RESUMO
Thromboembolic events (TE) in childhood are relatively rare but, serious complications of acute leukemia. The aim was to define the incidence and risk factors of thrombosis in children with leukemias. The electronic files of pediatric denovo/relapsed acute leukemia patients aged below 18 years, treated between 2011 and 2021 were retrospectively evaluated for thrombotic attacks. Thirty out of 469 patients developed 35 thrombotic events. The median age at the time of the TE was 11.8 (2-17.6) years, and the median time from diagnosis to TE was 9 (0-58) months. The frequency of TE was found at 7.4% (n = 35/469). When catheter related (n = 13) events, superficial venous events (n = 10), and arterial central nervous system thrombosis (n = 1) were excluded, the frequency of TE was decreased to 2.3% (n = 11/469). Children older than 10 years old (13.8%; n = 21/152) had significantly higher thromboembolic events than the others (4.4%; n = 14/317) (p = 0.03). The majority of attacks were symptomatic 66% (n = 23/35). The most common complaints were local pain, swelling, and redness 52% (n = 12/23). The majority of attacks in patients with relapsed (75%; 6/8) and newly diagnosed acute lymphoblastic leukemia (40%; 10/25%) developed during the induction phase. Thrombosis recurred in 13.3% (n = 4/30) of cases more than once. Thrombotic attacks were successfully treated with low molecular weight heparin 60% (n = 21/35), and recombinant tissue plasminogen activator 17% (n = 6/35). None of the children were lost due to thrombosis. Thrombosis is an important complication during acute leukemia treatment. Successful results are obtained with early diagnosis and treatment attempts by creating awareness.
RESUMO
The posttransplant lymphoproliferative disease is a severe cause of morbidity and mortality following allogeneic hematopoietic stem cell transplantation. Central Nervous System involvement in EBV-related PTLD is rare, and there is no standard treatment recommendation. We present our patient and discuss other previously reported cases of EBV-associated PTLD with CNS involvement.
Assuntos
Infecções por Vírus Epstein-Barr , Transplante de Células-Tronco Hematopoéticas , Transtornos Linfoproliferativos , Humanos , Rituximab/uso terapêutico , Herpesvirus Humano 4 , Infecções por Vírus Epstein-Barr/complicações , Infecções por Vírus Epstein-Barr/tratamento farmacológico , Transtornos Linfoproliferativos/etiologia , Transtornos Linfoproliferativos/complicações , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Sistema Nervoso CentralRESUMO
BACKGROUND: Refugee or asylum seekers (RAS) children are at increased risk of physical, developmental, and behavioral health issues. The aim of this study was to evaluate clinical and psychosocial outcomes of hematopoietic stem cell transplantation (HSCT) in RAS children and compare health-related quality of life (HRQOL) to those of Turkish peers. METHODS: This retrospective study included patients who underwent HSCT aged 0-18 years and completed 100-day post-transplant. The PedsQL 4.0 Generic Core Scale was used in children over 5 years old to compare HRQOL. RESULTS: A total of 166 RAS patients (M/F: 106 /60) underwent 174 HSCTs (six patients had two, and one had three HSCT) compared to 66 Turkish patients. The mean age of the patients in the RAS group was 7.8 ± 4.9 years and similar to controls. A total of 124 patients (75%) were from Syria, and 49 (25%) were from other countries in the Middle East and Africa. The cause of migration was war in 121 (74%) RAS patients. Complications of HSCT were no different between the groups. However, the rate of neutropenic sepsis was significantly higher in the RAS group (p = 0.004). The total scores of HRQOL were not different between RAS and controls. In the RAS group, ratings of social functioning were lower in patients with consanguinity or non-malignant disease or who had match-related donors. DISCUSSION: Identifying areas of difficulty in subscales of HRQOL may help physicians to classify patients who need additional supportive care. Regular monitoring and supporting physical needs may result in better functional outcomes after HSCT.
Assuntos
Transplante de Células-Tronco Hematopoéticas , Refugiados , Humanos , Criança , Pré-Escolar , Qualidade de Vida/psicologia , Turquia , Estudos Retrospectivos , Transplante de Células-Tronco Hematopoéticas/psicologiaRESUMO
INTRODUCTION: Chronic myeloid leukemia (CML) is rare in the first two decades of life comprising only 3% of newly diagnosed pediatric and adolescent leukemias. We studied the epidemiologic and clinical features of patients with CML diagnosed at younger than 3 years of age and evaluated treatment and long-term outcome. METHOD: Data from the International Pediatric I-BFM/CML Registry were retrospectively analyzed using the European LeukemiaNet criteria of the year 2006. Characteristics and treatment outcome of patients <3 years old at diagnosis were evaluated from standardized forms. RESULTS: Twenty-two patients (n = 22/479; 4.6%, male/female:14/8) were enrolled with a median age of 22 months (range, 10-34 m). Major symptoms comprised asthenia (30%), fever (30%), abdominal pain (20%), extramedullary signs (14%), hemorrhage (5%), and weight loss (5%). The extramedullary signs were specified in eight children: blueberry muffin (n = 1), sudden swollen abdomen (n = 1), sustained vomiting (n = 1), and cervical and inguinal lymph nodes (n = 5). Two of five children with cervical and inguinal lymph nodes were categorized as accelerated phase. Overall, 19 of 22 (86%) children were diagnosed in chronic phase, while the remaining three patients were in advanced phase. Median follow-up was 78 months (range, 7-196 m). Twenty-one out of 22 patients initially received imatinib, while one child received IFN + ARA-C. Imatinib was changed to second-line tyrosine kinase inhibitors (TKIs) in 29% of cases. During follow-up, 41% patients underwent stem cell transplantation (SCT). While on TKI, major molecular response (MMR) was achieved in 48% of children. Among the remaining patients, 21% are alive on TKI without MMR and 22% achieved complete molecular response following SCT. Twenty-one of 22 (95%) children are alive, while one patient died of posttransplant complications. CONCLUSION: This report demonstrates for the first time the efficacy and long-term effects of upfront imatinib in the so far largest cohort of children with CML diagnosed at very young age.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Transplante de Células-Tronco Hematopoéticas/mortalidade , Leucemia Mielogênica Crônica BCR-ABL Positiva/terapia , Sistema de Registros/estatística & dados numéricos , Pré-Escolar , Terapia Combinada , Feminino , Seguimentos , Humanos , Lactente , Leucemia Mielogênica Crônica BCR-ABL Positiva/patologia , Masculino , Prognóstico , Taxa de SobrevidaRESUMO
BACKGROUND: Invasive fungal infections, including hepatosplenic fungal infections (HSFI), cause significant morbidity and mortality in children with leukemia. There are not enough data to support for the best approach to diagnosis of HSFI in children, nor for the best treatment. PROCEDURE: In this multicentric study, we assessed the demographic data, clinical and radiologic features, treatment, and outcome of 40 children with leukemia and HSFI from 12 centers. RESULTS: All cases were radiologically diagnosed with abdominal ultrasound, which was performed at a median of 7 days, of the febrile neutropenic episode. Mucor was identified by histopathology in 1, and Candida was identified in blood cultures in 8 patients. Twenty-two had fungal infection in additional sites, mostly lungs. Nine patients died. Four received a single agent, and 36 a combination of antifungals. CONCLUSIONS: Early diagnosis of HSFI is challenging because signs and symptoms are usually nonspecific. In neutropenic children, persistent fever, back pain extending to the shoulder, widespread muscle pain, and increased serum galactomannan levels should alert clinicians. Abdominal imaging, particularly an abdominal ultrasound, which is easy to perform and available even in most resource-limited countries, should be recommended in children with prolonged neutropenic fever, even in the absence of localizing signs and symptoms.
Assuntos
Neutropenia Febril Induzida por Quimioterapia/imunologia , Leucemia/complicações , Hepatopatias/imunologia , Micoses/imunologia , Esplenopatias/imunologia , Adolescente , Antifúngicos/uso terapêutico , Neutropenia Febril Induzida por Quimioterapia/microbiologia , Criança , Pré-Escolar , Feminino , Humanos , Hospedeiro Imunocomprometido , Leucemia/imunologia , Hepatopatias/tratamento farmacológico , Hepatopatias/microbiologia , Masculino , Micoses/diagnóstico , Micoses/tratamento farmacológico , Estudos Retrospectivos , Esplenopatias/tratamento farmacológico , Esplenopatias/microbiologiaRESUMO
OBJECTIVES: To evaluate the long-term efficacy and safety of deferasirox therapy in a large observational cohort of children with transfusion-dependent thalassemia (TDT) and sickle cell anemia (SCA) in Turkey. METHODS: This was a multicenter, prospective cohort study including TDT and SCA patients aged 2-18 years with iron overload (≥100 mL/kg of pRBC or a serum ferritin [SF] level >1000 µg/L) receiving deferasirox. Patients were followed for up to 3 years according to standard practice. RESULTS: A total of 439 patients were evaluated (415 [94.5%] TDT, 143 [32.6%] between 2 and 6 years). Serum ferritin levels consistently and significantly decreased across 3 years of deferasirox therapy from a median of 1775.5 to 1250.5 µg/L (P < 0.001). Serum ferritin decreases were noted in TDT (1804.9 to 1241 µg/L), SCA (1655.5 to 1260 µg/L), and across age groups of 2-6 years (1971.5 to 1499 µg/L), 7-12 years (1688.5 to 1159.8 µg/L), and 13-18 years (1496.5 to 1107 µg/L). Serum ferritin decreases were also noted for all deferasirox dose groups but only significant in patients with doses ≥30 mg/kg/d (n = 120, -579.6 median reduction, P < 0.001). Only 9 (2%) patients had adverse events suspected to be related to deferasirox. Serum creatinine slightly increased but remained within the normal range. CONCLUSIONS: Deferasirox has long-term efficacy and safety in children with TDT and SCA, although higher doses (≥30 mg/kg/d) may be required to achieve iron balance.
Assuntos
Anemia Falciforme/complicações , Deferasirox/uso terapêutico , Quelantes de Ferro/uso terapêutico , Sobrecarga de Ferro/tratamento farmacológico , Sobrecarga de Ferro/etiologia , Talassemia/complicações , Adolescente , Anemia Falciforme/terapia , Biomarcadores , Transfusão de Sangue , Criança , Pré-Escolar , Estudos de Coortes , Deferasirox/administração & dosagem , Deferasirox/efeitos adversos , Feminino , Ferritinas/sangue , Ferritinas/metabolismo , Humanos , Ferro/sangue , Ferro/metabolismo , Quelantes de Ferro/administração & dosagem , Quelantes de Ferro/efeitos adversos , Sobrecarga de Ferro/metabolismo , Masculino , Talassemia/terapia , Resultado do Tratamento , TurquiaRESUMO
PURPOSE: To investigate the use of spectral domain optical coherence tomography (SD-OCT) findings in pediatric acute lymphoblastic leukemia (ALL) patients. METHODS: Children that were diagnosed with precursor B-cell ALL and classified as belonging to the medium-risk group for relapse were selected for this study. Individuals who were in continuous remission and on maintenance therapy were included in the study group. Cases that had central nervous system involvement were excluded. Age-matched, otherwise healthy children were selected for the control group. Each study participant underwent a comprehensive eye examination and SD-OCT evaluation. Thickness measurements were made within the retinal nerve fiber layer (RNFL), central macula, posterior polar, and peripapillary choroid. RESULTS: A total of 112 eyes of 56 children were included: 54 eyes in the study group and 58 in the control group. Compared to the control group, subfoveal and temporal choroidal thicknesses of the posterior pole were significantly thinner in the study group (P < 0.005). Similarly, peripapillary choroidal thicknesses were significantly thinner in most sectors of the study group (P < 0.005). There were no major differences between groups in terms of central macular thicknesses and overall RNFL thicknesses. CONCLUSIONS: Evidence of choroidal attenuation was found in this subgroup of pediatric ALL patients. Further studies are warranted to clarify the utility of SD-OCT in detecting subclinical ocular involvement and monitoring treatment response and risk of relapse in patients with pediatric leukemia.
Assuntos
Macula Lutea/patologia , Fibras Nervosas/patologia , Leucemia-Linfoma Linfoblástico de Células Precursoras/complicações , Doenças Retinianas/diagnóstico , Células Ganglionares da Retina/patologia , Tomografia de Coerência Óptica/métodos , Adolescente , Medula Óssea/patologia , Criança , Pré-Escolar , Corioide/patologia , Terapia Combinada , Estudos Transversais , Feminino , Humanos , Incidência , Masculino , Disco Óptico/patologia , Leucemia-Linfoma Linfoblástico de Células Precursoras/mortalidade , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Doenças Retinianas/epidemiologia , Doenças Retinianas/etiologia , Estudos Retrospectivos , Turquia/epidemiologiaRESUMO
Aceruloplasminemia is a rare autosomal recessive disease that affects the iron metabolism of the body. When there is a lack of ceruloplasmin ferroxidase activity, iron accumulates, especially in the brain, pancreas, liver, and retina. The first symptom is generally a persistent hypochromic microcytic anemia with a mild high-serum ferritin level. The affected patients are usually recognized at later ages, when the neurological symptoms appear. The neurological outcome has an adverse effect on the prognosis, which may result in fatality. Therefore, early diagnosis and intervention may prevent a devastating neurological damage. Here, we report a case of aceruloplasminemia in a teenage girl with hypochromic microcytic anemia.