Active clinical trials for "Hemolysis"

Treatment for patients with autoimmune destruction of blood cells is poor. The part of the body that fights infections is called the immune system and white blood cells (WBCs) are part of the immune system. Normally, a person's body creates WBCs to fight infections and eliminates WBCs which have stopped helping the body function. Patients with autoimmune destruction of blood cells have difficulty eliminating old WBCs. The abnormal WBCs build up and can damage other healthy cells, which can lead to anemia, fatigue, jaundice, internal bleeding, infection, and cancer. Few effective medications exist for treatment for patients with autoimmune cytopenias and those commonly used are fraught with side effects. Nevertheless, as scientific understanding of autoimmune diseases has improved, more directed and less toxic therapies are becoming available. A number of groups have been studying the efficacy of a medication called sirolimus in patients with autoimmune diseases. This medicine has been FDA-approved for over 20 years. Sirolimus is a medicine used in children with other diseases. Sirolimus works, in part, by eliminating old and abnormal WBCs. Our group and others have shown that sirolimus is effective in mice with autoimmunity and in children with a rare condition called Autoimmune Lymphoproliferative Syndrome (ALPS). We believe sirolimus will help children with autoimmune cytopenias. We believe it will improve their symptoms and make them less sick. We propose to study sirolimus in children with chronic and/or refractory autoimmune cytopenias.

The record Primary purpose is to assess the efficacy of eculizumab in adult patients with Atypical Hemolytic- Uremic Syndrome (aHUS) to control Thrombotic Microangiopathy (TMA) as characterized by thrombocytopenia, hemolysis and renal impairment.

Hematopoietic stem cell transplantation (HCT)-associated thrombotic microangiopathy (TMA) is an understudied complication of HCT that significantly affects transplant related morbidity and mortality. The investigators hypothesize that early intervention with complement blocker eculizumab will double survival in HCT recipients with high risk TMA, as compared to historical untreated controls. An optimal eculizumab dosing schedule can be determined for this population through eculizumab pharmacokinetic/pharmacodynamic (PK/PD) testing.

The primary objective of this study is to assess the efficacy of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA).

The purpose of the study is to assess the safety and efficacy of ravulizumab to control disease activity in adolescent and adult participants with aHUS who had not previously used a complement inhibitor.

Hemolytic Uremic Syndrome (HUS) is a foodborne disease which mainly affects children. It is caused by Escherichia coli bacteria, which release a toxin called Shiga toxin within the body. This infectious form of HUS, defined as STEC-HUS, can cause sporadic cases or outbreaks, as observed in different countries. Argentina has the highest incidence of STEC-HUS worldwide. The disease is endemic, representing approximately 95% of all HUS cases nationwide. STEC-HUS generally begins with diarrhea (with or without blood), and can also cause fever, abdominal pain, and cramps. Then the child may have pallor, altered consciousness, decreased urine output, seizures, and other symptoms. Although death is uncommon (it occurs in 2-4% of cases), it is a very serious disease that mainly affects the kidneys, and also other organs such as the brain. About half of children need to undergo a risky procedure such as dialysis (due to malfunctioning kidneys); and most of them also receive blood transfusions. Around 30% of the patients are left with lifelong consequences that can range from permanent kidney damage to the need for a transplant. So far there is no drug, antibiotic or vaccine to prevent or treat HUS. Current treatment protocols include hospitalization for all patients with HUS, and supportive therapy such as hydration and salt intake. Support therapy is not a specific treatment, but rather helps the body better defend itself against the disease. The purpose of this study is to establish whether it is safe and effective to treat patients who are diagnosed with STEC-HUS, with INM004 (study drug). INM004 is an investigational product "Fraction F(ab')2 of Equine Shiga Antitoxin Immunoglobulin". It is a concentrated and sterile serum obtained from healthy horses immunized against Shiga toxin that contains antibodies capable of neutralizing it. The initial hypothesis is that INM004 would neutralize the entry of Shiga toxin into the body's cells thus preventing the consequent toxic damage. With the proposed treatment, INM004 would eliminate the Shiga toxin, preventing the progression of HUS symptoms and its serious complications (such as the need for and duration of dialysis, duration of hospital stays, as well as neurological, cardiovascular, intestinal complications, among others) which are associated with high morbidity and mortality. This treatment could then have an impact in health costs of STEC-HUS as well as the social costs.

Prospective, non-randomized multicenter study on the safety and efficacy of combination therapy with bendamustine and rituximab for chronic cold agglutinin disease.

It is well established that hemolytic diseases predispose patients to the development of pigment gallstones. Gallstones are noted in at least 5% of children under the age of 10 years, increasing to 40-50% in the second to fifth decades. The co-inheritance of Gilbert's syndrome increases the risk of cholelithiasis four to five-fold. In patients with chronic hemolysis, total bile lipid concentration is decreased and the total bilirubin to total lipid ratio is increased. This suggests that the conjugating capacity of hepatocytes is surpassed by the excessive amount of bilirubin resulting from hemolysis. Increased bilirubin monoconjugate and unconjugated bilirubin can precipitate in bile and form complexes with inorganic ions, mostly calcium, and develop into stones. Patients with hemolytic disorders can also develop biliary sludge, a suspension of precipitated particulate matter in bile dispersed in a viscous, mucin-rich liquid phase . The chemical composition of the precipitates correlates well with the composition of the associated stone and sludge often stands as a harbinger of future stone development. There is strong data suggesting a benefit in treating cholelithiasis with UDCA and also in preventing gallstone development in various high risk scenarios. There are several proposed mechanisms for the positive effect of UDCA in primary prevention of pigment stones. Mucoglycoproteins are present in significant amounts in black pigment stones and contribute to the matrix of gallstones. UDCA suppresses the secretion of protein and decreases the levels of various proteins in bile . It has also been suggested that increased colonic bile salt may solubilize unconjugated bilirubin and may prevent calcium complexing. There is no published data at present on the role of UDCA in prevention and treatment of cholelithiasis in hemolytic diseases. The investigators hypothesise that UDCA can be of benefit to patients with hemolytic disorders in the primary prevention of pigment stones, possible resolution of biliary sludge and existent stones, and reduction of symptomatic episodes of cholelithiasis.

Protocol is intended to characterize the overall safety and tolerability of eculizumab in this population.

This protocol is designed to collect safety and efficacy data on patients that have been or will be treated with eculizumab for STEC-HUS, in the context of the 2011 STEC-HUS epidemic in Germany.