Stem Cell Transplantation (SCT) for Genetic Diseases
ThrombocytopeniaMetachromatic Leukodystrophy4 moreOBJECTIVES: I. Ascertain whether stem cell transplantation (SCT) is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism. II. Determine whether clinical manifestations of the specific disease may be arrested or reversed by this treatment.
Combination Chemotherapy With or Without Bone Marrow Transplantation in Treating Children With Acute...
Childhood Acute Erythroleukemia (M6)Childhood Acute Megakaryocytic Leukemia (M7)14 moreRandomized phase III trial to compare the effectiveness of different chemotherapy regimens with or without bone marrow transplantation in treating children who have acute myelogenous leukemia or myelodysplastic syndrome. Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with bone marrow transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. It is not yet known which treatment regimen is more effective for acute myelogenous leukemia or myelodysplastic syndrome
Daily vs Alternate Day Iron Supplementation for Pregnant Women With Iron Deficiency Anemia
Iron Deficiency AnemiaIron deficiency is thought to be the most common nutrient deficiency among pregnant women and the most common cause of anemia in pregnancy. The consequences of iron deficiency anemia are serious and can include diminished intellectual and productive capacity and possibly increased susceptibility to infection in mothers and infants, low birthweight, and premature births, hence the importance of appropriate treatment during pregnancy. Most guidelines recommend an increase in iron consumption by about 15-30 mg/day, an amount readily met by most prenatal vitamin formulations. This is adequate supplementation for non-anemic and non-iron-deficient women. However, women with iron deficiency anemia should receive an additional 30-20 mg/day until the anemia is corrected. It is not clear whether intermittent administration of oral iron is equivalent to once daily to rise the hemoglobin levels in pregnant women with iron deficiency anemia. Alternate day treatment with supplemental iron has been suggested as a way to improve its absorption because daily doses may suppress the mucosal uptake of iron even in the presence of iron deficiency until the intestinal mucosa completes its turnover. Another possible factor associated with the frequency of iron dosing is related to hepcidin, the central regulatory molecule in the metabolism of iron in mammals. The synthesis of hepcidin is controlled by 3 kinds of signals: inflammation; the need for increased erythropoiesis, and an iron status signal based on plasma iron levels and iron stores. If plasma iron levels or iron stores are increased, the resulting signal increases hepcidin levels, thereby blocking iron absorption and its release from stores (liver, macrophages) and preventing iron overload.This hepcidin effect, suppressing iron absorption, could last as long as 48h. In this study, the researchers aim to determine if alternate day dosing of iron in pregnant women with iron deficiency anemia results in improved levels of hemoglobin or hematocrit and ferritin. Hepcidin levels will be compared between patients on daily iron supplementation versus alternate day supplementation. The researchers will also evaluate if alternate day dosing of iron supplementation results in a better side effect profile, and with better patient compliance.
Combination of Thrombopoietin Mimetic and Immunosuppressive Therapy in Aplastic Anaemia
Aplastic AnemiaTo the investigator's Knowledge this is the first study that will assess Treatment with thrombopoietin Mimetic plus immunosuppressiveTherapy in Egyptian Patients with Aplastic Anaemia. Aim of the work : To evaluate the efficacy, tolerability and toxicity of the combination of thrombopoietin mimetic and immunosuppressive therapy in Egyptian patients with AA. To study the influence of this combination on patients' quality of life. To access evolution to paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndrome , acute leukemia or development of fibrosis
A Study to Assess the Safety and Pharmacokinetics of HBI-002, an Oral Carbon Monoxide Therapeutic,...
AnemiaSickle CellThis is a single center, open label Phase 1 clinical trial in normal adult subjects to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with single ascending doses (SAD), followed by multiple dose with doses daily for 7 days.
Intravenous Iron Carboxymaltose, Isomaltoside and Oral Iron Sulphate for Postpartum Anemia
Postpartum Anemia NosIron-deficiencyAnemia affects between 20 and 50 % of women in the postpartum period. It is associated with several adverse health consequences, such as impaired physical work capacity, deficits in cognitive function and mood, reduced immune function and reduced duration of breastfeeding. Postpartum anemia has also been shown to be a major risk factor for postpartum depression and to significantly disrupt maternal-infant interactions. Iron deficiency is the principal cause of anemia after delivery. Oral iron supplementation with ferrous sulfate has been considered the standard of care with blood transfusion reserved for more severe or symptomatic cases. In the last decade, two new intravenous iron compounds have been registered for clinical use: ferric carboxymaltose (Iroprem®) and iron isomaltoside (Monofer®). No study to date compared efficacy of iron carboxymaltose to iron isomaltoside for treatment of postpartum anemia. The objective of the study is to compare efficacy of intravenous iron carboxymaltose to intravenous iron isomaltoside and oral iron sulphate for treatment of postpartum anemia.
Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects...
β-thalassemiaIneffective Erythropoiesis1 moreThis is a Phase 2 open-label, single-arm design study with dose escalation by subject cohort . The study is designed to monitor the PTG-300 safety profile, to obtain preliminary evidence of efficacy of PTG-300 for the treatment in β-thalassemia.
Daily vs. Every Other Day Oral Iron Supplementation in Patients With Absolute Iron Deficiency Anemia...
AnemiaIron Deficiency AnemiaIron deficiency anemia is a global health problem and the most common cause of anemia worldwide. Patients with iron deficiency (ID) and IDA can present with a multitude of symptoms including fatigue, restless legs syndrome and pica.Oral iron supplementation is associated with increasing hemoglobin in multiple studies in women, pregnant women and elderly patients.However, the optimal dose and frequency of oral iron supplementation for treatment remains unclear. The current proposed study attempts to address this gap in the literature.
Efficacy of Iron Bisglycinate in Treatment of Iron Deficiency Anemia in Pregnant Women
Iron Deficiency Anemia of PregnancyAnemia in pregnancy is defined by the World Health Organization as hemoglobin levels of ≤ 11 g/dl. Globally, a prevalence rate of 38% was estimated by the World Health Organization for pregnant women.Treatment of iron deficiency anemia during pregnancy remains a main public health issue. Oral iron salts have been recommended for treatment of iron deficiency anemia e.g. ferrous fumarate. Increasing the dose of ferrous fumarate will subsequently increase the bioavailability of iron preparation, however it also increases the frequency of gastrointestinal tract side effects e.g. nausea, constipation, diarrhea, flatulence, and black stained stools. Besides, the increased bioavailable ferrous fumarate may decrease by many foods and / or chelating drugs in the gastrointestinal tract which interfere with its absorption leading to variability in the hemoglobin correction during the treatment. Ferrous bisglycinate is an iron amino acid chelate. It is formed by reaction of ferrous iron with two molecules of the amino acid glycine by a covalent bound in a process called chelation. Ferrous bisglycinate is claimed to have better patient compliance because of fewer gastrointestinal tract side effects. It is also claimed that ferrous bisglycinate improves iron absorption, storage and increase hemoglobin level better than the conventionally used iron salts.
Lactoferrin Versus Ferrous Sulphate for Treatment of Iron Deficiency Anaemia During Pregnancy
Iron Deficiency Anemia of PregnancyThis study evaluates the efficacy and tolerability of lactoferrin in contrast to ferrous sulphate in the context of iron deficiency anemia with pregnancy. Half of participants will receive lactoferrin, while the other half will receive ferrous sulphate.