|1.||Sickle Cell Anemia (Hemoglobin S Disease)
|2.||beta-Thalassemia (Cooley's Anemia)
|4.||Hematologic Neoplasms (Hematological Malignancy)
|1.||Fucharoen, Goonnapa: 20 articles (01/2015 - 01/2002)|
|2.||Fucharoen, Supan: 20 articles (01/2015 - 01/2002)|
|3.||Sanchaisuriya, Kanokwan: 13 articles (01/2013 - 01/2002)|
|4.||Patrinos, George P: 11 articles (07/2014 - 01/2004)|
|5.||Walters, Mark C: 10 articles (09/2014 - 08/2003)|
|6.||Perrine, Susan P: 9 articles (01/2016 - 12/2002)|
|7.||Faller, Douglas V: 9 articles (01/2016 - 12/2002)|
|8.||Harteveld, Cornelis L: 9 articles (12/2015 - 01/2005)|
|9.||Wiwanitkit, Viroj: 9 articles (07/2013 - 07/2004)|
|10.||Chui, David H K: 8 articles (01/2016 - 01/2003)|
04/15/2003 - "Our findings demonstrate the remarkable efficacy of lentivirus-mediated globin gene transfer in treating a fulminant blood disorder and strongly support the efficacy of gene therapy in the severe hemoglobinopathies."
06/01/2015 - "This automated methodology is simple, rapid and cost-effective for the fast determination of human globin chains, which could be an important diagnostic tool in the field of hemoglobinopathies."
01/01/2015 - "High-level fetal (γ) globin expression ameliorates clinical severity of the beta (β) hemoglobinopathies, and safe, orally-bioavailable γ-globin inducing agents would benefit many patients. "
01/01/2015 - "A full understanding of the molecular mechanisms of epigenetic silencing of HbF expression should facilitate the development of more effective treatment of β-globin chain hemoglobinopathies."
05/01/2014 - "Expression of fetal γ-globin in adulthood ameliorates symptoms of β-hemoglobinopathies by compensating for the mutant β-globin. "
|2.||Fetal Hemoglobin (Hemoglobin F)IBA
04/01/2014 - "Increased fetal hemoglobin (HbF) expression is beneficial for β-hemoglobinopathy patients; however, current inducing agents do not possess the ideal combination of efficacy, safety and ease of use. "
09/01/2011 - "An improved understanding of the regulation of the fetal hemoglobin genes holds promise for the development of targeted therapeutic approaches for fetal hemoglobin induction in the β-hemoglobinopathies. "
01/01/2012 - "To address how low titer, variable expression, and gene silencing affect gene therapy vectors for hemoglobinopathies, in a previous study we successfully used the HPFH (hereditary persistence of fetal hemoglobin)-2 enhancer in a series of oncoretroviral vectors. "
01/01/2015 - "Fetal hemoglobin (HbF) is the major modulator of the phenotype of the β hemoglobinopathies. "
01/01/2015 - "Fetal hemoglobin (Hb F) is an important genetic modulator of the beta-hemoglobinopathies. "
06/01/1997 - "Such a technique could be very useful in the hemoglobinopathies, which are common in Italy, and it could be easily adapted to perform comparative studies on animal hemoglobins not far from human species."
08/20/2014 - "The eletrophoretic hemoglobin profiles of the subjects included in this study indicated that no patients presented with hemoglobinopathy. "
07/01/2014 - "A descriptive study was conducted from the Hematology Unit of North Bengal Medical College over 1 year on the subjects underwent screening for hemoglobinopathies for detection of abnormal hemoglobin (Hb) variants by "cation-exchange high-performance liquid chromatography" principle along with other relevant tests. "
01/01/2014 - "The aim of the present study was to estimate the birth prevalence of hemoglobinopathies in newborns of Italian couples and couples of immigrants from endemic areas living in an urban area of northern Italy in order to assess the opportunity of implementing a neonatal screening programme for hemoglobin disorders. "
01/01/2010 - "Of these patients, 382 had some hemoglobin (Hb) abnormality (20.5%), 128 had a sickle cell hemoglobinopathy, representing a general frequency of 6.9%, which is similar to the percentage observed in previous studies on Mexican Mestizos. "
02/01/2003 - "Hematologic parameters such as Hb, hematocrit (Hct), mean cell volume (MCV), membrane-associated denatured Hb, and the oxygen equilibration curve were improved in chimeric hemoglobinopathy mice. "
06/01/1998 - "Hemoglobinopathies affecting maternal-fetal oxygen gradient during pregnancy: molecular, biochemical and clinical studies."
10/01/2015 - "Oxygen transport is altered in hemoglobinopathies. "
07/01/2015 - "Inspired-expired oxygen gap: an alternative method for oxygen saturation monitoring in a patient with an undiagnosed hemoglobinopathy."
10/01/2013 - "Plotting an oxygen dissociation curve is a simple reproducible method to determine when supplemental oxygen is required for a child with a hemoglobinopathy."
01/01/2009 - "Two healthy newborns, heterozygous for two different gamma-globin chain mutations, were observed during an electrophoretic screening for hemoglobinopathies in Sassari, North Sardinia (Italy). "
05/01/2006 - "Pharmacologic reinduction of the developmentally silenced fetal (gamma) globin genes has been achieved in hemoglobinopathy patients using short chain fatty acid derivatives, with therapeutic effects. "
12/15/2002 - "Orally bioactive compounds that induce gamma globin gene expression at tolerable doses are needed for optimal treatment of the beta-hemoglobinopathies. "
04/01/1999 - "Development of viral vectors for gene therapy of beta-chain hemoglobinopathies: optimization of a gamma-globin gene expression cassette."
01/01/1999 - "If there is a reciprocal elevation of gamma-globin expression upon repression, this approach could be useful in additional hemoglobinopathies. "
|6.||sildenafil (Viagra)FDA Link
04/01/2005 - "These data, in a small group of patients, indicate that sildenafil citrate is effective in the treatment of PH in hemoglobinopathies that cannot be treated with alternative oral drugs and is well tolerated long-term at a daily dose of 100 mg, though studies including more patients may uncover toxicities and limitations of efficacy."
01/01/2005 - "These data, described in a small group of patients, indicate that sildenafil citrate is effective in the treatment of PH in hemoglobinopathies and is well tolerated long-term at a daily dose of 100 mg."
02/01/2014 - "Response to "efficacy and safety of sildenafil for the treatment of severe pulmonary hypertension in patients with hemoglobinopathies: results from a long-term follow up " Haematologica 2014;99(2):e17-18."
02/01/2014 - "Efficacy and safety of sildenafil for the treatment of severe pulmonary hypertension in patients with hemoglobinopathies: results from a long-term follow up."
04/01/2005 - "Efficacy and safety of sildenafil in the treatment of severe pulmonary hypertension in patients with hemoglobinopathies."
|7.||DNA (Deoxyribonucleic Acid)IBA
10/01/2012 - "Fortunately, improved knowledge of the human genome and the development of new genomic tools, such as genome-wide genotyping arrays and next-generation DNA sequencers, offer new opportunities to use genetics to better understand the causes of the many complications observed in β-hemoglobinopathy patients. "
01/01/2008 - "For example, based on studies on hemoglobinopathies, it was understood that gene expression can be under the control of DNA elements that locate away from the genes on the linear chromosome template. "
01/01/1996 - "In present study, the rearrangement patterns of the -530 motif in the Chinese population, including 43 patients with various hemoglobinopathies and part of their relatives (34), as well as 20 hematologically normal individuals, were investigated with the method of ds-DNA cycle sequencing. "
01/01/1985 - "The usefulness of DNA gene analyses in family studies of hemoglobinopathy was discussed."
11/01/2015 - "Hemoglobinopathy investigation was performed by HPLC (Bio-Rad VARIANT-II), alkaline and acid electrophoresis (Sebia Hydrasis2), capillary zone electrophoresis (Sebia CAPILLARYS2™) and DNA sequencing. "
09/01/2003 - "The presence of hemoglobinopathy (Hb AS) is also a mark of its originality."
01/01/1997 - "The patient had a second apparently fortuitous hereditary affection: hemoglobinopathy (Hb AS). "
10/01/2014 - "The aim of the study is to examine the relationship between sickle cell trait (Hb AS) and other sickle hemoglobinopathies and the risk of thromboembolism during pregnancy or the puerperium. "
08/01/2001 - "A study of reproductive outcome in Mobile, AL was conducted among a large maternal cohort with sickle-cell disease (Hb SS), sickle-cell trait (Hb AS), and no hemoglobinopathies (Hb AA). "
01/01/1990 - "These studies have left unanswered some questions, however, such as whether antifibrinolytics are as effective in children as adults, in white patients as black, and in patients with normal hemoglobin as well as those with sickle cell hemoglobinopathies. "
|9.||Hydroxyurea (Hydrea)FDA LinkGeneric
04/01/2013 - "In this study we explored the association between genetic variations in MAP3K5 and PDE7B genes, residing on chromosome 6q23, and disease severity in β-hemoglobinopathy patients, as well as the association between these variants with response to hydroxyurea (HU) treatment. "
04/01/2013 - "A total of 24-patients with different Hemoglobinopathies were given hydroxyurea and their response was studied in-vivo and in-vitro on mononuclear cells collected from them simultaneously. "
10/01/2003 - "With the increasing use of hydroxyurea in the management of SS disease or other hemoglobinopathies, the importance of spleen monitoring must be further emphasized in these patients."
01/01/1999 - "Successful hydroxyurea treatment of a patient with SD hemoglobinopathy."
08/01/2014 - "The role of hydroxyurea in the treatment of patients with HbSD-Punjab, a rare hemoglobinopathy with phenotypic expression similar to that of sickle cell anemia is unknown. "
01/01/2006 - "Variable efficacy of recombinant human erythropoietin in anemic pregnant women with different forms of heterozygous hemoglobinopathy."
01/01/2006 - "The aim of this study was to determine the response to recombinant human erythropoietin (rhEPO) in anemic pregnant women with heterozygous hemoglobinopathies. "
04/01/2014 - "Does erythropoietin have a role in the treatment of β-hemoglobinopathies?"
05/01/1997 - "The mechanism of resistance to recombinant human erythropoietin (EPO) in hemodialysis patients with hemoglobinopathy is not yet fully understood. "
02/01/1994 - "For example, newer uses of recombinant human erythropoietin include treatment of anemia of prematurity, AIDS, and some hemoglobinopathies. "
|1.||Stem Cell Transplantation
01/01/2012 - "Advances in stem cell transplantation and gene therapy in the β-hemoglobinopathies."
01/01/2011 - "Advances in allogeneic stem cell transplantation for hemoglobinopathies."
01/01/2011 - "One-third of these cases were performed in combination with HLA typing, allowing the birth of unaffected children who were also HLA identical to the affected siblings with hemoglobinopathies in these families, with successful or pending stem cell transplantation in a dozen of them. "
01/01/2007 - "Stem cell transplantation in hemoglobinopathies."
03/01/2005 - "This review focuses on the current status of stem cell transplantation for hemoglobinopathies."
|2.||Transplantation (Transplant Recipients)
01/01/2011 - "Established treatments like bone marrow or cord blood transplantation are so far the only real cure for a limited number of patients with severe hemoglobinopathies. "
10/01/2015 - "In this commentary, we review the progress of UCB banking technology; we also analyze the current data on pediatric and adult unrelated UCB, including the recent expansion of interest in transplantation for hemoglobinopathies, and discuss emerging studies on the use of autologous UCB for neurologic diseases and regenerative medicine. "
01/01/2011 - "However, since in most of the studies reported in literature the engraftment state was observed in the nucleated cells, in this study we determined the donor origin of the mature erythrocytes of patients with persistent mixed chimerism after transplantation for hemoglobinopathies. "
07/01/2012 - "This case highlights the dilemma surrounding second hematopoietic stem cell transplantations in hemoglobinopathies and the need for a careful, well informed, and collaborative decision-making process by patients, families, and medical professionals."
09/01/2011 - "These results suggest that only CB units containing an expected infused cell dose >5 × 10(7)/kg should be considered for transplantation for hemoglobinopathy."
|3.||Hematopoietic Stem Cell Transplantation
01/01/2010 - "Allogeneic hematopoietic stem cell transplantation (HSCT) is the only potential cure for most bone marrow (BM) failure syndromes and hemoglobinopathies. "
02/01/2003 - "Allogeneic hematopoietic stem cell transplantation is the only cure for patients with hemoglobinopathies. "
09/01/2014 - "Combined umbilical cord blood and bone marrow from HLA-identical sibling donors for hematopoietic stem cell transplantation in children with hemoglobinopathies."
04/01/2014 - "Allogeneic hematopoietic stem cell transplantation for patients with a hemoglobinopathy can be curative but is limited by donor availability. "
04/01/2014 - "Unrelated donor allogeneic hematopoietic stem cell transplantation for patients with hemoglobinopathies using a reduced-intensity conditioning regimen and third-party mesenchymal stromal cells."
09/03/2015 - "Correction of murine hemoglobinopathies by prenatal tolerance induction and postnatal nonmyeloablative allogeneic BM transplants."
02/01/2015 - "This has led to the utilization of marrow, cord, peripheral blood stem cell and haploidentical stem cell sources, and novel reduced toxicity or reduced intensity conditioning regimens to transplant non-malignant disorders such as immune dysfunctions, marrow failure syndromes, metabolic disorders and hemoglobinopathies. "
07/01/2014 - "Autologous stem cell transplants in patients with hemoglobinopathies are limited. "
08/01/2011 - "Recurrent formation of intrahepatic stones after liver transplant is a rare and severe complication in patients with hemoglobinopathies. "
12/01/2010 - "GSTA1, GSTP1, and GSTM1 genotyping prior to HSCT in children with congenital hemoglobinopathies may allow better prediction of oral BU kinetics and the need for BU dose adjustment, as well as prediction of transplant related toxicity such as GVHD, thereby improving clinical outcome."
08/01/1998 - "Partial splenectomy is a safe and effective procedure in children less than 4 years of age with hemoglobinopathies. "
10/08/2015 - "Our data suggest the increased thrombotic risk associated with splenectomy, and patients with hemoglobinopathies is a possible consequence of increased levels of circulating mature reticulocytes expressing inside-out PS-exposed autophagic vesicles because of asplenia. "
07/01/1999 - "In the Eastern Province of Saudi Arabia, an area known for various hemoglobinopathies, splenectomy is performed rather frequently. "
08/01/1998 - "Partial splenectomy is an alternative in patients with hemoglobinopathies. "
08/01/1998 - "Partial splenectomy in children under 4 years of age with hemoglobinopathy."