44th Annual Meeting of the European-Society-for-Blood-and-Marrow-Transplantation (EBMT) -- MAR 18... more 44th Annual Meeting of the European-Society-for-Blood-and-Marrow-Transplantation (EBMT) -- MAR 18-21, 2018 -- Lisbon, PORTUGALWOS: 000487702802045…European Soc Blood & Marrow Transplanta
BackgroundALD is a rare X‐linked peroxisomal metabolic disorder with many distinct phenotypes of ... more BackgroundALD is a rare X‐linked peroxisomal metabolic disorder with many distinct phenotypes of disease that emerge on a wide scale from adrenal insufficiency to fatal cALD which progresses to a vegetative state within a few years. Currently, HSCT is the only treatment method known to stabilize disease progression in patients with cALD. In this study, we aim to report our HSCT experience in patients with cALD and the factors that determine the success of HSCT, as a single‐center experience.MethodsThe study cohort involves 23 boys with cALD and three patients with ALD trait and new‐onset abnormal behavior who underwent allogeneic HSCT between January 2012 and September 2019 in our transplantation center. Loes scoring, NFS, scale and MFD were performed for evaluating the severity of the cerebral disease. The study cohort was divided into two groups according to baseline NFS and Loes score: early‐stage (NFS ≤ 1 and Loes score <9) and advanced stage (NFS > 1 or Loes score ≥9).ResultsThe pretransplant stage of disease impacted both OS and MFD‐free survival. The estimated OS and MFD‐free survival at 3 years in patients with advanced disease were 46.1% (95% CI 19.0–73.2) and 23.1% (95% CI 0.2–46.0), respectively, and all patients with the early disease were alive (p: .004) and MFD‐free (p < .001) at 3 years.ConclusionThis study demonstrated that early HSCT is vital in patients with cALD. The early‐stage disease had a significant survival advantage and free from disease progression after HSCT.
Journal of Pediatric Hematology Oncology, Mar 31, 2021
Background: Total body irradiation (TBI) is the cornerstone of conditioning regimens in pediatric... more Background: Total body irradiation (TBI) is the cornerstone of conditioning regimens in pediatric hematopoietic stem cell transplantation for acute lymphoblastic leukemia. As the late effects and survival comparison between TBI and chemotherapy were well analyzed before, in this study, we aim to focus on the first 100 days and early complications of transplantation. Methods: This retrospective study involves 72 pediatric patients (0 to 18 y) underwent first hematopoietic stem cell transplantation for acute lymphoblastic leukemia between October 2015 and May 2019. Patients are divided into 2 groups regarding conditioning regimens. Conditionings includes either TBI 1200 cGy/6 fractions/3 days and etoposide phosphate or busulfan, fludarabine, and thiotepa. Busulfan was administered IV and according to body weight. Results: The incidences of acute graft versus host disease grade 2 to 4, veno-occlusive disease, capillary leakage syndrome, thrombotic microangiopathy, blood stream infection, hemorrhagic cystitis and posterior reversible encephalopathy syndrome before day 100 were similar for both conditioning regimens; however, patients received TBI-based conditioning had significantly longer neutrophil engraftment time (17.5 vs. 13 d, P=0.001) and tended to have more engraftment syndrome (ES) (45.5% for TBI vs. 24.0% for chemotherapy, P=0.069). Multivariate analysis showed that TBI-based conditioning was associated with a longer neutrophil engraftment time (hazard ratio [HR]=1.20, P=0.006), more cytomegalovirus (CMV) reactivation (HR=3.65, P=0.038) and more ES (HR=3.18, P=0.078). Conclusions: Our findings support chemotherapy-based regimens with early neutrophil engraftment, less ES and CMV reactivation compared with TBI. Although there is no impact on survival rates, increased incidence of ES and CMV reactivation should be considered in TBI-based regimens.
A 13-year-old girl was hospitalized for fever, malaise, intractable diarrhea, and intermittent ga... more A 13-year-old girl was hospitalized for fever, malaise, intractable diarrhea, and intermittent gastrointestinal hemorrhage. Despite aggressive antimicrobial and supportive treatment, she died with massive bleeding from the upper gastrointestinal tract. Autopsy study revealed systemic polyarteritis nodosa of classic form in the right lung and gastrointestinal tract and of microscopic form in kidneys.
Alpha-mannosidosis is a rare lysosomal storage disorder with an autosomal recessive inheritance. ... more Alpha-mannosidosis is a rare lysosomal storage disorder with an autosomal recessive inheritance. Deficient alpha-mannosidase activity leads to lysosomal accumulation of mannose-rich oligosaccharides. The disease characterized by mental retardation, skeletal changes, hearing impairment, and recurrent infections. Stem cell transplantation has been shown to be an effective treatment. It works by providing increased levels of α-mannosidase in the localized extracellular milieu to provide improvements in skeletal malformations, neurocognitive, and sensorineural function. In this case report, we describe a pair of siblings with α-mannosidosis who successfully underwent HSCT from matched unrelated donors. In both siblings, enzyme levels reached to normal limits and improvements in clinical symptoms were recognized early after HSCT. We conclude that HSCT should be considered as a therapeutic approach in patients with alpha-mannosidosis before disease-related complications have developed.
41st Annual Meeting of the European-Society-for-Blood-and-Marrow-Transplantation -- MAR 22-25, 20... more 41st Annual Meeting of the European-Society-for-Blood-and-Marrow-Transplantation -- MAR 22-25, 2015 -- Istanbul, TURKEYWOS: 000351632902206European Soc Blood & Marrow Transplanta
44th Annual Meeting of the European-Society-for-Blood-and-Marrow-Transplantation (EBMT) -- MAR 18... more 44th Annual Meeting of the European-Society-for-Blood-and-Marrow-Transplantation (EBMT) -- MAR 18-21, 2018 -- Lisbon, PORTUGALWOS: 000487702802045…European Soc Blood & Marrow Transplanta
BackgroundALD is a rare X‐linked peroxisomal metabolic disorder with many distinct phenotypes of ... more BackgroundALD is a rare X‐linked peroxisomal metabolic disorder with many distinct phenotypes of disease that emerge on a wide scale from adrenal insufficiency to fatal cALD which progresses to a vegetative state within a few years. Currently, HSCT is the only treatment method known to stabilize disease progression in patients with cALD. In this study, we aim to report our HSCT experience in patients with cALD and the factors that determine the success of HSCT, as a single‐center experience.MethodsThe study cohort involves 23 boys with cALD and three patients with ALD trait and new‐onset abnormal behavior who underwent allogeneic HSCT between January 2012 and September 2019 in our transplantation center. Loes scoring, NFS, scale and MFD were performed for evaluating the severity of the cerebral disease. The study cohort was divided into two groups according to baseline NFS and Loes score: early‐stage (NFS ≤ 1 and Loes score <9) and advanced stage (NFS > 1 or Loes score ≥9).ResultsThe pretransplant stage of disease impacted both OS and MFD‐free survival. The estimated OS and MFD‐free survival at 3 years in patients with advanced disease were 46.1% (95% CI 19.0–73.2) and 23.1% (95% CI 0.2–46.0), respectively, and all patients with the early disease were alive (p: .004) and MFD‐free (p < .001) at 3 years.ConclusionThis study demonstrated that early HSCT is vital in patients with cALD. The early‐stage disease had a significant survival advantage and free from disease progression after HSCT.
Journal of Pediatric Hematology Oncology, Mar 31, 2021
Background: Total body irradiation (TBI) is the cornerstone of conditioning regimens in pediatric... more Background: Total body irradiation (TBI) is the cornerstone of conditioning regimens in pediatric hematopoietic stem cell transplantation for acute lymphoblastic leukemia. As the late effects and survival comparison between TBI and chemotherapy were well analyzed before, in this study, we aim to focus on the first 100 days and early complications of transplantation. Methods: This retrospective study involves 72 pediatric patients (0 to 18 y) underwent first hematopoietic stem cell transplantation for acute lymphoblastic leukemia between October 2015 and May 2019. Patients are divided into 2 groups regarding conditioning regimens. Conditionings includes either TBI 1200 cGy/6 fractions/3 days and etoposide phosphate or busulfan, fludarabine, and thiotepa. Busulfan was administered IV and according to body weight. Results: The incidences of acute graft versus host disease grade 2 to 4, veno-occlusive disease, capillary leakage syndrome, thrombotic microangiopathy, blood stream infection, hemorrhagic cystitis and posterior reversible encephalopathy syndrome before day 100 were similar for both conditioning regimens; however, patients received TBI-based conditioning had significantly longer neutrophil engraftment time (17.5 vs. 13 d, P=0.001) and tended to have more engraftment syndrome (ES) (45.5% for TBI vs. 24.0% for chemotherapy, P=0.069). Multivariate analysis showed that TBI-based conditioning was associated with a longer neutrophil engraftment time (hazard ratio [HR]=1.20, P=0.006), more cytomegalovirus (CMV) reactivation (HR=3.65, P=0.038) and more ES (HR=3.18, P=0.078). Conclusions: Our findings support chemotherapy-based regimens with early neutrophil engraftment, less ES and CMV reactivation compared with TBI. Although there is no impact on survival rates, increased incidence of ES and CMV reactivation should be considered in TBI-based regimens.
A 13-year-old girl was hospitalized for fever, malaise, intractable diarrhea, and intermittent ga... more A 13-year-old girl was hospitalized for fever, malaise, intractable diarrhea, and intermittent gastrointestinal hemorrhage. Despite aggressive antimicrobial and supportive treatment, she died with massive bleeding from the upper gastrointestinal tract. Autopsy study revealed systemic polyarteritis nodosa of classic form in the right lung and gastrointestinal tract and of microscopic form in kidneys.
Alpha-mannosidosis is a rare lysosomal storage disorder with an autosomal recessive inheritance. ... more Alpha-mannosidosis is a rare lysosomal storage disorder with an autosomal recessive inheritance. Deficient alpha-mannosidase activity leads to lysosomal accumulation of mannose-rich oligosaccharides. The disease characterized by mental retardation, skeletal changes, hearing impairment, and recurrent infections. Stem cell transplantation has been shown to be an effective treatment. It works by providing increased levels of α-mannosidase in the localized extracellular milieu to provide improvements in skeletal malformations, neurocognitive, and sensorineural function. In this case report, we describe a pair of siblings with α-mannosidosis who successfully underwent HSCT from matched unrelated donors. In both siblings, enzyme levels reached to normal limits and improvements in clinical symptoms were recognized early after HSCT. We conclude that HSCT should be considered as a therapeutic approach in patients with alpha-mannosidosis before disease-related complications have developed.
41st Annual Meeting of the European-Society-for-Blood-and-Marrow-Transplantation -- MAR 22-25, 20... more 41st Annual Meeting of the European-Society-for-Blood-and-Marrow-Transplantation -- MAR 22-25, 2015 -- Istanbul, TURKEYWOS: 000351632902206European Soc Blood & Marrow Transplanta
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