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1.
Clin Transplant ; 38(5): e15366, 2024 May.
Artículo en Inglés | MEDLINE | ID: mdl-38775798

RESUMEN

In children with high-risk childhood acute leukemia who undergo allogeneic hematopoietic stem-cell transplantation (allo-HSCT), relapse is still the leading cause of treatment failure. The prognosis is poor, yet prospective studies have only limited data on risk factors and outcomes. We aimed to understand the outcomes and prognostic factors for patients with acute lymphoblastic leukemia (ALL) who relapsed following allo-HSCT. We analyzed retrospectively 46 children with childhood acute lymphoblastic leukemia who had relapsed after receiving their first alloHSCT. All these patients received salvage chemotherapy which consisted of fludarabine, cytarabine, and idarubicin before performing a second alloHSCT. The median follow-up of the 46 patients after the first transplantation was 366 days. The median time from first allo-HSCT to relapse was 278.4 ± 238.4 days. Forty-six patients received salvage chemotherapy before the second alloHSCT, and CR was achieved in 32 of 46 patients. However, only 17 (37%) of 46 patients received a second allo-HSCT, and 15 of 46 patients died from disease progression, infections, and bleeding. Twelve patients are still alive after the second allo-HSCT. Two-year overall survival (OS) was 38.9%. Local therapy was given to 10 (21.8%) patients, either as part of systemic therapy or alone. In multivariate analyses, the time of relapse and curative salvage therapy with a second allo-HSCT were identified as significant prognostic factors for OS. Children with leukemia who had relapsed after the first allo-HSCT received salvage chemotherapy. Our statistical analysis showed that the second HSCT could be beneficial for outcomes if patients relapsed beyond 180 days of the first allo-HSCT.


Asunto(s)
Trasplante de Células Madre Hematopoyéticas , Leucemia-Linfoma Linfoblástico de Células Precursoras , Trasplante Homólogo , Humanos , Femenino , Masculino , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Leucemia-Linfoma Linfoblástico de Células Precursoras/mortalidad , Niño , Estudios Retrospectivos , Preescolar , Pronóstico , Estudios de Seguimiento , Adolescente , Tasa de Supervivencia , Recurrencia Local de Neoplasia/patología , Recurrencia Local de Neoplasia/terapia , Factores de Riesgo , Lactante , Enfermedad Injerto contra Huésped/etiología , Terapia Recuperativa , Acondicionamiento Pretrasplante , Recurrencia
2.
Klin Padiatr ; 236(3): 180-188, 2024 May.
Artículo en Inglés | MEDLINE | ID: mdl-38729129

RESUMEN

BACKGROUND: Oral mucositis is one of the side effects developed post-hematopoietic stem cell transplant. This retrospective study aimed to assess the efficacy of a mouthwash mixture (lidocaine, sodium alginate, sucralfate, pheniramine) versus hyaluronic acid and a solution of sodium bicarbonate in terms of healing time and weight gain in the treatment of oral mucositis in pediatric patients undergoing allogeneic hematopoietic stem cell transplantation with hemato-oncological malignancies. METHODS: A total of 171 patients that received chemotherapy for the hematopoietic stem cell transplant were divided into three groups; group 1, treated with a mixed mouthwash of lidocaine, sodium alginate, sucralfate, and pheniramine; group 2, treated with hyaluronic acid; and group 3, treated with an aqueous solution of 5% sodium bicarbonate. Weight and mucositis scale scores derived from medical records of patients. RESULTS: There was a statistically significant difference in the mucositis scale scores between the groups on the transplant day and days 5, 10, 15 and 20 after the transplantation. At these measurement points, Group 2 (receiving hyaluronic acid) had a lower score, and Group 3 (who received sodium bicarbonate) had a higher score, especially on days 5 and 10 after the transplantation. CONCLUSION: The results suggest that hyaluronic acid is a more effective treatment option than the other oral care solutions that are frequently used for prophylaxis and treatment of oral mucositis.


Asunto(s)
Trasplante de Células Madre Hematopoyéticas , Estomatitis , Humanos , Trasplante de Células Madre Hematopoyéticas/efectos adversos , Niño , Estomatitis/prevención & control , Estomatitis/inducido químicamente , Estomatitis/tratamiento farmacológico , Masculino , Femenino , Estudios Retrospectivos , Adolescente , Preescolar , Antisépticos Bucales/uso terapéutico , Ácido Hialurónico/uso terapéutico , Ácido Hialurónico/administración & dosificación , Ácido Hialurónico/efectos adversos , Bicarbonato de Sodio/uso terapéutico , Bicarbonato de Sodio/administración & dosificación , Higiene Bucal , Antineoplásicos/efectos adversos , Neoplasias Hematológicas/terapia , Lidocaína/uso terapéutico , Sucralfato/uso terapéutico
3.
J Pediatr Hematol Oncol ; 44(1): e35-e39, 2022 01 01.
Artículo en Inglés | MEDLINE | ID: mdl-34966102

RESUMEN

Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is one of the most severe and life-threatening complications after hematopoietic stem cell transplantation (HSCT). Defibrotide (DF) is approved for adult and pediatric patients with VOD/SOS with renal or pulmonary dysfunction after HSCT in the United States, and for severe VOD/SOS post-HSCT in patients above 1 month of age in the European Union. Several studies have examined whether DF prophylaxis can reduce the incidence of VOD/SOS in high-risk patients. A total of 334 pediatric allogeneic HSCT were included in this study. All patients received DF at the dose of 25 mg/kg/d, from the first day of the conditioning regimen to the 30th day after transplantation for VOD/SOS prophylaxis. Seventeen patients (5.08%) developed VOD/SOS; 4 of these had moderate, while 13 had mild VOD/SOS. None of the patients were developed severe or very severe VOD/SOS. In conclusion, we showed that prophylactic intervention with DF lowered the incidence of VOD/SOS in high-risk pediatric patients.


Asunto(s)
Trasplante de Células Madre Hematopoyéticas , Enfermedad Veno-Oclusiva Hepática , Polidesoxirribonucleótidos/administración & dosificación , Acondicionamiento Pretrasplante , Adolescente , Adulto , Aloinjertos , Niño , Preescolar , Femenino , Enfermedad Veno-Oclusiva Hepática/epidemiología , Enfermedad Veno-Oclusiva Hepática/etiología , Enfermedad Veno-Oclusiva Hepática/prevención & control , Humanos , Incidencia , Lactante , Masculino , Polidesoxirribonucleótidos/efectos adversos , Estudios Retrospectivos , Estados Unidos
4.
Eur J Haematol ; 102(2): 123-130, 2019 Feb.
Artículo en Inglés | MEDLINE | ID: mdl-30300449

RESUMEN

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.


Asunto(s)
Anemia de Células Falciformes/complicaciones , Deferasirox/uso terapéutico , Quelantes del Hierro/uso terapéutico , Sobrecarga de Hierro/tratamiento farmacológico , Sobrecarga de Hierro/etiología , Talasemia/complicaciones , Adolescente , Anemia de Células Falciformes/terapia , Biomarcadores , Transfusión Sanguínea , Niño , Preescolar , Estudios de Cohortes , Deferasirox/administración & dosificación , Deferasirox/efectos adversos , Femenino , Ferritinas/sangre , Ferritinas/metabolismo , Humanos , Hierro/sangre , Hierro/metabolismo , Quelantes del Hierro/administración & dosificación , Quelantes del Hierro/efectos adversos , Sobrecarga de Hierro/metabolismo , Masculino , Talasemia/terapia , Resultado del Tratamiento , Turquía
5.
Pediatr Blood Cancer ; 66(5): e27636, 2019 05.
Artículo en Inglés | MEDLINE | ID: mdl-30706992

RESUMEN

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.


Asunto(s)
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/terapia
6.
Pediatr Hematol Oncol ; 36(6): 376-381, 2019 Sep.
Artículo en Inglés | MEDLINE | ID: mdl-31522599

RESUMEN

Deficiency of Adenosine Deaminase 2 (DADA2) is a monogenic autoinflammatory disorder characterized by livedo reticularis, skin ulcers, subcutaneous rash, aphthous ulcers, and leukocytoclastic vasculitis, neurological signs such as early onset stroke and polyneuropathy. A minority of DADA2 patients suffer from severe cytopenia and lymphoproliferation. Herein, we report an adolescent patient, followed up as having a hematological disorder for many years, eventually diagnosed as having DADA2. In view of the presence of elevated acute phase reactants, hepatosplenomegaly, low IgM level, lymphopenia, anemia, and neutropenia, and a subtle neurological involvement we considered DADA2 diagnosis. The diagnosis was confirmed by identification of a novel L451W mutation in CECR1 gene. The patient has been successfully treated with etanercept, monthly intravenous immunoglobulin replacement, and low-dose methylprednisolone. In conclusion, although the absence of skin and neurological findings, low IgM levels, and persistent lymphopenia should lead the physicians to consider DADA2 in patients with particularly complicated hematological abnormalities.


Asunto(s)
Adenosina Desaminasa/deficiencia , Péptidos y Proteínas de Señalización Intercelular/genética , Linfopenia/genética , Trastornos Linfoproliferativos/genética , Adenosina Desaminasa/genética , Adolescente , Enfermedad Crónica , Femenino , Homocigoto , Humanos , Linfopenia/sangre , Trastornos Linfoproliferativos/sangre , Masculino , Mutación
7.
Turk J Pediatr ; 65(4): 693-697, 2023.
Artículo en Inglés | MEDLINE | ID: mdl-37661686

RESUMEN

BACKGROUND: Pneumatosis cystoides intestinalis (PI) is a rare but important condition in which widespread air sacs are found in the submucosa, and subserosa of the bowel wall. Although it has several etiologies, children receiving chemotherapy are at risk for PI. Preferred imaging tools for the diagnosis are abdominal direct radiography and computed tomography. In patients with PI, rupture of intramural air sacs is the source of benign pneumoperitoneum, causing free air without true intestinal perforation. Intestinal perforation or obstruction are indications for surgical intervention. CASE: Here, we present a 4-year-old patient diagnosed with acute myeloblastic leukemia (AML), who underwent allogeneic hematopoietic stem cell transplantation (HSCT) from a matched sibling donor (MSD) and developed PI after HSCT. The patient was consulted to the pediatric surgery department, and her oral feeding was stopped. Broad spectrum antibiotics (teicoplanin, metronidazol and vancomycin) were initiated. Her fever increased during the 24-hour monitoring, there was no stool passage, CRP ( > 25 mg/dL, normal value < 1 mg/dL) and abdominal distension increased and there was prolonged neutropenia and radiologic investigations could not rule out intestinal perforation, so the patient underwent exploratory laparotomy. No intestinal perforation was found. There was no sign in the intestinal wall and numerous gas-filled cysts of various sizes. CONCLUSIONS: PI is an uncommon complication, and direct radiography/computed tomography scans are very helpful in making the diagnosis in suspicious cases. PI, should be kept in mind, especially in transplanted or relapsed leukemia patients receiving intensive chemotherapy.


Asunto(s)
Trasplante de Células Madre Hematopoyéticas , Leucemia Mieloide Aguda , Neumatosis Cistoide Intestinal , Animales , Femenino , Humanos , Niño , Preescolar , Neumatosis Cistoide Intestinal/inducido químicamente , Neumatosis Cistoide Intestinal/diagnóstico por imagen , Leucemia Mieloide Aguda/terapia , Donantes de Tejidos , Antibacterianos
8.
JAMA Netw Open ; 6(5): e2312903, 2023 05 01.
Artículo en Inglés | MEDLINE | ID: mdl-37219908

RESUMEN

Importance: Cancer was a common noncommunicable disease in Syria before the present conflict and is now a major disease burden among 3.6 million Syrian refugees in Turkey. Data to inform health care practice are needed. Objective: To explore sociodemographic characteristics, clinical characteristics, and treatment outcomes of Syrian patients with cancer residing in the southern border provinces of Turkey hosting more than 50% of refugees. Design, Setting, and Participants: This was a retrospective hospital-based cross-sectional study. The study sample consisted of all adult and children Syrian refugees diagnosed and/or treated for cancer between January 1, 2011, and December 31, 2020, in hematology-oncology departments of 8 university hospitals in the Southern province of Turkey. Data were analyzed from May 1, 2022, to September 30, 2022. Main Outcomes and Measures: Demographic characteristics (date of birth, sex, and residence), date of first cancer-related symptom, date and place of diagnosis, disease status at first presentation, treatment modalities, date and status at last hospital visit, and date of death. The International Statistical Classification of Diseases and Related Health Problems, Tenth Revision and International Classification of Childhood Cancers, Third Edition, were used for the classification of cancer. The Surveillance, Epidemiology, and End Results system was applied for staging. The diagnostic interval was defined as the number of days from first symptoms until the diagnosis. Treatment abandonment was documented if the patient did not attend the clinic within 4 weeks of a prescribed appointment throughout the treatment. Results: A total of 1114 Syrian adult and 421 Syrian children with cancer were included. The median age at diagnosis was 48.2 (IQR, 34.2-59.4) years for adults and 5.7 (IQR, 3.1-10.7) years for children. The median diagnostic interval was 66 (IQR, 26.5-114.3) days for adults and 28 (IQR, 14.0-69.0) days for children. Breast cancer (154 [13.8%]), leukemia and multiple myeloma (147 [13.2%]), and lymphoma (141 [12.7%]) were common among adults, and leukemias (180 [42.8%]), lymphomas (66 [15.7%]), and central nervous system neoplasms (40 [9.5%]) were common among children. The median follow-up time was 37.5 (IQR, 32.6-42.3) months for adults and 25.4 (IQR, 20.9-29.9) months for children. The 5-year survival rate was 17.5% in adults and 29.7% in children. Conclusions and Relevance: Despite universal health coverage and investment in the health care system, low survival rates were reported in this study for both adults and children with cancer. These findings suggest that cancer care in refugees requires novel planning within national cancer control programs with global cooperation.


Asunto(s)
Leucemia , Refugiados , Adulto , Niño , Humanos , Siria , Estudios Transversales , Estudios Retrospectivos , Turquía , Instituciones de Atención Ambulatoria , Hospitales Universitarios
9.
Bone Marrow Transplant ; 57(5): 760-767, 2022 05.
Artículo en Inglés | MEDLINE | ID: mdl-35210564

RESUMEN

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.


Asunto(s)
Enfermedad Injerto contra Huésped , Trasplante de Células Madre Hematopoyéticas , Talasemia , Talasemia beta , Niño , Enfermedad Injerto contra Huésped/etiología , Trasplante de Células Madre Hematopoyéticas/efectos adversos , Humanos , Estudios Retrospectivos , Talasemia/complicaciones , Talasemia/terapia , Acondicionamiento Pretrasplante/efectos adversos , Turquía/epidemiología , Talasemia beta/complicaciones , Talasemia beta/terapia
10.
Turk J Pediatr ; 63(1): 11-22, 2021.
Artículo en Inglés | MEDLINE | ID: mdl-33686822

RESUMEN

BACKGROUND: Mucormycosis is a fatal invasive fungal infection seen most often in patients with compromised defense mechanisms. The aim of this article was to review the data of pediatric mucor in the South of Turkey. METHODS: Twenty pediatric cases with biopsy proven mucormycosis were reported, between January 2007 through January 2017. Data were extracted from the medical charts of patients retrospectively. RESULTS: Underlying conditions were hematological malignancy (75%), in whom 93% had acute leukemia, aplastic anemia (15%), diabetes mellitus (5%) and other malignancies (5%). The main sites of infection were sinus (85%); alone (29.4%) or with cerebral (17.6%), and orbital involvement (17.6%). Pulmonary involvement was reported in 11 patients (55%), two of them had the alone form and nine cases were associated with nasal sinus involvement. Disseminated mucormycosis was documented in 45%. Fever and pain/swelling of organs were the most commonly encountered signs and symptoms. Treatment compromised of am-photericin B monotherapy in five patients. All patients except one received liposomal formu-lations (LAmB). A combination of surgery and antifungal therapy was performed in 75%. Crude survival was 55%; among 15 cases treated with a combination of surgery and antifun-gal therapy, survival rate was 8/15 (53%). The overall mortality rate was high in patients diagnosed with disseminated infection (100%). CONCLUSIONS: Mucormycosis in pediatric cases requires a high index of suspicion and urgent evaluation of clinical samples. Surgical debridement should be considered when feasible. Initial medical therapy should include an amphotericin preparation with or step-down to posaconazole.


Asunto(s)
Mucormicosis , Anfotericina B , Antifúngicos/uso terapéutico , Niño , Humanos , Mucormicosis/diagnóstico , Mucormicosis/epidemiología , Mucormicosis/terapia , Estudios Retrospectivos , Turquía/epidemiología
11.
Turk J Haematol ; 38(2): 101-110, 2021 06 01.
Artículo en Inglés | MEDLINE | ID: mdl-33508912

RESUMEN

Objective: This study aimed to observe the preventive effect of prophylactic treatment on joint health in people with hemophilia (PwH) and to investigate the importance of integration of ultrasonographic examination into clinical and radiological evaluation of the joints. Materials and Methods: This national, multicenter, prospective, observational study included male patients aged ≥6 years with the diagnosis of moderate or severe hemophilia A or B from 8 centers across Turkey between January 2017 and March 2019. Patients were followed for 1 year with 5 visits (baseline and 3th, 6th, 9th, and 12th month visits). The Hemophilia Joint Health Score (HJHS) was used for physical examination of joints, the Pettersson scoring system was used for radiological assessment, point-of-care (POC) ultrasonography was used for bilateral examinations of joints, and the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) score was used for evaluation of ultrasonography results. Results: Seventy-three PwH, of whom 62 had hemophilia A and 11 had hemophilia B, were included and 24.7% had target joints at baseline. The HJHS and HEAD-US scores were significantly increased at the 12th month in all patients. These scores were also higher in the hemophilia A subgroup than the hemophilia B subgroup. However, in the childhood group, the increment of scores was not significant. The HEAD-US total score was significantly correlated with both the HJHS total score and Pettersson total score at baseline and at the 12th month. Conclusion: The HEAD-US and HJHS scoring systems are valuable tools during follow-up examinations of PwH and they complement each other. We suggest that POC ultrasonographic evaluation and the HEAD-US scoring system may be integrated into differential diagnosis of bleeding and long-term monitoring for joint health as a routine procedure.


Asunto(s)
Hemofilia A/prevención & control , Artropatías/diagnóstico , Proyectos de Investigación/estadística & datos numéricos , Ultrasonografía/métodos , Adolescente , Adulto , Anciano , Niño , Diagnóstico Diferencial , Diagnóstico Precoz , Estudios de Seguimiento , Hemofilia A/diagnóstico , Hemofilia A/terapia , Hemorragia/diagnóstico , Hemorragia/etiología , Humanos , Artropatías/prevención & control , Articulaciones/diagnóstico por imagen , Articulaciones/patología , Masculino , Persona de Mediana Edad , Pruebas en el Punto de Atención , Estudios Prospectivos , Factores Protectores , Proyectos de Investigación/tendencias , Índice de Severidad de la Enfermedad , Turquía/epidemiología
12.
Turk J Haematol ; 37(3): 139-144, 2020 08 28.
Artículo en Inglés | MEDLINE | ID: mdl-32181630

RESUMEN

Objective: Immune thrombocytopenia (ITP) is a rare autoimmune disease and hematologic disorder characterized by reduced platelet counts that can result in significant symptoms, such as bleeding, bruising, epistaxis, or petechiae. The thrombopoietin receptor agonist eltrombopag (EPAG) is a second-line agent used to treat chronic ITP purpura in adults and children. Materials and Methods: The present retrospective study evaluated the efficacy, safety, and side effects of EPAG treatment in pediatric patients with acute refractory and chronic immune thrombocytopenia, particularly focusing on iron-deficiency anemia. Results: The diagnosis was chronic ITP in 89 patients and acute refractory ITP in 16 patients. The mean age of patients was 9.5±4.5 years (minimum-maximum: 1.2-18 years) at the beginning of EPAG treatment. The overall response rate was 74.3% (n=78). The mean time for platelet count of ≥50x109/L was 11.6±8 weeks (range: 1-34 weeks). The treatment was stopped for 27 patients (25.7%) at an average of 6.8±9 months (range: 1-38 months). The reason for discontinuation was lack of response in 18 patients, nonadherence in 4 patients, and hepatotoxicity in 2 patients. Response to treatment continued for an average of 4 months after cessation of EPAG in 3 patients. Conclusion: Results of the current study imply that EPAG is an effective therapeutic option in pediatric patients with acute refractory and chronic ITP. However, patients must be closely monitored for response and side effects during treatment, and especially for iron deficiency.


Asunto(s)
Anemia Ferropénica/tratamiento farmacológico , Benzoatos/uso terapéutico , Hidrazinas/uso terapéutico , Púrpura Trombocitopénica Idiopática/tratamiento farmacológico , Pirazoles/uso terapéutico , Administración Oral , Adolescente , Anemia Ferropénica/diagnóstico , Benzoatos/administración & dosificación , Benzoatos/efectos adversos , Niño , Preescolar , Femenino , Humanos , Hidrazinas/administración & dosificación , Hidrazinas/efectos adversos , Lactante , Masculino , Púrpura Trombocitopénica Idiopática/diagnóstico , Pirazoles/administración & dosificación , Pirazoles/efectos adversos , Estudios Retrospectivos , Resultado del Tratamiento , Turquía
13.
J Pediatr Hematol Oncol ; 30(8): 608-11, 2008 Aug.
Artículo en Inglés | MEDLINE | ID: mdl-18799939

RESUMEN

Homozygous protein C deficiency affects approximately 1/400,000 to 1/1,000,000 live births. Homozygous protein C deficiency is associated with catastrophic and fatal purpura fulminans-like or thrombotic complications and disseminated intravascular coagulation. In the present patient, genetic study revealed Arg178Trp, a mutation found widely in European population; but this is the first case of homozygous Arg178Trp mutation who suffered from catastrophic purpura fulminans phenotype.


Asunto(s)
Vasculitis por IgA/etiología , Mutación , Deficiencia de Proteína C/genética , Secuencia de Bases , Coagulación Intravascular Diseminada/etiología , Femenino , Homocigoto , Humanos , Recién Nacido , Datos de Secuencia Molecular , Linaje , Reacción en Cadena de la Polimerasa , Deficiencia de Proteína C/complicaciones , Deficiencia de Proteína C/fisiopatología
15.
Indian J Hematol Blood Transfus ; 34(4): 707-710, 2018 Oct.
Artículo en Inglés | MEDLINE | ID: mdl-30369745

RESUMEN

Objective Child patients of chronic thrombocytopenic purpura with severe and resistant thrombocytopenia were evaluated to observe whether their clinical or laboratory states improve by one of the thrombomimetic therapeutic agent called Eltrombopag as in adults in a single center of different country from previous studies. Materials and Methods Nineteen patients with chronic immune thrombocytopenia were treated with Eltrombopag to dose in international guidelines. Results Approximately half (11/19:58%) of the patients benefitted from the treatment with Eltrombopag either by an increase of platelet levels at safe levels with a decrease in the frequency of bleedings which needed rescue treatment. Conclusion Thrombomimetic treatment options have strengthened the clinician's hand where the regular treatment options became insufficient.

16.
Pediatr Hematol Oncol ; 23(5): 433-7, 2006.
Artículo en Inglés | MEDLINE | ID: mdl-16728364

RESUMEN

Mucormycosis is an uncommon, severe, life-threatening fungal infection in the immunocompromised host. Mucormycosis with aplastic anemia is seen rarely. Only a few cases of cardiac mucormycosis with aplastic anemia have been reported in the literature. The authors present a case with severe aplastic anemia that did not respond to classic and immunosuppressive treatment for disease and developing invasive cardiac mucormycosis despite empiric treatment for febrile neutropenia.


Asunto(s)
Anemia Aplásica/complicaciones , Cardiomiopatías/microbiología , Mucormicosis/etiología , Adolescente , Cardiomiopatías/patología , Resultado Fatal , Fiebre , Humanos , Masculino , Mucormicosis/diagnóstico , Mucormicosis/patología , Neutropenia , Infecciones Oportunistas
17.
Mol Clin Oncol ; 4(4): 622-624, 2016 Apr.
Artículo en Inglés | MEDLINE | ID: mdl-27073678

RESUMEN

Following joint hemorrhages, intramuscular hemorrhages are the second most prevalent bleeding pattern in hemophiliac patients. Hematomas of the iliopsoas muscle are a well-known complication of hemophilia; however, obturator muscle hematomas are rare. We herein report a case of spontaneous bleeding of the bilateral external obturator muscles, which occured three times within a period of 9 months in a hemophilia patient with factor VIII inhibitors. To the best of our knowledge, this is the first published case of an obturator externus muscle hematoma in hemophilia. In addition to hip hemarthrosis, iliopsoas hematomas and acute appendicitis, obturator muscle hematoma should be considered as one of the diagnostic alternatives for pelvic pain in hemophiliaψ patients. Magnetic resonance imaging enables rapid diagnosis of obturator muscle hematoma.

18.
Turk J Pediatr ; 44(3): 261-2, 2002.
Artículo en Inglés | MEDLINE | ID: mdl-12405443

RESUMEN

Cardiac complications of iron overload are the most common cause of death in patients with thalassemia major. These complications include recurrent pericarditis, refractory congestive heart failure and rhythm disorders. The usual rhythm disturbances are supraventricular or ventricular premature contractions and first-or second-degree heart block. Complete heart block is a very rare complication of thalassemia major. Herein, we report a case of complete heart block with thalassemia major. The patient also had serious congestive heart failure. Management of the heart block with pacemaker brought no clinical improvement, and she died in the second month of hospitalization.


Asunto(s)
Bloqueo Cardíaco/complicaciones , Talasemia beta/complicaciones , Adolescente , Electrocardiografía , Resultado Fatal , Femenino , Bloqueo Cardíaco/diagnóstico , Insuficiencia Cardíaca/etiología , Hemosiderosis/complicaciones , Humanos
19.
Turk J Pediatr ; 46(1): 32-7, 2004.
Artículo en Inglés | MEDLINE | ID: mdl-15074372

RESUMEN

Anthracycline and cytosine arabinoside are used in combination as the standard therapy for remission induction of acute nonlymphoblastic leukemia. Idarubicin, a synthetic daunorubicin analogue, shows an improved spectrum activity and diminishes acute or chronic toxicity when compared with the other anthracyclines. This study has been carried out in our clinic in order to evaluate the efficiency of the acute nonlymphoblastic leukemia protocol which includes idarubicin. Thirty-eight patients admitted to our Department between 1992-1999 and diagnosed as acute nonlymphoblastic leukemia (ANLL) were included in the study. Their median age was 7 years 6 months (range, 8 months to 14 years). Induction therapy consisted of idarubicin plus cytosine arabinoside and etoposide. Consolidation therapy consisted of two courses, followed by maintenance therapy with thioguanine, cytosine arabinoside, vincristine and cyclophoshamide. The complete remission rate was found to be 71%. The overall survival estimate was found to be 40% for one year and 23% for three years. We established that the protocol with idarubicin reached a higher remission ratio when compared with the other protocols with anthracycline. However, the degree of the hematologic toxicity ratios related to the therapy increased the complication ratios, which affected the long-term life analyses directly. Therefore this protocol may be revised according to socioeconomical conditions, especially in the developing countries.


Asunto(s)
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Leucemia Mieloide Aguda/tratamiento farmacológico , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/administración & dosificación , Examen de la Médula Ósea , Niño , Preescolar , Citarabina/administración & dosificación , Supervivencia sin Enfermedad , Etopósido/administración & dosificación , Femenino , Humanos , Idarrubicina/administración & dosificación , Lactante , Leucemia Mieloide Aguda/patología , Masculino , Metotrexato/administración & dosificación , Estudios Retrospectivos , Resultado del Tratamiento
20.
Curr Ther Res Clin Exp ; 64(7): 461-72, 2003 Jul.
Artículo en Inglés | MEDLINE | ID: mdl-24944396

RESUMEN

BACKGROUND: In vitro studies have shown that nonionic radiographic contrast material may induce the generation of thrombin in blood, whereas ionic contrast agents, such as iohexol, do not. However, knowledge of the effects of contrast material on coagulation and fibrinolytic systems in vivo is limited. OBJECTIVE: This study was designed to assess the effects of the nonionic radiographic contrast material iopamidol on hemostasis in patients undergoing coronary angiography or cardiac catheterization. METHODS: Patients aged ≥18 years with chest pain and/or dyspnea who underwent coronary angiography or cardiac catheterization with intra-arterial contrast material were assessed for hemostasis. Blood samples were drawn before and 3 minutes after injection of iopamidol. Complete blood count and coagulation profile (bleeding time, clotting time, clot retraction time, euglobulin lysis time [ELT], prothrombin and partial thromboplastin times, coagulation factor I [CFI] level, and platelet factor 3 [PF-3] availability) were assessed. The natural coagulation inhibitors protein C, protein S, and antithrombin III (AT-III) also were measured. RESULTS: Thirty patients (7 males, 23 females; mean [SD] age, 51.3 [20.2] years; range, 17-79 years) were included in this single-center study. All hematologic variables (hemoglobin, white blood cell count, and platelet count) decreased significantly (P<0.001, P<0.001, and P<0.05, respectively) after administration of iopamidol but remained within normal limits. Mean levels of protein C, protein S, and AT-III did not change significantly after administration of iopamidol. Bleeding time was not changed significantly, and PF-3 availability was prolonged in both groups, but the changes were not statistically significant. CONCLUSIONS: In this study population, although hemostasis remained grossly intact after injection of nonionic contrast material, the coagulation system may have been affected by the accelerated consumption of CFI and platelets. The affected variables were platelets, clot retraction time, ELT, and natural coagulation inhibitors (protein C, protein S, and AT-III). Although the natural coagulation inhibitors remained within the normal range, the correlations were found significant. These changes in hemostasis affected the vascular phase. If the vascular compartment, especially the endothelium, remained intact, the infusion of nonionic agents in low concentrations might be safe for angiography and other procedures; however, more studies are needed.

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