A retrospective cohort of 55 patients with refractory or relapsed neuroblastoma treated with I-131 MIBG in Poland in 2006-2019 was analyzed. The endpoints were overall survival (OS), event-free survival (EFS), cumulative incidence (CI) of second cancers and CI of hypothyroidism. Survival curves were estimated using the Kaplan-Meier method and compared between the cohorts by the log-rank test. Cox modeling was adopted to estimate hazard ratios for OS and EFS, considering factors with P < 0.2. RESULTS: Fifty-five patients with a median age of 78.4 months (range 18-193) with neuroblastoma underwent one or more (4 patients) courses of MIBG I-131 therapy. Fifteen patients were not administered chemotherapy, 3 children received standard-dose chemotherapy, and 37 patients were administered high-dose chemotherapy (HDCT) (busulfan-melphalan in 24 and treosulfan-based in 12 patients). Forty-six patients underwent stem cell transplantation, with autologous (35 patients), haploidentical (6), allogeneic (4), and syngeneic grafts (1). The median time from first MIBG therapy to SCT was 22 days. Children with relapsing tumors had inferior OS compared to those with primary resistant disease (21.2% vs 58.7%, p=0.0045). Survival was better in patients without MYCN gene amplification. MIBG therapy was never curative, except in patients further treated with HDCT with stem cell rescue irrespective of the donor type. 31 patients were referred for immune therapy after MIBG therapy, and the 5-year OS in this group was superior to the untreated children (55.2% vs 32.7%, p=0.003), but the difference in the 5-year EFS was not significant (25.6% vs 32.9%, p=ns). In 3 patients, a second malignancy was diagnosed. In 19.6% of treated children, hypothyroidism was diagnosed within 5 years after MIBG therapy. CONCLUSION: MIBG therapy can be incorporated into the therapeutic strategy of relapsed or resistant neuroblastoma patients as preconditioning with HDCT rather than stand-alone therapy. Follow-up is required due to the incidence of thyroid failure and risk of second cancers.

Disseminated Juvenile Xanthogranuloma and Hemophagocytic Lymphohistiocytosis Developed During Treatment of Acute Lymphoblastic Leukemia: Case Report.

The association between acute lymphoblastic leukemia (ALL), non-Langerhans cell histiocytosis (non-LCH), and hemophagocytic lymphohistiocytosis (HLH), to the best of our knowledge, has not been published to date. Juvenile xanthogranuloma (JXG), as a type of non-LCH, is usually a benign disease limited to the skin. Systemic involvement is rarely reported. The present case report describes a 15-year-old boy diagnosed with disseminated JXG involving skin and bone marrow concurrent with severe symptoms of HLH during ALL therapy. Examination of immunoglobulin heavy chain genes in B-cell precursor leukemic blasts and histiocytes in the skin and bone marrow revealed identical rearrangements, confirming clonal relationship between both diseases. Implementation of corticosteroids, vinblastine, etoposide, cyclosporine, and tocilizumab resulted in partial skin lesion resolution with no improvement of bone marrow function; therefore, hematopoietic stem cell transplantation (HSCT) was eventually performed. The patient's hematological and general status has improved gradually; however, remarkable recovery of skin lesions was observed after empirical antitubercular therapy. Mycobacterium spp. infection should be considered as a possible secondary HLH trigger. Triple association of ALL, non-LCH, and HLH highlights heterogeneity of histiocytic disorders and possible common origin of dendritic and lymphoid cells.

Gastrointestinal peptides in children before and after hematopoietic stem cell transplantation.

BACKGROUND: Gastrointestinal tract function and it's integrity are controlled by a number of peptides whose secretion is influenced by severe inflammation. In stomach the main regulatory peptide is ghrelin. For upper small intestine cholecystokinin and lower small intestine glucagon-like peptide- 1 are secreted, while fibroblast growth factor-21 is secreted by several organs, including the liver, pancreas, and adipose tissue [12]. Hematopoietic stem cell transplantation causes serious mucosal damage, which can reflect on this peptides. METHODS: The aim of the study was to determine fasting plasma concentrations of ghrelin, cholecystokinin, glucagon- like peptide-1, and fibroblast growth factor-21, and their gene expressions, before and 6 months after hematopoietic stem cell transplantation.27 children were studied, control group included 26 healthy children. RESULTS: Acute graft versus host disease was diagnosed in 11 patients (41%, n = 27). Median pre-transplantation concentrations of gastrointestinal peptides, as well as their gene expressions, were significantly lower in studied group compared with the control group. Only median of fibroblast growth factor-21 concentration was near-significantly higher before stem cell transplantation than in the control group. The post-hematopoietic transplant results revealed significantly higher concentrations of the studied peptides (except fibroblast growth factor-21) and respective gene expressions as compare to pre transplant results. Median glucagone like peptide-1 concentrations were significantly decreased in patients with features of acute graft versus host disease. Moreover, negative correlation between glucagone like peptide-1 concentrations and acute graft versus host disease severity was found. CONCLUSIONS: Increased concentrations and gene expressions of gastrointestinal tract regulation peptides can be caused by stimulation of regeneration in the severe injured organ. Measurement of these parameters may be a useful method of assessment of severity of gastrointestinal tract complications of hematopoietic stem cell transplantation.

Visfatin concentrations in children with leukemia before and after stem cell transplantation.

Visfatin (VF) is an adipocytokine that performs many functions, including enhancing cell proliferation and biosynthesis of nicotinamide mononucleotides and dinucleotides. It also seems to be involved in the development of glucose metabolism disturbances. The goal of the study was the determination of VF concentrations in children with leukemia who are treated with stem cell transplantation. VF concentrations were measured in plasma before and after oral glucose tolerance test (OGTT; 60 and 120 minutes) in 22 children with leukemia treated with hematopoietic stem cell transplantation (HSCT) and healthy control subjects (n = 24). The HSCT group was studied twice: before HSCT (22 children) and approximately 6 months after HSCT (12 of 22 children). After fasting, concentrations of glucose, insulin, triglycerides, total cholesterol, high-density lipoprotein, and high-sensitivity C-reactive protein (hsCRP) were determined. Significantly lower (p < 0.05) median values of VF concentrations at all time points in the OGTT were found in pre- HSCT children compared with control subjects. The median VF concentration was significantly higher after HSCT compared with before HSCT. The decrease in VF in leukemic children in complete remission may be caused by myelosuppression and immunosuppression after prolong chemotherapy and is beneficial because of the decrease in its antiapoptotic activity. VF can serve as an additional biochemical marker for remission in patients with leukemia. Normalization of plasma VF concentration after HSCT might be caused by a process of immune reconstitution and prolonged inflammation (e.g., infections, graft-versus-host disease), injury to organs (e.g., lungs, gut, liver), and endocrinology deficiencies.

Matched sibling versus matched unrelated allogeneic hematopoietic stem cell transplantation in children with severe acquired aplastic anemia: experience of the polish pediatric group for hematopoietic stem cell transplantation.

In the study, 48 children with severe acquired aplastic anemia (SAA) transplanted from matched sibling donor (MSD) between 1991 and 2009, and 38 children with SAA transplanted from matched unrelated donor (MUD) between 2000 and 2009 were evaluated. Engraftment was achieved in 45 (93.75 %) patients after MSD-hematopoietic stem cell transplantation (HSCT) and in 33 (86.8 %) after MUD-HSCT. Transplant-related mortality rate after MSD-HSCT was 8 %, while 37 % after MUD-HSCT. After MSD-HSCT 44 (91.7 %) patients are alive for 1-216 months (median: 85 months), while after MUD-HSCT 24 (63.2 %) patients for 1-84 months (median: 16 months). The 5-year probability of event-free survival after MSD-HSCT and MUD-HSCT was 87 and 53 %, respectively, while 5 years of overall survival was 91 and 64 %, respectively. It was concluded that MSD-HSCT as the first line treatment for children with SAA is a safe therapeutic approach with a low rate of treatment failures and excellent outcome. Results of MUD-HSCT in pediatric patients with SAA who failed to respond to immunosuppressive therapy are still inferior than those of MSD-HSCT. Treatment failures of MUD-HSCT are mainly related to infectious complications and graft failure. It seems, however, that HLA-matching of unrelated donors at allelic level along with early MUD-HSCT after FCA (FLUDA, low-dose cyclophosphamide, and anti-thymocyte globulin) conditioning, perhaps using lower Thymoglobulin dose could enable further improvement of long-term results in children with SAA who lack MSD.

[Genotyping and minimal residual disease study in children with acute myeloid leukemia: preliminary results]. / Genotypowanie i oznaczania choroby resztkowej u dzieci z ostra bialaczka mieloblastycznq: wstepne wyniki.

The aim of the paper is to present the initial results of molecular examination which was started in 2006 for children with acute myeloid leukemia. Better knowledge of biology of this disease, can result in establishing of new risk factors what allows more precise patient stratification to different therapeutic groups. Study was obtained patients until to 18 years of age treated according to AML-BFM 2004 INTERIM protocol in 14 centers of the Polish Pediatric Leukemia/Lymphoma Study Group. Mononuclear cells were collected from bone marrow on time points established according to the AML-BFM 2004 INTERIM protocol. Collected cells were isolated on Ficoll gradient, and RNA and DNA were isolated using TRIZOL reagent. To synthesize cDNA an amount of 1 mg of total RNA was used. To perform quantitative RT-PCR and RQ-PCR reactions 4 fusion gene transcripts (AML1-ETO, CBFb-MYH11, PML-RARA /subtype bcrl and bcr3/) were used according to the protocol established by Europe Against Cancer Program. An expression of WT1 gene was tested additionally. An analysis of ABL control gene was used to normalize of achieved results. Determination of duplication of FLT3 gene in DNA sample was performed with starters complementary to JM region. Genotyping was performed in 75 patients with acute myeloid leukemia so far. AML1-ETO fusion gene transcript was found in 14 patients (19%). PML-RARA (subtype bcr3) and CBFB-MYH11 gene transcripts were detected in 3 (4%) and 3 (4%) patients, respectively. Duplication of FLT3 gene was found in 4 (5.3%) cases. Between 67 tested children over expression of WT1 was present in 51 patients (76%). Analysis of MRD level in subsequent time points showed systematic decrease of number of fusion gene transcript copies and gene WT1 expression. To establish the rate of molecular marker presence in AML in children and the influence of the presence of MRD on the treatment results as well, the study has to be conducted on a larger group of patients with longer follow-up.

[Treatment failure in children with acute myelocytic leukemia: over 25-year experience of Polish Pediatric Leukemia/Lymphoma Study Group with four consecutive unified treatment protocols for childhood acute myelocytic leukemia]. / Niepowodzenia leczenia w ostrej bialaczce szpikowej u dzieci: ponad 25-letnie doswiadczenia Polskiej Pediatrycznej Grupy ds. Leczenia Bialaczek i Chloniaków.

Four consecutive intensive unified regimens (BFM-AML-83, PGP-AML 94, PGP-AML 98 AML-BFM 2004 Interim) for acute myelocytic leukemia (AML) have been conducted by the Polish Pediatric Leukemia/Lymphoma Study Group (PPLLSG) since 1983. The last one, introduced four years ago is still active, and only preliminary result may be presented. There were 726 children with AML diagnosed (226, 102, 247 and 151 in the I, II , III and IV periods, respectively), and 603 of them were eligible for evaluation (208, 83, 195 and 117, respectively). Complete remission rates were: 71.4%, 67.5%, 81.4% and 87% in consecutive periods, respectively. Five-year overall survival (OS) and event-free survival (EFS) rates were: 33% and 32% for PGP-AML 83 regimen, 38% and 36% for PGP-AML 94 regimen, and 53% and 46% for PGP-AML 98 regimen, respectively. For AML-BFM Interim 2004 the 3-year OS and EFS were 57% and 57%, respectively. Despite continuous improvement of the treatment results, the number of failures have remained too high, but the pattern have changed in the following way: Early deaths (from diagnosis to 15 day of treatment) decreased only in the fourth period to 3%. "Aplasia deaths" (between day 15 and 42) decreased gradually from 16% in the first period to 1.5% and 2.2% in the third and in the fourth period, respectively. Deaths in remission decreased from 10% in first and second period to 3.5% at present. Number of non responders increased between first and second period from 6% to 18%, later decreased to 8.2% at present. These trends e.g. decrease of early death and treatment related mortality reflect both the better efficacy of antileukemic treatment and the improvement of supportive care.

Prognostic impact of combined fludarabine, treosulfan and mitoxantrone resistance profile in childhood acute myeloid leukemia.

BACKGROUND: The role of cellular drug resistance in childhood acute myeloid leukemia (AML) has not yet been established. The aim of the study was the analysis of the clinical value of ex vivo drug resistance in pediatric AML. PATIENTS AND METHODS: A cohort of 90 children with de novo AML were assayed for drug resistance profile by the 3-4,5-dimethylthiazol-2-yl-2,5-difenyl tetrazolium bromide (MTT) assay and prognostic model of in vitro drug sensitivity was analyzed. RESULTS: Children who relapsed during follow-up showed higher in vitro resistance of leukemic blasts to most of the drugs tested, except for cytarabine, cladribine, vincristine, mercaptopurine and thioguanine. A combined in vitro drug resistance profile to fludarabine, treosulfan and mitoxantrone (FTM score) was defined and it had an independent prognostic significance for disease free survival in pediatric AML. CONCLUSION: The combined fludarabine, treosulfan and mitoxantrone resistance profile to possibly may be used for better stratification of children with AML or indicate the necessity for additional therapy.

[Results of combined chemoreduction and local treatment for intraocular retinoblastoma]. / Wyniki leczenia siatkówczaka--chemioterapia skojarzona z leczeniem miejscowym.

PURPOSE: Of this study was to evaluate the results of combined treatment for intraocular retinoblastoma. MATERIALS AND METHODS: We examined 23 children (32 eyes), 14 with unilateral and 9 with bilateral disease. 53% of eyes had the stage V of the disease, according to Reese-Ellsworth. RESULTS: In I-III group according to R-E, it was possible to save more than 90% of eyes. In group IV-V we saved 20% of eyes. CONCLUSIONS: Chemoreduction combined with local treatment is an optimal method of treatment for retinoblastoma, especially in I-III group.

Skin involvement in highly malignant non-Hodgkin lymphomas of childhood and adolescence.

OBJECTIVE: The aim of this study was to demonstrate the clinical spectrum of highly malignant cutaneous non-Hodgkin lymphoma (NHL) in children and to define the outcome among these patients. MATERIAL AND METHODS: A retrospective analysis of children with NHL treated at Polish oncology centers was carried out in order to determine patients with skin involvement. Thirteen subjects with primary and 4 with secondary cutaneous NHL were studied. The diagnosis of NHL was based on histological and immunohistochemical examination of skin biopsy. RESULTS: Nine of 13 cases of primary cutaneous NHL presented as a tumors. Other manifestations were as follows: hard infiltration, edema of subcutaneous tissue, maculopapular lesions, and generalized erythroderma. The mean time between the first cutaneous symptoms and diagnosis of NHL was 5.6 +/- 3.3 months. Secondary cutaneous lesions during the course of NHL were described as maculopapular or nodular eruption. In addition, 2 subjects had generalized ichthyosis. Among patients with primary cutaneous NHL, 11 (84.6%) subjects are still alive without any signs of the disease. Two children (15.4%) died. In patients with secondary skin involvement during the course of NHL only one child is still alive with a residual tumor mass in the mediastinum. The estimated 5-year overall survival for primary cutaneous NHL was significantly better than for individuals with secondary cutaneous NHL (p = 0.02). CONCLUSIONS: Primary cutaneous NHL has a relatively favorable prognosis. On the other hand, cutaneous metastases of extracutaneous NHL seem to be a poor prognostic factor.

[Soft tissue sarcomas of bladder and prostate in children treated according to the CWS-96 protocol--report of the Polish Paediatric Solid Tumours Study Group]. / Przebieg i powiklania terapii u dzieci z miesakami pecherza moczowego i prostaty leczonych wedlug Schematu CWS-96.

AIM: analysis of the course, outcome and therapy complications in bladder/prostate soft tissue sarcomas (STS) in children treated from I'1997 to II'2003 according to CWS-96 protocol in Poland. MATERIAL AND METHODS: 22 children (M/F: 17/5, age: 8 months - 17 years 2 months; median 5,3y). Histopathology: RMS-20 patients (RME-14), non-RMS-2 patients. RESULTS: 96% presented with advanced neoplasm (III - 14, IV - 7). In 18 patients (82%) primary tumour exceeded 5cm; in 17 (77%) extended beyond site of origin. Lymph nodes metastases were stated in six (27%), distant metastases in seven patients (32%). No patient underwent primary complete tumour excision. All received chemotherapy for high risk group. Response to chemotherapy after three cycles was favourable in eight and unfavourable in eight children. Delayed resections were performed in 11 patients (mutilating - 7) proving complete in only five. Radiotherapy (32-50,4Gy) was given to 16 patients, mainly after delayed incomplete surgery. Five patients developed local relapse, four--continual disease progression. None of these nine patients had ever complete tumour resection. Nine patients died: seven due to neoplasm progression, two of therapy complications (septic shock, ARDS). Thirteen patients are alive (59%) with mean follow-up of 42 months. Eight children had bladder removed with continent ileal pouch formed in four. CONCLUSIONS: 1) Advanced stages of bladder/prostate sarcomas in children suggest the necessity of earlier diagnosis. 2) Due to adjuvant chemo- and radiotherapy approximately 60% of patients with this diagnosis may be nowadays cured of bladder/prostate sarcomas without mutilating surgical procedures.

[Genitourinary soft tissue sarcomas located outside bladder and prostate in children treated according to the CWS-96 protocol--report from the Polish Paediatric Solid Tumours Study Group]. / Przebieg i powiklania terapii u dzieci z miesakami ukladu moczowo-plciowego z wylaczeniem pekcherza moczowego i prostaty leczonych wedlug schematu CWS-96.

AIM: Analysis of therapy efficacy in non-bladder/prostate genitourinary sarcomas in children treated from I'1997 to VI'2003 with CWS-96 protocol in Poland. MATERIAL AND METHODS: 19 children (M/F: 15/4, age: 3m-17,5y; median 7,2y). Histopathology: RMS - 15pts (RME-13), non-RMS- 4. RESULTS: Primary site: testes - 9 patients, paratesticular region - 6, uterus - 2, vagina and ovary-1 of each. 63% presented with low stage neoplasm (I - 7, II - 5). Primary tumour exceeded 5cm and/or invaded surrounding tissues in 7 patients (37%). 3 patients had regional, 2 patients--distant lymph nodes metastases. Primary excision: complete in 7 patients, incomplete - 12 (microscopically - 5, macroscopically - 7). Six of 7 patients with macroscopic tumour residues responded to chemotherapy (CR-4, GR-2). One patient (stage III triton tumour of uterus) did not, respond but obtained complete remission after mutilating delayed surgery. No other patient required delayed tumour resection. Radiotherapy (23,5-54 Gy) was given to 8 patients. 3 children developed local relapse, 3 patients died (16%): 2 due to neoplasm progression, 1 of neutropenia-related sepsis. 16 patients are alive (84%) with mean follow-up 48 months. The only permanent complications result from mutilating surgery. CONCLUSIONS: 1) prognosis in children with non-bladder/prostate genitourinary sarcomas is favourable despite incomplete primary excision of the neoplasm. 2) chemotherapy and radiotherapy were accompanied by severe but transient myelosupression in the HR group.

[Soft tissue sarcoma of the bladder and prostate. A report of the Polish Paediatric Solid Tumour Group (PPSTG)]. / Nowotwory tkanek miekkich w pecherzu moczowym i gruczole krokowym w materiale polskiej pediatrycznej grupy guzów litych (PPGGL).

Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children younger then 15 years of age. The treatment of RMS localized in bladder or prostate still remains controversial. The aim of this study was analysis of treatment results in children with soft tissue sarcoma of bladder and prostate. From 1993 to 2001 the PPSTG has used three protocols to treat soft tissue sarcomas in children. The CWS-91 regimen has been served to treat stage I-III and SIOP-IV Intergroup study in stage IV. Since 1996 the CWS-96 protocol for all patients in stage I-IV has been used. After biopsy confirmation of the diagnosis patients were treated with chemotherapy and subsequent surgery. Radiotherapy was administrated with total dose 32Gy v 44.5Gy v 48Gy. The group of 19 patients aged from 3 months to 18 years with median age 6 y 4 m were evaluated. The median follow-up time was 42 months. RMS-embryonal was diagnosed in 11 patients, RMS-alveolare in 4 and others types in 4. Sixteen patients presented clinical stage III and 3 stage IV The tumours were primarily localised in bladder in 16 patients and prostate in 3. Eight were tumours of volume bigger than 10 cm. After induction chemotherapy two patients received partial cystectomy, 5 complete cystectomy and 3 complete cystectomy with genitourinary reconstructive. Eight patients did not receive surgery. The most common treatment failure was isolated, local relapse in 8 children particularly in patients with any second surgery. The EFS for all patients was 52% and for patients with localised RMS tumours 65%. Significant prognostic factors are the initial tumours volume, the lymph nodes infiltration and the response to the first chemotherapy cycle. Surgery is the most important procedure in local control of soft tissue sarcoma. Reconstructive options in poor and non responder patients can improve the EFS and life quality of those patients.

[Efficacy of idarubicin in the treatment of childhood acute non-lymphoblastic leukemia: report of Polish Pediatric Leukemia/Lymphoma Study Group]. / Zastosowanie idarubicyny w leczeniu ostrej bialaczki nielimfoblastycznej u dzieci--Raport Polskiej Grupy Pediatrycznej ds Bialaczek i Chloniaków.

UNLABELLED: Results of treatment of childhood ANLL remained unsatisfactory for a long time, and introduction of a new drug seemed justified as the EFS achieved in this disease between 1993-97 was 42%. In 1998 a new protocol containing idarubicine in dose 12 mg/m2 (each dose regardless of treatment phase) was introduced. Between 1998 and 2001, 137 children with ANLL were referred to nine participating centers of PPLLSG. Thirty nine were uneligible, so 98 were evaluated. Among them 56 were qualified to standard risk group (SRG) and 35 to high risk group (HRG). In 7 children the risk group was not established due to early death. Remission rate was 79% in the whole group and 98% and 63% in SRG and HRG, respectively. The actuarial 3-year overall survival (OS), 3-year event-free survival (EFS) and 3-year event-free interval (EFI) are respectively: In the whole group 61%, 54% and 67%; in SRG 81%, 70% and 71%; in HRG 40%, 40%, and 53%. CONCLUSIONS: Due to introduction if idarubicine significant improvement of the results was achieved in SRG (better OS, EFS and EFI). Better new treatment methods are required in HRG.