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Anemia Aplásica , Antifúngicos , Mucormicosis , Rhizopus , Humanos , Mucormicosis/diagnóstico , Mucormicosis/microbiología , Anemia Aplásica/complicaciones , Anemia Aplásica/terapia , Rhizopus/aislamiento & purificación , Antifúngicos/uso terapéutico , Masculino , Niño , Diabetes Mellitus , Resultado del Tratamiento , Complicaciones de la Diabetes/microbiologíaRESUMEN
Autoimmune cytopenias are a heterogeneous group of disorders characterized by immune-mediated destruction of haematopoietic cell lines. Effective and well-tolerated treatment options for relapsed-refractory immune cytopenias are limited. In this study, the aim was to evaluate the efficacy and safety of sirolimus in this disease group within the paediatric age group. The study enrolled patients in the paediatric age group who used sirolimus with a diagnosis of immune cytopenia between December 2010 and December 2020, followed at six centres in Turkey. Of the 17 patients, five (29.4%) were treated for autoimmune haemolytic anaemia (AIHA), six (35.2%) for immune thrombocytopenic purpura (ITP) and six (35.2%) for Evans syndrome (ES). The mean response time was 2.7 months (range, 0-9 months). Complete response (CR) and partial response (PR) were obtained in 13 of 17 patients (76.4%) and nonresponse (NR) in four patients (23.5%). Among the 13 patients who achieved CR, three of them were NR in the follow-up and two of them had remission with low-dose steroid and sirolimus. Thus, overall response rate (ORR) was achieved in 12 of 17 patients (70.5%). In conclusion, sirolimus may be an effective and safe option in paediatric patients with relapsed-refractory immune cytopenia.
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Anemia Hemolítica Autoinmune , Inmunosupresores , Púrpura Trombocitopénica Idiopática , Sirolimus , Humanos , Sirolimus/uso terapéutico , Femenino , Masculino , Niño , Preescolar , Anemia Hemolítica Autoinmune/tratamiento farmacológico , Púrpura Trombocitopénica Idiopática/tratamiento farmacológico , Lactante , Adolescente , Inmunosupresores/uso terapéutico , Resultado del Tratamiento , Recurrencia , Turquía , Trombocitopenia/tratamiento farmacológico , Inducción de Remisión , CitopeniaRESUMEN
The purpose of this study was to identify risk factors and improve preventive strategies for febrile neutropenia (FEN) in children with leukemia who were receiving ciprofloxacin prophylaxis. The study included 100 children with leukemia [n=80 with acute lymphoblastic leukemia and n=20 with acute myeloblastic leukemia (AML)]. Patients were divided into two groups based on whether they had three or fewer FEN episodes (Group 1) or more than three FEN episodes (Group 2). Group 1 contained 63 (63%) of the 100 patients, while Group 2 contained 37 (37%). Older age (≥7 years), leukemia type, prolonged neutropenia (>10 days), and the presence of neutropenia and hypogammaglobulinemia at diagnosis were all risk factors for having more than three FEN episodes. Our findings suggest that, in addition to ciprofloxacin prophylaxis, identifying risk factors and improving preventive strategies could help reduce FEN episodes in children with leukemia.
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Neutropenia Febril , Leucemia Mieloide Aguda , Leucemia-Linfoma Linfoblástico de Células Precursoras , Humanos , Niño , Ciprofloxacina/uso terapéutico , Leucemia Mieloide Aguda/complicaciones , Leucemia Mieloide Aguda/tratamiento farmacológico , Leucemia-Linfoma Linfoblástico de Células Precursoras/complicaciones , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamiento farmacológico , Factores de Riesgo , Neutropenia Febril/etiología , Neutropenia Febril/prevención & controlRESUMEN
INTRODUCTION: This is the first study of simultaneous occurrences of Familial Mediterranean Fever (FMF) in patients with haemophilia. AIM: The aim was to investigate the frequency and clinical characteristics of FMF in patients with severe haemophilia. METHODS: Our study included 30 patients with severe haemophilia (26 haemophilia A and four haemophilia B). All 30 patients are screened for MEFV genotypes in FMF according to the new Eurofever/PRINTO diagnostic criteria. All cohorts were genetically tested for FMF and thrombophilia. RESULTS: Eight (26%) of 30 haemophilic patients were diagnosed with FMF. Six different heterozygous FMF mutations including M694V (n = 2), E148Q (n = 2), V726A(n = 1), P369S (n = 1), E148Q/K695R (n = 1) and E148Q/M694I (n = 1) were identified. Seven had haemophilia A and only one had haemophilia B. Four (50%) patients had a positive family history and three of them had extraarticular findings specific to FMF. Only one haemophilia B patient received colchicine. Target joints in the knee, ankle, and elbow were identified in three FMF patients. The number of target joints in eight patients with FMF was significantly lower than in twenty-two patients without FMF (p < .05). The annual number of suspected joint bleedings in FMF patients admitted to the hospital was 40; however, 15 (37.5%) were documented bleedings in ultrasounds. Hereditary thrombophilia was detected in seven of eight patients. CONCLUSION: Our data indicate that screening for FMF may be useful in patients with haemophilia who develop arthritis without prominent bleeding and have a positive family history in many Mediterranean countries, including Turkey.
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Fiebre Mediterránea Familiar , Hemofilia A , Hemofilia B , Humanos , Fiebre Mediterránea Familiar/complicaciones , Fiebre Mediterránea Familiar/diagnóstico , Fiebre Mediterránea Familiar/epidemiología , Hemofilia A/complicaciones , Hemofilia A/epidemiología , Pirina/genética , FenotipoAsunto(s)
Anomalías Múltiples , Síndrome de Noonan , Leucemia-Linfoma Linfoblástico de Células T Precursoras , Síndrome de Sotos , Niño , Humanos , Síndrome de Noonan/complicaciones , Síndrome de Noonan/genética , Leucemia-Linfoma Linfoblástico de Células T Precursoras/complicaciones , Leucemia-Linfoma Linfoblástico de Células T Precursoras/genéticaRESUMEN
BACKGROUND: This study aims to investigate the diagnostic utility of new erythrocytes, leukocytes, and reticulocytes parameters for the identification of subclinical iron deficiency (ID) in children under 6 years with nonsevere acute infection in pediatric outpatients. METHODS: The study included 102 children with acute infections and 31 true ID. Traditional and new hematology parameters were measured in a Sysmex-XN®, along with C-reactive protein level, and iron parameters. Participants' ID were categorized as: the ferritin < 100 ng/mL, transferrin saturation < 20% was defined as "subclinical or functional ID (FID) in Group 1"; ferritin < 30 ng/mL, transferrin saturation < 20%, as "absolute-ID (AID)" in Group 2; ferritin < 12 ng/mL without anemia and infection, as "true ID" in Group 3. RESULTS: The frequencies of FID and AID among the 102 children with acute infection were 24% and 76%, respectively. Compared with the Group 2 patients, Group 1 had a significantly higher mean percentage of hypochromic erythrocytes (Hypo-He), and significantly lower levels of hemoglobin (Hb) and Hb content of reticulocytes (RET-He) (p < 0.05 for all). Compared with Group 2 and Group 3 patients, Group 1 had a significantly higher mean percentage of immature reticulocyte fraction (IRF) and immature granulocyte (IG) values (p < 0.05 for all). The RET-He, IRF%, Hypo-He%, and IG% cut-off values for predicting FID during infection were 27.0 pg, 10.6%, 2.5%, and 0.35% respectively. DISCUSSION: The RET-He, Hypo-He, IRF, and IG may be useful parameters for identifying subclinical ID in small children with nonsevere acute infection in pediatric outpatients.
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Anemia Ferropénica , Deficiencias de Hierro , Humanos , Niño , Preescolar , Reticulocitos/química , Reticulocitos/metabolismo , Pacientes Ambulatorios , Anemia Ferropénica/diagnóstico , Anemia Ferropénica/metabolismo , Eritrocitos/metabolismo , Hemoglobinas/análisis , Ferritinas/metabolismo , Leucocitos/metabolismo , Transferrinas/metabolismoRESUMEN
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.
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Trasplante de Células Madre Hematopoyéticas , Refugiados , Humanos , Niño , Preescolar , Calidad de Vida/psicología , Turquía , Estudios Retrospectivos , Trasplante de Células Madre Hematopoyéticas/psicologíaRESUMEN
This is the first study to have investigated the prognostic role of cytokines and soluble human leukocyte antigen-G (sHLA-G) levels in pediatric leukemia patients who have undergone allogeneic stem cell transplantation (allo-SCT). Forty-one patients with acute leukemia (n = 28, acute lymphoblastic leukemia (ALL) and n = 13, acute myeloblastic leukemia) were recruited. Patients were examined at diagnosis (n = 26), in the pre-transplantation period (PreTx) (n = 26), on the day of transplantation (Tx0) (n = 41), and on post-transplantation Days 14 (PostTx14) (n = 41) and 28 (PostTx28) (n = 41). Serum levels of pro-inflammatory cytokines (interleukin [IL]-1, IL-2, IL-6, Tumor necrosis factor [TNF]-α), anti-inflammatory cytokines (IL-4, IL-10), and sHLA-G were measured by Enzyme-Linked ImmunoSorbent Assay. Median levels of all cytokines tested and sHLA-G were significantly higher at diagnosis and at the post-transplant time points than at PreTx (all p < 0.05). At the time of diagnosis (specifically ALL) and at PostTx14, elevated IL-4, IL-10, and/or sHLA-G were associated with higher post-transplant relapse rates (all p < 0.05). Elevated IL-2 and TNF-α at Tx0 were associated with lower survival rates (both p < 0.05). Levels of serum cytokines and sHLA-G may be useful predictors of survival and relapse in pediatric leukemia patients who undergo allo-SCT.
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Trasplante de Células Madre Hematopoyéticas , Leucemia , Niño , Citocinas , Antígenos HLA-G , Humanos , Interleucina-10 , Interleucina-2 , Interleucina-4 , Leucemia/terapia , Pronóstico , Recurrencia , Factor de Necrosis Tumoral alfaRESUMEN
It is well known that stem cell transplantation is curative for many diseases; however, graft versus host disease (GVHD), which is a common posttransplant complication, has still a substantial place among the causes of transplant-related morbidity and mortality. The association between ABO incompatibility and GVHD is controversial. There is also limited available data about the association between blood component transfusions during the peritransplant period and GVHD development in the pediatric setting. Hence, we retrospectively evaluated both the impact of ABO-mismatch and transfusions of RBC and platelets between day -7 pre-transplant and +30 post-transplant to the development of acute GVHD (aGVHD). We analyzed 139 allotransplants in 133 patients who were transplanted by myeloablative conditioning. Fifty-one patients out of 133 (36.7 %) were found to have aGVHD within +100 days post-transplantation. Of them 40 patients had grade I-II and 11 patients had grade III-IV aGVHD. Increased risk of aGVHD is associated with ABO minor mismatch (p: 0.030). Nevertheless, there was no association between ABO mismatch and severity of aGVHD. The median number of RBC transfusions in aGVHD patients was higher than the number of transfusions in patients without aGVHD; however, the difference was not statistically significant (p: 0.11). Platelet transfusion numbers were statistically similar between aGVHD patients and the patients without aGVHD (p: 0.79). In conclusion, major and bi-directional ABO-incompatibility between donors and recipients, and RBC and platelet transfusions between day -7 pretransplant and day +30 post-transplant do not contribute to aGVHD development in children undergoing HSCT by myeloablative conditioning, while ABO minor mismatch is associated with the development of aGVHD.
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Anemia Hemolítica Autoinmune , Enfermedad Injerto contra Huésped , Trasplante de Células Madre Hematopoyéticas , Sistema del Grupo Sanguíneo ABO , Incompatibilidad de Grupos Sanguíneos , Niño , Enfermedad Injerto contra Huésped/etiología , Trasplante de Células Madre Hematopoyéticas/efectos adversos , Humanos , Estudios RetrospectivosRESUMEN
Thoracic air leak syndromes (TALS) are very rare among the noninfectious pulmonary complications (PCs). They can either be idiopathic or have several risk factors such as allogeneic hematopoietic stem cell transplantation (allo-HSCT), graft versus host disease and rarely pulmonary aspergillosis. We present a 14-year-old girl with hypoplastic myelodysplastic syndrome who developed graft versus host disease on day 60, TALS on day 150, bronchiolitis obliterans syndrome on day 300, pulmonary aspergillosis on day 400 and COVID-19 pneumonia on day 575 after allo-HSCT. This is the first report of a child who developed these subsequent PCs after allo-HSCT. Therefore, the manifestations of these unfamiliar PCs like TALS and COVID-19 pneumonia, and concomitant pulmonary aspergillosis with management options are discussed.
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COVID-19/complicaciones , Enfermedad Injerto contra Huésped/patología , Trasplante de Células Madre Hematopoyéticas/efectos adversos , Síndromes Mielodisplásicos/terapia , Neumonía Viral/patología , Aspergilosis Pulmonar/patología , Enfisema Pulmonar/patología , Adolescente , COVID-19/virología , Femenino , Enfermedad Injerto contra Huésped/etiología , Humanos , Síndromes Mielodisplásicos/patología , Neumonía Viral/etiología , Pronóstico , Aspergilosis Pulmonar/etiología , Enfisema Pulmonar/etiología , Factores de Riesgo , SARS-CoV-2/aislamiento & purificaciónRESUMEN
This study investigated the frequency of and predictive factors for autoimmune lymphoproliferative syndrome (ALPS) in children with lymphoma, chronic immune cytopenia, and nonmalignant organomegaly. Thirty-four children with suspected ALPS (n=13, lymphoma; n=12, immune cytopenia; n=9, nonmalignant organomegaly) were included. Double-negative T-cells, lymphocyte apoptosis, and genetic findings were analyzed. Patients were stratified into two groups as proven/probable ALPS and clinically suspected patients according to the ALPS diagnostic criteria. Of the 34 patients, 18 (53%) were diagnosed with proven/probable ALPS. One patient had a mutation (c.652-2A>C) in the FAS gene. The remaining 16 (47%) patients were defined as clinically suspected patients. Predictive factors for ALPS were anemia and thrombocytopenia in patients with lymphoma, splenomegaly and lymphadenopathy in patients with immune cytopenia, and young age in patients with nonmalignant organomegaly. ALPS may not be rare in certain risk groups. Our study indicates that screening for ALPS may be useful in children having lymphoma with cytopenia at diagnosis, in those having nonmalignant organomegaly with immune cytopenia, and in those having chronic immune thrombocytopenic purpura or autoimmune hemolytic anemia with organomegaly developing during follow-up.
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Síndrome Linfoproliferativo Autoinmune/diagnóstico , Leucopenia/diagnóstico , Linfoma/diagnóstico , Trombocitopenia/diagnóstico , Adolescente , Anemia/diagnóstico , Anemia/etiología , Anemia/inmunología , Anemia Hemolítica Autoinmune/diagnóstico , Anemia Hemolítica Autoinmune/etiología , Anemia Hemolítica Autoinmune/inmunología , Apoptosis/inmunología , Síndrome Linfoproliferativo Autoinmune/complicaciones , Síndrome Linfoproliferativo Autoinmune/inmunología , Síndrome Linfoproliferativo Autoinmune/patología , Niño , Preescolar , Enfermedad Crónica , Estudios Transversales , Femenino , Humanos , Lactante , Leucopenia/etiología , Leucopenia/inmunología , Linfadenopatía/diagnóstico , Linfadenopatía/etiología , Linfadenopatía/inmunología , Linfoma/etiología , Linfoma/inmunología , Masculino , Mutación , Valor Predictivo de las Pruebas , Púrpura Trombocitopénica Idiopática/diagnóstico , Púrpura Trombocitopénica Idiopática/etiología , Púrpura Trombocitopénica Idiopática/inmunología , Esplenomegalia/diagnóstico , Esplenomegalia/etiología , Esplenomegalia/inmunología , Linfocitos T/inmunología , Linfocitos T/patología , Trombocitopenia/etiología , Trombocitopenia/inmunología , Receptor fas/genéticaRESUMEN
BACKGROUND: Renal dysfunction is an underestimated complication of thalassemia major. OBJECTIVES: The aim of this study is to compare the glomerular and tubular functions in children with ß- Thalassemia major (ß- TM) with healthy controls and assess the oxidative stress caused by high ferritin levels. DESIGN AND SETTING: This prospective cross-sectional study was conducted in tertiary care hospital. METHODS: Complete blood count (CBC), calcium (Ca), urea, creatinine (Cr), serum cystatin C before transfusion and urinary calcium (uCa), creatinine (uCr), protein (UPr) levels were analyzed in fresh samples. Beta-2-microglobulin (uß2-MG), N- acetylglucosaminidase (uNAG), retinol binding protein (uRBP), malonedialdehyde (uMDA) secretion and creatinine levels were analyzed. Serum total antioxidant capacity (sTAC) and total oxidant capacity (sTOC) were measured with colorimetric micro-ELISA method. Last four serum ferritin values were recorded and the mean value was used for statistical analyzes. RESULTS: Data from 47 patients and 32 controls were analyzed. The urinary RBP/Cr, Ca/Cr and Protein/Cr, were significantly higher in ß-TM group. A statistically insignificant increase in urinary ß2MG/Cr, uNAG/Cr, MDA/Cr was also found in the TM group. Proteinuria was present in 46 % (n: 22) and hypercalciuria in 34 % (n: 16) of the patients with ß- TM. Serum total antioxidant capacity and total oxidant status (TOS) levels were significantly elevated in the patient group. Serum ferritin was significantly correlated with proteinuria, cystatin C levels, urinary Protein/Cr and uRBP/Cr. CONCLUSION: Asymptomatic renal dysfunction is prevalent in ß- TM patients that necessitate regular screening. Urinary RBP may be useful for early diagnosis.
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Pruebas de Función Renal/métodos , Estrés Oxidativo/fisiología , Estudios de Casos y Controles , Niño , Estudios Transversales , Femenino , Humanos , Masculino , Estudios Prospectivos , Talasemia betaRESUMEN
AIM: Since the beginning of the Syrian civil war, more than 3.5 million Syrians have been under temporary protection status in Turkey. Because beta-thalassemia (BT) is a prevalent disorder in the Mediterranean countries, we decided to estimate the prevalence of and make an overview of the demographic, socioeconomic, medical characteristics, and healthcare problems of refugee children with BT. PATIENTS: Eighteen Turkish Pediatric Hematology Oncology Centers (PHOC) with 318 refugee children from 235 families participated in the study. The mean age of the patients was 8.1 ± 4.8 years (0.5-21 years). The mean time after immigration to Turkey was 2.5 ± 1.5 years (range, 0.1-7 years). Seventy-two (22.6%) of them were born and diagnosed with BT in Turkey. On physical examination, 82 patients (26%) were underweight and 121 patients (38%) were stunted. The appearance of a thalassemic face was reported for 207 patients (65.1%). Hepatomegaly and splenomegaly were reported in 217 (68.2%) and 168 (52.8%) patients, respectively. The median ferritin level was 2508 ng/mL (range, 17-21 000 ng/mL) at the first admission, and 2841 ng/mL (range, 26-12 981 ng/mL) at the last visit after two years of follow-up in a PHOC (P > 0.05). The most frequently encountered mutation was IVSI-110 (G>A) (31%). Before immigration, only 177 patients (55.6%) reported the use of chelators; after immigration it increased to 268 (84.3%). CONCLUSION: Difficulties in communication, finding a competent translator capable in medical terminology, nonregular use of medications, and insensitivity to prenatal diagnosis were preliminary problems. The current extent of migration poses emerging socioeconomic and humanitarian challenges for refugee patients with BT.
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Emigración e Inmigración/estadística & datos numéricos , Hospitalización/estadística & datos numéricos , Refugiados/estadística & datos numéricos , Factores Socioeconómicos , Talasemia beta/epidemiología , Adolescente , Adulto , Niño , Preescolar , Estudios Transversales , Demografía , Femenino , Estudios de Seguimiento , Humanos , Lactante , Masculino , Prevalencia , Pronóstico , Tasa de Supervivencia , Turquía/epidemiología , Adulto Joven , Talasemia beta/terapiaRESUMEN
OBJECTIVE: To report a rare side effect of metformin, an oral antidiabetic drug that is used for the treatment of type 2 diabetes mellitus. CLINICAL PRESENTATION AND INTERVENTION: A 17-year-old boy was hospitalized for receiving acute lymphoblastic leukemia treatment that was composed of vincristine, L-asparaginase, daunorubicin, and prednisone. Hyperglycemia was determined without any clinical sign and metformin was started for steroid-induced insulin resistance. On the second day of metformin treatment, the patient's hemoglobin level decreased, and a direct Coombs test was positive for immunoglobulin G but negative for complement. An indirect Coombs test was negative. The glucose-6-phosphate dehydrogenase level was within the normal range. Drug-induced hemolytic anemia was suspected and metformin was discontinued. The jaundice gradually disappeared and there was no requirement for red blood cell transfusions. CONCLUSION: This case showed that physicians should be aware of the potential side effect of metformin although it is infrequent.