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1.
Pediatr Transplant ; 28(3): e14758, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38659218

RESUMO

BACKGROUND: Data on the risk factors and outcomes for pediatric patients with SARS-CoV-2 infection (COVID-19) following hematopoietic stem cell transplantation (HSCT) are limited. OBJECTIVES: The study aimed to analyze the clinical signs, risk factors, and outcomes for ICU admission and mortality in a large pediatric cohort who underwent allogeneic HSCT prior to COVID-19 infection. METHOD: In this nationwide study, we retrospectively reviewed the data of 184 pediatric HSCT recipients who had COVID-19 between March 2020 and August 2022. RESULTS: The median time from HSCT to COVID-19 infection was 209.0 days (IQR, 111.7-340.8; range, 0-3845 days). The most common clinical manifestation was fever (58.7%). While most patients (78.8%) had asymptomatic/mild disease, the disease severity was moderate in 9.2% and severe and critical in 4.4% and 7.6%, respectively. The overall mortality was 10.9% (n: 20). Deaths were attributable to COVID-19 in nine (4.9%) patients. Multivariate analysis revealed that lower respiratory tract disease (LRTD) (OR, 23.20, p: .001) and lymphopenia at diagnosis (OR, 5.21, p: .006) were risk factors for ICU admission and that HSCT from a mismatched donor (OR, 54.04, p: .028), multisystem inflammatory syndrome in children (MIS-C) (OR, 31.07, p: .003), and LRTD (OR, 10.11, p: .035) were associated with a higher risk for COVID-19-related mortality. CONCLUSION: While COVID-19 is mostly asymptomatic or mild in pediatric transplant recipients, it can cause ICU admission in those with LRTD or lymphopenia at diagnosis and may be more fatal in those who are transplanted from a mismatched donor and those who develop MIS-C or LRTD.


Assuntos
COVID-19 , Transplante de Células-Tronco Hematopoéticas , Humanos , COVID-19/epidemiologia , COVID-19/terapia , COVID-19/mortalidade , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Criança , Masculino , Feminino , Estudos Retrospectivos , Adolescente , Turquia/epidemiologia , Pré-Escolar , Fatores de Risco , SARS-CoV-2 , Lactente , Transplante Homólogo , Índice de Gravidade de Doença
2.
Am J Infect Control ; 52(1): 81-86, 2024 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-37591312

RESUMO

BACKGROUND: Children who underwent hematopoietic stem cell transplant (HSCT) transplants are at high risk of developing central-line-associated bloodstream infections (CLABSIs). The present study aimed to identify possible risk factors for mortality by analyzing the clinical and laboratory characteristics of patients diagnosed with CLABSI in our pediatric hematopoietic stem cell transplant unit. METHODS: The initial CLABSI episodes of 102 children were analyzed. Medical records of the patients were evaluated by preformed standardized surveys. Univariate analysis and multivariate logistic regression analysis were performed to identify risk factors for mortality. RESULTS: Thirty-five patients (34.3%) were female. The median age was 48 months (3-204). The median time to onset of CLABSI was 19 days (4-150). The gram-negative and gram-positive bacteria ratio among the causative agents was 57.8% to 34.3%. The mortality rate was 12.6%. The presence of severe neutropenia, initiation of inappropriate empirical antibiotic therapy, the presence of hypotension, persistent bacteremia, pediatric intensive care unit admission, growth of carbapenemase-positive gram-negative microorganism and multidrug-resistant bacteria were significantly high in the mortality group when compared to survivors. The presence of hypotension, inappropriate empirical antibiotic therapy, and persistent bacteremia were found to be independent risk factors for mortality. CONCLUSIONS: Rational use of antibiotics, active surveillance and screening of patients together with improved infection control practices may reduce the incidence and the consequences of CLABSIs.


Assuntos
Bacteriemia , Infecções Relacionadas a Cateter , Cateterismo Venoso Central , Transplante de Células-Tronco Hematopoéticas , Hipotensão , Sepse , Criança , Humanos , Feminino , Pré-Escolar , Masculino , Infecções Relacionadas a Cateter/epidemiologia , Infecções Relacionadas a Cateter/etiologia , Estudos Retrospectivos , Sepse/etiologia , Bacteriemia/microbiologia , Cateteres , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Antibacterianos/uso terapêutico , Fatores de Risco , Hipotensão/complicações , Cateterismo Venoso Central/efeitos adversos
3.
J Clin Immunol ; 43(8): 2062-2075, 2023 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-37726596

RESUMO

BACKGROUND: Purine nucleoside phosphorylase (PNP) deficiency is a rare autosomal recessive combined immunodeficiency. The phenotype is profound T cell deficiency with variable B and NK cell functions and results in recurrent and persistent infections that typically begin in the first year of life. Neurologic findings occur in approximately two-thirds of patients. The mechanism of neurologic abnormalities is unclear. Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for PNP deficiency. METHODS: We report here six patients from five unrelated families with PNP deficiency treated in two centers in Turkey. We evaluated the neurological status of patients and compared to post-transplantation period if available. Then, we performed PubMed, Google Scholar, and Researchgate searches using the terms "PNP" and "hematopoietic stem cell transplantation" to find all reported cases of PNP transplantation and compared to our cohort. RESULTS: Six patients were treated in two centers in Turkey. One patient died from post-transplant complications. The other four patients underwent successful HSCT with good immune reconstitution after transplantation (follow-up 21-48 months) and good neurological outcomes. The other patient with a new mutation is still waiting for a matching HLA donor. DISCUSSION: In PNP deficiency, clinical manifestations are variable, and this disease should be considered in the presence of many different clinical findings. Despite the comorbidities that occurred before transplantation, HSCT currently appears to be the only treatment option for this disease. HSCT not only cures immunologic disorders, but probably also improves or at least stabilizes the neurologic status of patients.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Doenças da Imunodeficiência Primária , Erros Inatos do Metabolismo da Purina-Pirimidina , Humanos , Purina-Núcleosídeo Fosforilase/genética , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/métodos , Doenças da Imunodeficiência Primária/diagnóstico , Doenças da Imunodeficiência Primária/terapia , Doenças da Imunodeficiência Primária/etiologia , Erros Inatos do Metabolismo da Purina-Pirimidina/terapia
4.
Clin Transplant ; 37(9): e15083, 2023 09.
Artigo em Inglês | MEDLINE | ID: mdl-37534623

RESUMO

INTRODUCTION: In children with inborn errors of immunity (IEI) who will receive a hematopoietic stem cell transplant (HSCT) treosulfan-based conditioning is currently preferred. The aim of this study was to investigate early and late outcomes in pediatric IEI patients receiving pre-HSCT treosulfan and to examine the effect of treosulfan dose monitoring on outcomes. METHODS: Seventy-three pediatric patients receiving this management between 2015 and 2022 were included. RESULTS: Overall survival rate was 80%, and event-free survival was 67.8%. A larger treosulfan dose AUC after first application increased the rate of early toxicity (p = .034) and slowed lymphocyte engraftment (r = .290; p = .030). Underlying disease, treosulfan AUC, donor type, stem cell type, number of immunosuppressive agents, the dose of anti-thymocyte globulin, and post-transplantation cyclophosphamide did not to increase risk of acute graft-versus-host disease. The risk of mixed chimerism (MC) in patients with autoimmune lymphoproliferative syndrome and leukocyte adhesion deficiency were higher than those with severe combined immunodeficiency (p = .021 and p = .014, respectively). The risk of MC was lower in those receiving peripheral blood stem cells (SC) compared with bone marrow derived SC (OR = .204, p = .022). CONCLUSION: The AUC of the treosulfan dose was not associated with poorer late outcomes. Treosulfan is an agent that can be used safely in the IEI patient group,  level measurement appears essential to identify early toxicities. Prospective studies with more extended follow-up periods are needed.


Assuntos
Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Humanos , Criança , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Estudos Prospectivos , Intervalo Livre de Doença , Bussulfano/uso terapêutico , Doença Enxerto-Hospedeiro/etiologia , Condicionamento Pré-Transplante/efeitos adversos
5.
Medeni Med J ; 38(2): 102-110, 2023 Jun 20.
Artigo em Inglês | MEDLINE | ID: mdl-37338860

RESUMO

Objective: Neuroblastoma is one of the common tumors of childhood. The demonstration of new factors such as isocitrate dehydrogenase 1 (IDH1) and isocitrate dehydrogenase 2 (IDH2) mutations will be important in the diagnosis and treatment. IDH1 and IDH2 mutations have been found in many types of cancer, such as malignant gliomas, acute myeloid leukemias, chondrosarcoma, and thyroid carcinoma. This study aimed to investigate the presence of IDH1 or IDH2 mutations in patients with neuroblastoma and to determine whether these mutations were different in terms of age, clinical findings, and response to treatment. Methods: Biopsy specimens of 25 patients with pediatric neuroblastoma patients were evaluated for IDH mutations. The clinical and laboratory features of the patients with/without mutation were retrospectively analyzed from a hospital database. Results: A total of 25 patients for whom genetic analysis could be performed were included in the study (60% male, n=15). The mean age was 32.2±25.9 months (3 days-96 months). IDH1 mutation was detected in 8 (32%) and IDH2 mutations in 5 (20%) patients. These mutations showed no statistically significant relationship with age, tumor localization, laboratory results, stage, and prognosis. However, in the case of IDH mutation, patients were diagnosed at the advanced stage. Conclusions: This study demonstrated the relationship between neuroblastoma and IDH mutation for the first time. Because to the fact that the mutation is very heterogeneous, it would be appropriate to conduct a larger series of patients in terms of the impact of the clinical significance of each mutation on the diagnosis and prognosis.

6.
Pediatr Transplant ; 27(4): e14504, 2023 06.
Artigo em Inglês | MEDLINE | ID: mdl-36919672

RESUMO

BACKGROUND: Retinoblastoma (RB) is the most common intraocular malignancy in childhood. Advanced RB, associated with exceedingly poor prognosis, requires more intensive multiagent chemotherapy than conventional regimens. Rescue of the bone marrow after intensive chemotherapy is achieved with stem cell transplantation. The sequential courses (tandem transplantation) of high-dose chemotherapy followed by autologous stem cell transplantation allow for even greater dose intensity in consolidation with the potential to use different active chemotherapeutics at each transplant and have proven feasible and successful in treating children with recurrent/refractory solid tumors. CASE DESCRIPTION: We report an infant with trilateral high-risk RB who received tandem high-dose chemotherapy (HDC) followed by autologous stem cell transplantation after the conventional chemotherapy. A 5-month-old female patient presented with strabismus, and the ophthalmoscopic examination showed intraocular tumoral lesions in both eyes. Magnetic resonance imaging (MRI) concluded the trilateral retinoblastoma diagnosis due to a tumoral mass in the optic chiasm. The follow-up ophthalmologic examinations and the MRI detected stable disease after six cycles of multiagent chemotherapy. CONCLUSIONS: Rescue with autologous stem cell transplantation after HDC allows for an increase in chemotherapy intensity. Tandem transplantation provides the chance to perform different chemotherapeutics at each transplant and enables an increase in the chemotherapy intensity, thus providing a positive effect on disease-free survival.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Neoplasias da Retina , Retinoblastoma , Criança , Lactente , Humanos , Feminino , Retinoblastoma/diagnóstico , Retinoblastoma/tratamento farmacológico , Transplante Autólogo , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Recidiva Local de Neoplasia , Transplante de Células-Tronco , Neoplasias da Retina/diagnóstico , Neoplasias da Retina/tratamento farmacológico , Terapia Combinada
7.
Pediatr Transplant ; 27(1): e14364, 2023 02.
Artigo em Inglês | MEDLINE | ID: mdl-35851981

RESUMO

BACKGROUND: BKV-HC is one of the most significant complications of HSCT. This retrospective study aimed to determine the frequency of BKV-HC in pediatric patients undergoing HSCT, detect the associated risk factors for the development of BKV-HC, and explore the effects of post-transplantation Cy use. METHODS: Three hundred twenty-seven patients (girls: 121, boys: 206) were analyzed according to sex, conditioning regimen, transplantation type, donor relatedness, stem cell source, the presence and grade of aGVHD, CMV co-existence, and Cy use. RESULTS: Multivariate analysis confirmed the prognostic importance of age (OR: 4.865), TBI use, the presence of aGVHD (OR: 2.794), CMV coinfection (OR: 2.261), and Cy use (OR: 27.353). A statistically significant difference was found between the mean BKV-HC follow-up times compared with post-transplantation Cy intake (p < .001). The BKV-HC rate increased as the number of risk factors of the patient increased. CONCLUSION: BKV-HC is an essential complication of HSCT primarily associated with Cy use, the presence of aGVHD, and donor relatedness. The present study shows that the use of Cy in the post-transplantation period further increases BKV-HC risk in pediatric patients, regardless of dose.


Assuntos
Vírus BK , Cistite , Infecções por Citomegalovirus , Transplante de Células-Tronco Hematopoéticas , Infecções por Polyomavirus , Infecções Tumorais por Vírus , Masculino , Feminino , Humanos , Criança , Transplante de Medula Óssea/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Estudos Retrospectivos , Infecções Tumorais por Vírus/complicações , Infecções Tumorais por Vírus/epidemiologia , Cistite/epidemiologia , Cistite/etiologia , Hemorragia/etiologia , Fatores de Risco , Ciclofosfamida , Infecções por Citomegalovirus/etiologia , Infecções por Polyomavirus/complicações , Infecções por Polyomavirus/epidemiologia
8.
Bone Marrow Transplant ; 57(5): 760-767, 2022 05.
Artigo em Inglês | MEDLINE | ID: mdl-35210564

RESUMO

We report the national data on the outcomes of hematopoietic stem cell transplantation (HSCT) for thalassemia major (TM) patients in Turkey on behalf of the Turkish Pediatric Stem Cell Transplantation Group. We retrospectively enrolled 1469 patients with TM who underwent their first HSCT between 1988 and 2020 in 25 pediatric centers in Turkey. The median follow-up duration and transplant ages were 62 months and 7 years, respectively; 113 patients had chronic graft versus host disease (cGVHD) and the cGVHD rate was 8.3% in surviving patients. Upon the last visit, 30 patients still had cGvHD (2.2%). The 5-year overall survival (OS), thalassemia-free survival (TFS) and thalassemia-GVHD-free survival (TGFS) rates were 92.3%, 82.1%, and 80.8%, respectively. cGVHD incidence was significantly lower in the mixed chimerism (MC) group compared to the complete chimerism (CC) group (p < 0.001). In survival analysis, OS, TFS, and TGFS rates were significantly higher for transplants after 2010. TFS and TGFS rates were better for patients under 7 years and at centers that had performed over 100 thalassemia transplants. Transplants from matched unrelated donors had significantly higher TFS rates. We recommend HSCT before 7 years old in thalassemia patients who have a matched donor for improved outcomes.


Assuntos
Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Talassemia , Talassemia beta , Criança , Doença Enxerto-Hospedeiro/etiologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Estudos Retrospectivos , Talassemia/complicações , Talassemia/terapia , Condicionamento Pré-Transplante/efeitos adversos , Turquia/epidemiologia , Talassemia beta/complicações , Talassemia beta/terapia
9.
Transplant Cell Ther ; 27(10): 859.e1-859.e10, 2021 10.
Artigo em Inglês | MEDLINE | ID: mdl-34216791

RESUMO

Relapse after allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the most frequent cause of post-transplantation mortality. Isolated extramedullary (EM) relapse (iEMR) after HSCT is relatively rare and not well characterized, particularly in pediatric patients. We retrospectively analyzed 1527 consecutive pediatric patients with acute leukemia after allo-HSCT to study the incidence, risk factors, and outcome of iEMR compared with systemic relapse. The 5-year cumulative incidence of systemic relapse (either bone marrow [BM] only or BM combined with EMR) was 24.8%, and that of iEMR was 5.5%. The onset of relapse after allo-HSCT was significantly longer in EM sites than in BM sites (7.19 and 5.58 months, respectively; P = .013). Complete response (CR) 2+/active disease at transplantation (hazard ratio [HR], 3.1; P < .001) and prior EM disease (HR, 2.3; P = .007) were independent risk factors for iEMR. Chronic graft-versus-host disease reduced the risk of systemic relapse (HR, 0.5; P = .043) but did not protect against iEMR. The prognosis of patients who developed iEMR remained poor but was slightly better than that of patients who developed systemic relapse (3-year overall survival, 16.5% versus 15.3%; P = .089). Patients experiencing their first systemic relapse continued to have further systemic relapse, but only a minority progressed to iEMR, whereas those experiencing their iEMR at first relapse developed further systemic relapse and iEMR at approximately similar frequencies. A second iEMR was more common after a first iEMR than after a first systemic relapse (58.8% versus 13.0%; P = .001) and was associated with poor outcome. iEMR has a poor prognosis, particularly after a second relapse, and effective strategies are needed to improve outcomes.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Leucemia Mieloide Aguda , Criança , Humanos , Cinética , Leucemia Mieloide Aguda/terapia , Recidiva , Estudos Retrospectivos , Fatores de Risco
11.
Pediatr Transplant ; 25(7): e14063, 2021 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-34092004

RESUMO

BACKGROUND: Primary immunodeficiency diseases (PID) are characterized by the occurrence of frequent infections and are caused by many genetic defects. Hematopoietic stem cell transplantation (HSCT) is the only curative treatment option for the majority of PID. As a Pediatric Hematology-Oncology-Immunology Transplantation Unit, we wanted to present our HSCT experience regarding treatment of primary immunodeficiency diseases. METHODS: 58 patients were included in the study between January 2014 and June 2019. We searched 9/10 or 10/10 matched-related donor (MRD) firstly, in the absence of fully matched-related donor. We screened matched unrelated donor (MUD) from donor banks. MRD was used in 24 (41.3%) patients, MUD in 20 (34.4%) patients, and haploidentical donors in 14 (24.1%) patients. Demographic data, HSCT characteristics, and outcome were evaluated. While 16 patients had severe combined immunodeficiency (SCID), the remaining was non-SCID. RESULTS: Of the 58 patients, 38 were male and 20 were female. Median age at transplantation was 12 months (range: 2.5-172 months). Combined immunodeficiencies consisted 67.2% of patients. Mean follow-up time was 27 months (6 months-5 years). Median neutrophil, lymphocyte, and thrombocyte engraftment days were similar in comparison of both donor type and stem cell source. The most common complication was acute GvHD in 15 (25.8%) patients. In total, five patients (31%) belonging to the SCID group and 10 patients (23.8%) belonging to the non-SCID group died. Our total mortality rate was 15 (25.8%) in all patients. CONCLUSIONS: We would like to present our HSCT experiences as a pediatric immunology transplantation center. Existing severe infections before transplantation period, BCGitis, and CMV are important issues of transplantation in Turkey. However, the follow-up time is shorter than some studies, our results regarding complications and survival are similar to previous reports.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Doenças da Imunodeficiência Primária/terapia , Adolescente , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Masculino , Turquia
13.
Hemoglobin ; 45(1): 25-29, 2021 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-33478286

RESUMO

Allogeneic hematopoietic stem cell transplantation (HSCT) is the curative therapy for ß-thalassemias that induces severe life-threatening complications. The human leukocyte antigen (HLA) registries and umbilical cord blood banks have carried out diligent searches to find matched unrelated donors (MUDs) for about 70.0% of patients from 2000 onwards. The chance of finding a non-sibling fully matched family donors is higher in some ethnic groups in which consanguineous marriages are common. We have studied and compared transplant complications and outcomes in different graft types (sibling, non-sibling family and unrelated). The non-sibling matched family donor (MFD) group consisted of four mothers, three fathers, five cousins, one paternal uncle and one paternal aunt. There was no significant difference in the mean transfused CD34+ cells, engraftment, median days of neutrophil and platelet recovery were achieved (p > 0.05). The distribution of postttransplant complication did not show any significant difference between groups (p > 0.05). In univariate analysis and multivarite analyses, age, gender, Pesaro risk group (I-II vs. III) and ABO incompatibilty demonstrated a significant difference in disease free survival (p < 0.05). Furthermore, in the second step of investigating overall survival (OS), age, gender and Pesaro risk group (I-II vs. III) showed a significant difference (p < 0.05). There was no significant difference in transplant-related mortality (TRM) between groups. Non-sibling related donor transplants are important for populations where consanguineous marriages are common. Transplant groups according to graft type had similar thalassemia-free survival (TFS) and OS when using a treosulfan-based regimen in our study.


Assuntos
Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Talassemia beta , Família , Sangue Fetal , Humanos , Talassemia beta/terapia
14.
J Pediatr Hematol Oncol ; 43(5): e608-e612, 2021 07 01.
Artigo em Inglês | MEDLINE | ID: mdl-32852396

RESUMO

The COVID-19 outbreak has caused anxiety among children with hematology-oncology disease and their families, as it has in every segment of society. In this study, we aimed to detect the anxiety levels of children with hematologic or oncologic disease and of their parents after the COVID-19 outbreak. The sample consisted of 15 patients 12 to 18 years of age receiving treatment in the Pediatric Hematology and Oncology Unit in Altinbas University Medical Faculty Bahçelievler Medikalpark Hospital and 33 parents of the same unit patients between 6 and 18 years of age, and their 35 healthy peers and their parents. The State-Trait Anxiety Inventory was applied to participant children and their parents to evaluate their general anxiety and pandemic-related anxiety levels. Children with a hematology-oncology disease and their families were compared with healthy peers and their families. No significant difference was observed for pandemic-related anxiety levels (P>0.05). Both parent groups exhibited higher anxiety levels with regard to the pandemic than did their children (P<0.05). Children with hematology-oncology disease reported significantly higher trait anxiety levels when compared with healthy peers (P=0.01). The families of children who had not received stem cell transplantation had higher state and trait anxiety scores than the families of children who had received the transplantation (P<0.05). Even though they were in the high-risk group, children with a hematology-oncology disease and their families had pandemic-related anxiety levels comparable with those of healthy peers and their families.


Assuntos
Ansiedade/patologia , COVID-19/complicações , Neoplasias Hematológicas/epidemiologia , SARS-CoV-2/isolamento & purificação , Adolescente , Ansiedade/etiologia , Ansiedade/psicologia , COVID-19/transmissão , COVID-19/virologia , Estudos de Casos e Controles , Criança , Feminino , Nível de Saúde , Neoplasias Hematológicas/virologia , Humanos , Masculino , Inquéritos e Questionários , Turquia/epidemiologia
15.
Pediatr Transplant ; 25(5): e13942, 2021 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-33320995

RESUMO

BACKGROUND: Post-transplant relapse has a dismal prognosis in children with acute leukemia undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Data on risk factors, treatment options, and outcomes are limited. PROCEDURE: In this retrospective multicenter study in which a questionnaire was sent to all pediatric transplant centers reporting relapse after allo-HSCT for a cohort of 938 children with acute leukemia, we analyzed 255 children with relapse of acute leukemia after their first allo-HSCT. RESULTS: The median interval from transplantation to relapse was 180 days, and the median follow-up from relapse to the last follow-up was 1844 days. The 3-year overall survival (OS) rate was 12.0%. The main cause of death was disease progression or subsequent relapse (82.6%). The majority of children received salvage treatment with curative intent without a second HSCT (67.8%), 22.0% of children underwent a second allo-HSCT, and 10.2% received palliative therapy. Isolated extramedullary relapse (hazard ratio (HR): 0.607, P = .011) and relapse earlier than 365 days post-transplantation (HR: 2.101, P < .001 for 0-180 days; HR: 1.522, P = .041 for 181-365 days) were found in multivariate analysis to be significant prognostic factors for outcome. The type of salvage therapy in chemosensitive relapse was identified as a significant prognostic factor for OS. CONCLUSION: A salvage approach with curative intent may be considered for patients with post-transplant relapse, even if they relapse in the first year post-transplantation. For sustainable remission, a second allo-HSCT may be recommended for patients who achieve complete remission after reinduction treatment.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Leucemia/mortalidade , Leucemia/terapia , Doença Aguda , Adolescente , Criança , Pré-Escolar , Terapia Combinada , Feminino , Seguimentos , Transplante de Células-Tronco Hematopoéticas/métodos , Humanos , Lactente , Recém-Nascido , Leucemia/diagnóstico , Masculino , Prognóstico , Recidiva , Estudos Retrospectivos , Terapia de Salvação , Análise de Sobrevida , Transplante Homólogo , Turquia/epidemiologia , Adulto Jovem
16.
Turk J Med Sci ; 50(8): 1916-1921, 2020 12 17.
Artigo em Inglês | MEDLINE | ID: mdl-32628434

RESUMO

Aim: The main purpose of this study is to determine the current status of long-term follow-up (LTFU) for childhood cancer survivors and the challenges of LTFU for pediatric cancer survivors at pediatric oncology institutions in Turkey. Material and methods: A questionnaire was e-mailed to the directors of 33 pediatric oncology centers (POCs) registered in the Turkish Pediatric Oncology Group (TPOG). Of these 33 active TPOG institutions, 21 participated in the study and returned their completed questionnaires. Results: Only 1 of the 21 participating centers had a separate LTFU clinic. The remaining centers provided LTFU care for childhood cancer survivors at the pediatric oncology outpatient clinic. Of these centers, 17 (80.9%) reported difficulty in transition from the pediatric clinic to the adult clinic, 14 (66.6%) reported insufficient care providers, and 12 (57.1%) reported insufficient time and transportation problems. As neglected late effects, 16 (76.1%) centers reported psychosocial and getty job problems and 11 (52.3%) reported sexual and cognitive problems. None of the centers had their own LTFU guidelines for their daily LTFU practice Conclusion: This study was the first to gain an overview of the needs of POCs and the gaps in survivorship services in Turkey. The results from this study will help to develop a national health care system and national guidelines for pediatric cancer survivors.


Assuntos
Assistência ao Convalescente/métodos , Sobreviventes de Câncer/estatística & dados numéricos , Países em Desenvolvimento , Pediatria/métodos , Inquéritos e Questionários/estatística & dados numéricos , Criança , Estudos Transversais , Humanos , Transição para Assistência do Adulto , Turquia
17.
J Clin Immunol ; 40(3): 466-474, 2020 04.
Artigo em Inglês | MEDLINE | ID: mdl-32020378

RESUMO

PURPOSE: Autosomal recessive (AR) CARD9 deficiency is an inherited immune disorder which results in impaired innate immunity against various fungi. Superficial and invasive fungal infections, mainly caused by Candida or Trichophyton species, are the hallmark of CARD9 deficiency. Together with the increasing number of CARD9-deficient patients reported, different pathogenic fungal species have been described such as Phialophora, Exophiala, Corynespora, Aureobasidium, and Ochroconis. Saprochaete capitata is an opportunistic infectious agent in immunocompromised patients and is a common cause of invasive fungal disease in patients with hematological malignancies. In this study, we investigated the causative genetic defect in a patient with S. capitata fungal infection which disseminated to lymph nodes and common bile duct. METHODS: The identification of the isolated yeast strain was made by direct microscopic examination and confirmed by internal transcribed spacer (ITS) sequencing. We applied whole exome sequencing to search for the disease-causing mutation. Sanger sequencing was used to validate the mutation in the patient and his parents. RESULTS: S. capitata was isolated from the biopsy specimen as the causative microorganism responsible for the invasive fungal disease in the patient. Whole exome sequencing revealed a homozygous c.883C > T, (p.Q295*) mutation in CARD9, confirmed by Sanger sequencing. CONCLUSIONS: This is the first report of invasive Saprochaete infection associated with autosomal recessive (AR) CARD9 deficiency in the literature and thereby further extends the spectrum of fungal diseases seen in these patients.


Assuntos
Proteínas Adaptadoras de Sinalização CARD/genética , Candidíase Mucocutânea Crônica/diagnóstico , Colestase/diagnóstico , Infecções Fúngicas Invasivas/diagnóstico , Saccharomycetales/fisiologia , Deleção de Sequência/genética , Adolescente , Candidíase Mucocutânea Crônica/genética , Colestase/genética , Transtornos Cromossômicos , Genes Recessivos , Humanos , Hospedeiro Imunocomprometido , Infecções Fúngicas Invasivas/genética , Iraque , Masculino , Sequenciamento do Exoma
20.
Turk Pediatri Ars ; 54(1): 35-39, 2019.
Artigo em Inglês | MEDLINE | ID: mdl-31217707

RESUMO

AIM: To identify the psychosocial and economic problems of the pediatric patients with cancer who were treated at the Dr. Sami Ulus Obstetrics and Gynecology and Child Health and Diseases Training and Research Hospital's Pediatric Oncology Department and their relatives during this process. MATERIAL AND METHODS: We interviewed a total of 100 patients who were treated at Dr. Sami Ulus Obstetrics and Gynecology and Child Health and Diseases Hospital's Pediatric Oncology Department between 1996 and 2015, and were now followed up without treatment and their relatives using survey questions on the psychosocial and economic problems they experienced. RESULTS: Most of patients were from provinces outside Ankara. The average monthly income was below the level that would meet the family needs in 80% of the families and only 16% had extra income. Additional economic support had been received by 93% of the families in the patient group. Twenty-five families (25%) had been forced to sell property during the treatment. Forty-nine (49%) families had borrowed money from acquaintances and relatives or had taken out a bank loan. Serious psychological problems were experienced during and after the treatment by 46% of the mothers; 41% of the families had used religious procedures more commonly during the treatment period to cope with the psychological problems. Education was subject to a 1-2 year pause in 83% of the patients. CONCLUSION: Childhood-age patients with cancer and their families experience significant psychosocial and economic problems during and after the treatment process. Providing medical treatment and psychosocial support in harmony is an important factor that increases the success of cancer treatment. The patient and the family will require psychosocial support mechanisms throughout life, starting from the moment they face the disease. Developing national social support programs and legal regulations to form a basis for such programs are required in our country.


AMAÇ: Dr. Sami Ulus Kadin Dogum ve Çocuk Sagligi ve Hastaliklari Egitim ve Arastirma Hastanesi Çocuk Onkoloji Klinigi'nde tedavi edilmis kanserli çocuk hastalarin ve yakinlarinin bu süreçte karsilastiklari psikososyal ve ekonomik sorunlari belirlemek. GEREÇ VE YÖNTEMLER: Dr. Sami Ulus Çocuk Sagligi ve Hastaliklari Hastanesi Çocuk Onkoloji Klinigi'nde 1996­2015 yillari arasinda tedavi görmüs ve su anda hastaliksiz olarak izlemde olan toplam 100 hasta ve yakini ile görüsülerek yasadiklari psikososyal ve ekonomik sorunlara iliskin anket sorulari yönlendirildi. BULGULAR: Hastalarimizin çogu Ankara disi illerden gelmekte idi. Hasta ailelerinin %80'inin ortalama aylik gelirinin ailenin gereksinimlerini karsilama düzeyinin altinda oldugu ve sadece %16'sinin ek geliri oldugu saptandi. Hasta grubunda ailelerimizin %93'ü ek ekonomik destek almislardi. Yirmi bes aile (%25) tedavi süresince sahip olduklari bazi mal varliklarini satmak zorunda kalmisti. Kirk dokuz (%49) aile tanidik ve akrabalarindan borç almisti ya da bankadan kredi çekmisti. Annelerin %43'ünün tedavi sirasinda ve sonrasi ciddi psikolojik sorunlar yasadigi, psikolojik sorunlarla basa çikabilmek için ailelerin %40'inda tedavi döneminde dini ibadetlere egilimde artis oldugu gözlendi. Hastalarimizin %83'ünün egitimlerinde 1­2 yil gibi bir kayip yasandigi görüldü. ÇIKARIMLAR: Çocukluk çagi kanser hastalari ve aileleri tedavi sürecinde ve sonrasinda önemli oranda psikosoyal ve ekonomik sorunlar yasamaktadir. Kanserde, tibbi tedavi ile psikososyal destegin bir uyum içerisinde yürütülmesi tedavi basarisini artiran önemli bir etmendir. Hastanin ve ailesinin hastalikla tanistigi ilk andan baslayarak tüm hayat boyunca psikososyal destek mekanizmalarina gereksinim vardir. Ülkemizde ulusal olarak bu yönde gelistirilecek sosyal destek programlari ve bu programin yürütülecegi zemini olusturacak yasal düzenlemelere gereksinim vardir.

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