Clinical trials for
Translating trial titles and descriptions to plain English...
A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.
The purpose of this research study is to look at genes and determine how they interact with each other to find changes that could explain why some people's immune systems may respond to blood transfusions. This response is called an alloimmune response. We strongly believe that when someone has an alloimmune response, it is caused by changes in their genes. We plan to compare changes in the genes of individuals that develop red blood cell alloimmunization after blood transfusions with those that do not develop alloimmunization. This may help us to create more targeted therapeutic interventions, which may improve the health of alloimmune responders.
The goal of this clinical research study is to find out about the safety and effects of a drug called panobinostat when given to adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor. HDAC inhibitors have been shown to significantly increase hemoglobin F induction, which is well documented to improve outcomes in sickle cell disease. HDAC inhibitors are also known to potently inhibit cell-specific inflammation, which is a primary contributor to the debilitating effects of sickle cell disease. Given the relevance of these mechanisms of action in SCD, panobinostat may prove to contribute significantly to the management of SCD patients, a population in critical need of further effective treatment options.
Background: Some people with the same disorder on a genetic level have more complications than others. Researchers want to look for a link between the PKLR gene and sickle cell disease (SCD) symptoms. The PKLR gene helps create a protein, called pyruvate kinase that is essential in normal functioning of the red blood cell. Differences in the PKLR gene, called genetic variants, may cause some changes in the pyruvate kinase protein and other proteins, that can affect functioning of the red blood cell adding to the effect of SCD. Researchers can study these differences by looking at DNA (the material that determines inherited characteristics). Objective: To study how the PKLR gene affects sickle cell disease. Eligibility: Adults ages 18-80 of African descent. They may have sickle cell disease or not. They must not have had a transfusion recently or have a known deficiency of pyruvate kinase. They cannot be pregnant. Design: Participants will be screened with questions. Participants will have blood drawn by needle in an arm vein. The blood will be genetically tested. Not much is known about how genes affect SCD, so the test results will not be shared with participants or their doctors. ...
Background The pediatric-adult care transition is a risk-disrupting time for patients with chronic disease. This care transition takes place during adolescence; a period of psychological upheavals and adaptations of family roles. During this period, medication adherence is non-optimal and absenteeism at medical appointments is high. Sickle cell disease (SCD) is the first genetic disease detected in France. It is chronic disease characterized by frequent painful vaso-occlusive crises (VOC) requiring emergency hospitalization when they are severe. Other serious complications are acute chest syndromes (ACS) and stroke. In order to improve the health status of teenagers with sickle cell disease, it is necessary to anticipate this care transition and to involve the pediatric and adult sectors. The biopsychosocial health approach and the Social-Ecological Model of Adolescent and Young Adult Readiness to Transition (SMART) describe a care transition integrating bioclinical and psychosocial factors such as integration of the patient's family, education on disease and therapeutics, psychological management of pain and medico-social orientation. The pediatric-adult transition program proposed is based on this biopsychosocial approach. It aims to improve the health status of adolescents with SCD, their quality of life and the use of health care service. Objective of the study To assess the impact of a pediatric-adult transition program on the incidence of sickle-cell-related complications leading to hospitalization on 24-months after transfer to the adult sector. The evaluation focuses on severe complications leading to hospitalization, such as VOC, ACS, and stroke. Study design Multicenter Open-label individual Randomized Controlled Trial Population : Patients aged at least 16 years old with sickle cell disease, and their parents (or legal representatives Number of subject : 196 patients (98 patients by arm) The study will last 24 months Expected results For patients and families Better health and quality of life for patients is expected, including better use of medical care after the transition program. It is also expected a better experience of the pediatric-adult care transition and indirectly a better experience of intrafamilial relations. For health professionals This project is expected to provide solutions to improve the pediatric-adult care transition of patients with chronic disease. Indeed, the methodological quality of the study will make it possible to evaluate the efficiency of the proposed program, to possibly adapt it and test it to other chronic diseases presenting the same care transition problematic. In terms of public health SCD mainly affects populations of sub-Saharan origin, with low visibility and high social vulnerability. By focusing on this population, this project will reduce the social inequalities in health, experienced by patients with SCD and their families. By improving the health, quality of life and care of patients with SCD, this project is expected to decrease the cost of the pediatric-adult care transition period.
There is limited information on what causes injury to the heart in individuals with Sickle Cell Disease (SCD). Researchers in this study want to see if decreased blood flow to the heart during stress could be causing the heart damage seen in SCD patients. They also want to test people who don't have SCD to see if their hearts react the same way under stress. Primary Objective * To estimate the coronary flow reserve (CFR) (also referred to as myocardial perfusion reserve), as measured by PET stress-rest myocardial perfusion imaging, in SCD patients with and without diastolic dysfunction, and healthy controls. Secondary Objectives * To investigate the relationship between decreased CFR (quantified with PET stress- rest myocardial perfusion imaging) and presence of abnormal diastolic parameters
This study aims to evaluate the use of virtual reality as an adjunct to standard care for patients with sickle cell disease experiencing vaso-occlusive crises.
HSCT is currently the only curative option for SCD but less than 20% of SCD patients have a MD donor available. So far, all curative approaches beyond a MSD HSCT at young age are non-satisfactory. With the lack of a suitable donor for the vast majority of patients, the major question of this trial is, if a haploidentical αß/CD19+ T-cell depleted HSCT can be a valid alternative to a MSD HSCT. The main challenge in non-malignant diseases is to offer a safe and GvHD-free HSCT without rejection.
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.
The goal of this study is to evaluate the effects of transauricular vagus and trigeminal nerve stimulation on the treatment of pain in children with Sickle Cell Disease. * The primary objective is to determine the feasibility of using a tAN device to treat pain in pediatric participants with SCD by assessing the completion rate, stimulation tolerability, and acceptability of the intervention measures. * The secondary objective is to assess the preliminary efficacy of using a tAN device as a remedy for pain in pediatric SCD subjects by analyzing inflammatory serum biomarker levels, the Functional Status Scale (FSS), the Wong-Baker FACES questionnaire, tracking analgesic consumption, and other assessments.
Brief Summary: \* A short description of the clinical study, including a brief statement of the clinical study's hypothesis, written in language intended for the lay public. Limit: 5000 characters. Severe forms of sickle cell syndrome are characterized by the occurrence of repeated vaso-occlusive crises (CVO), early complications and a high morbidity and mortality in these patients. Intensified management is then required, with the introduction of hydroxyurea treatment and then, if it proves ineffective, a transfusion program or even a haematopoietic stem cell allograft. These latter treatments present significant risks of adverse effects for the patient (haemochromatosis, erythrocyte alloimmunisation for the transfusion program, risk of GVH, chemotherapy-related toxicity, MVO for the allograft). Hydroxyurea (HU) is the first treatment based on the specific pathophysiology of sickle cell disease. It is the first line of therapeutic intensification for adult patients and children (age ≥ 2 years) with major sickle cell disease. By mainly increasing the percentage of fetal haemoglobin (HbF), HU decreases the frequency of CVO, complications, hospitalizations and prolongs the life expectancy of patients. The initial dose of HU, recommended by the ANSM, is 15 mg/kg/d once daily. However, the optimal dose cannot be predicted at the start of treatment, which is why a dosage adjustment is essential. The usual dose is between 15 and 35 mg/kg per day. Typically, the dose is increased every 3 months until a mild myelosuppression tolerated by the patient is reached, indicating that the maximum tolerated dose (MTD) has been reached. When the dose of HU has reached the MTD, the ratio of clinical (reduced frequency of vaso-occlusive attacks) and biological (better % of HbF) benefits to risk (toxicity) is optimal for the patient. The disadvantages of this practice are that: * dose escalation can be long (9-12 months) * clinicians may be reluctant to escalate HU to MTD * patients are treated sub-optimally during the therapeutic adaptation period. Recent work has shown that it is beneficial for the patient to adjust the initial dose using a pharmacological therapeutic approach in addition to monitoring haematological tolerance. Thus, by customizing the dose of HU using an area under the curve (AUC) measurement at the initial intake of HU at a standardized dose (20 mg/kg/day), the MTD would be achieved in a faster time frame of 6-9 months. The primary objective of our trial is to identify the methodology that will most effectively decrease the time to reach the MTD (therapeutic target). The immediate benefit will be a reduction in CVO which is the major clinical problem and leads to a risk of complications in sickle cell disease.
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
Short description of the protocol intended for the lay public. Include a brief statement of the study hypothesis (Limit : 5000 characters) The sickle cells anemia is a monogenic disease linked to the presence of Hemoglobin S due to a mutation in the Hemoglobin Beta chain. The lack of circulating oxygen induces a polymerization of the Hemoglobin S which change the red cell conformation into sickle. Those cells interact and causes vaso-occlusive crisis (CVO). The MEOPA is a medical gas used as an antalgic and a sedative especially in sickle cells disease patients. The nitrous oxide, oxide the cobalt ion in the vitamin B12 which inactivate it irreversibly creating a functional deficiency. During the metabolism of vitamin B12, homocysteine is transformed in methionine which is used in to form the myelin sheath and helped in producing DNA. Numerous studies already shown that the longer the exposition to MEOPA is the greater the functional deficiency of vitamin B12 occur. A few studies shown a symptomatic deficiency of vitamin B12 due to the exposition of MEOPA in sickle cells patient but there is no explanation on the necessary amount of exposure or if some patients are more at risk. When there is a deficiency of vitamin B12 the symptoms can go from a simple orthostatic hypotension to a combined spinal sclerosis. The participation to the study will be proposed to every patient hospitalized for a CVO in the follow up of the emergency room visit or directly in pediatric reanimation. During a usual blood test, a small amount of blood (4mL) will be collected in addition to dose the Vitamin B12, the vitamin B9, the homocysteine, and the methionine. A small amount of urine will also be collected to dose the methylmalonic acid, all those elements are a part of the metabolism of B12 vitamin. The same sample will be taken on the day of departure of the hospital. During the hospitalization the pain management, a daily neurological exam, and the exposition to the MEOPA will be assessed meticulously. An appointment will take place at 7 days and at one month after the hospital departure to evaluate the possible neurological defect. Each patient can only be included once.
Feasibility and reliability of ultrasound in the inpatient hematology setting.
The purpose of this research is to determine whether a new stress management and mindfulness program can improve quality of life in individuals with sickle cell disease. The program is based on a number of empirically supported treatments including techniques from cognitive-behavioral therapy like overcoming motivational barriers to self-care, learning to see things as accurately as possible, practicing assertive communication, relaxation exercises like deep slow breathing, mindfulness meditation, and other stress management strategies.
The goal of this randomized control clinical trial is to learn if virtual reality can be used to treat sickle cell pain in children. The main questions it aims to answer are: Does virtual reality reduce pain severity during a child's hospital stay for a vaso-occlusive pain crisis? Does virtual reality decrease the daily use of opiates? Researchers will compare standard therapy to the use of standard therapy plus a daily virtual reality experience to see if virtual reality works to treat sickle cell pain. All patients will: \- Be asked to fill out a pain assessment survey three times daily for up to 3 days If randomized to intervention arm, patients will: * Participate in an immersive virtual reality experience once daily for up to 3 days * Fill out a survey twice daily to monitor for side effects from virtual reality experience * Fill out a satisfaction survey once during the study period
In a hybrid type I effectiveness-implementation trial, our three-center research teams aim to examine whether empowering adults with sickle cell disease (SCD) with patient-facing SCD-specific guidelines through an mHealth application with booklets will decrease acute healthcare utilization and be cost-effective over booklets with the guidelines alone. Our team, head will test our hypotheses with the following aims: Aim 1: evaluate the effectiveness of the patient-facing guidelines mHealth app + booklet intervention to decrease acute healthcare utilization (hospitalizations, emergency room visits, and day hospital visits) in adults with SCD over the standard care in a randomized controlled trial, Aim 2: evaluate the implementation outcomes of the mHealth app + booklet using the capability, opportunity, and motivation-behavior (COM-B) and reach, effectiveness, adoption, implementation, and maintenance (RE-AIM) frameworks, and Aim 3: evaluate the cost-effectiveness of patient-facing mHealth app + booklets vs. standard care in adults with SCD. is hybrid effectiveness-implementation trial design, according to the COM-B and RE-AIM frameworks with a mixed-methods approach, will give valuable insights into the effects, facilitators, and barriers to the implementation that will influence the effects of the patient-facing guidelines intervention.
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 injection in treating sickle cell disease.
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft failure and to aid in immune reconstitution following hematopoietic stem cell transplantation.
Sickle cell disease is a chronic disease characterized by multiple vaso-occlusive complications. The basic treatment for patients with a vaso-occlusive crisis (VOC) is based on adequate hydration, oxygen therapy and pain control. Loco Regional Anesthesia (LRA) is one of the major treatments in resuscitation anesthesia for both anesthesia and analgesia. LRA allows effective and almost immediate pain control by blocking nerve afferents in a given area. LRA could decrease the inflammatory response during crises and accelerate resolution of the crisis.
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Trials actively recruiting for Sickle Cells Patients
Translating trial titles and descriptions to plain English...
A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.
The purpose of this research study is to look at genes and determine how they interact with each other to find changes that could explain why some people's immune systems may respond to blood transfusions. This response is called an alloimmune response. We strongly believe that when someone has an alloimmune response, it is caused by changes in their genes. We plan to compare changes in the genes of individuals that develop red blood cell alloimmunization after blood transfusions with those that do not develop alloimmunization. This may help us to create more targeted therapeutic interventions, which may improve the health of alloimmune responders.
The goal of this clinical research study is to find out about the safety and effects of a drug called panobinostat when given to adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor. HDAC inhibitors have been shown to significantly increase hemoglobin F induction, which is well documented to improve outcomes in sickle cell disease. HDAC inhibitors are also known to potently inhibit cell-specific inflammation, which is a primary contributor to the debilitating effects of sickle cell disease. Given the relevance of these mechanisms of action in SCD, panobinostat may prove to contribute significantly to the management of SCD patients, a population in critical need of further effective treatment options.
Background: Some people with the same disorder on a genetic level have more complications than others. Researchers want to look for a link between the PKLR gene and sickle cell disease (SCD) symptoms. The PKLR gene helps create a protein, called pyruvate kinase that is essential in normal functioning of the red blood cell. Differences in the PKLR gene, called genetic variants, may cause some changes in the pyruvate kinase protein and other proteins, that can affect functioning of the red blood cell adding to the effect of SCD. Researchers can study these differences by looking at DNA (the material that determines inherited characteristics). Objective: To study how the PKLR gene affects sickle cell disease. Eligibility: Adults ages 18-80 of African descent. They may have sickle cell disease or not. They must not have had a transfusion recently or have a known deficiency of pyruvate kinase. They cannot be pregnant. Design: Participants will be screened with questions. Participants will have blood drawn by needle in an arm vein. The blood will be genetically tested. Not much is known about how genes affect SCD, so the test results will not be shared with participants or their doctors. ...
Background The pediatric-adult care transition is a risk-disrupting time for patients with chronic disease. This care transition takes place during adolescence; a period of psychological upheavals and adaptations of family roles. During this period, medication adherence is non-optimal and absenteeism at medical appointments is high. Sickle cell disease (SCD) is the first genetic disease detected in France. It is chronic disease characterized by frequent painful vaso-occlusive crises (VOC) requiring emergency hospitalization when they are severe. Other serious complications are acute chest syndromes (ACS) and stroke. In order to improve the health status of teenagers with sickle cell disease, it is necessary to anticipate this care transition and to involve the pediatric and adult sectors. The biopsychosocial health approach and the Social-Ecological Model of Adolescent and Young Adult Readiness to Transition (SMART) describe a care transition integrating bioclinical and psychosocial factors such as integration of the patient's family, education on disease and therapeutics, psychological management of pain and medico-social orientation. The pediatric-adult transition program proposed is based on this biopsychosocial approach. It aims to improve the health status of adolescents with SCD, their quality of life and the use of health care service. Objective of the study To assess the impact of a pediatric-adult transition program on the incidence of sickle-cell-related complications leading to hospitalization on 24-months after transfer to the adult sector. The evaluation focuses on severe complications leading to hospitalization, such as VOC, ACS, and stroke. Study design Multicenter Open-label individual Randomized Controlled Trial Population : Patients aged at least 16 years old with sickle cell disease, and their parents (or legal representatives Number of subject : 196 patients (98 patients by arm) The study will last 24 months Expected results For patients and families Better health and quality of life for patients is expected, including better use of medical care after the transition program. It is also expected a better experience of the pediatric-adult care transition and indirectly a better experience of intrafamilial relations. For health professionals This project is expected to provide solutions to improve the pediatric-adult care transition of patients with chronic disease. Indeed, the methodological quality of the study will make it possible to evaluate the efficiency of the proposed program, to possibly adapt it and test it to other chronic diseases presenting the same care transition problematic. In terms of public health SCD mainly affects populations of sub-Saharan origin, with low visibility and high social vulnerability. By focusing on this population, this project will reduce the social inequalities in health, experienced by patients with SCD and their families. By improving the health, quality of life and care of patients with SCD, this project is expected to decrease the cost of the pediatric-adult care transition period.
There is limited information on what causes injury to the heart in individuals with Sickle Cell Disease (SCD). Researchers in this study want to see if decreased blood flow to the heart during stress could be causing the heart damage seen in SCD patients. They also want to test people who don't have SCD to see if their hearts react the same way under stress. Primary Objective * To estimate the coronary flow reserve (CFR) (also referred to as myocardial perfusion reserve), as measured by PET stress-rest myocardial perfusion imaging, in SCD patients with and without diastolic dysfunction, and healthy controls. Secondary Objectives * To investigate the relationship between decreased CFR (quantified with PET stress- rest myocardial perfusion imaging) and presence of abnormal diastolic parameters
This study aims to evaluate the use of virtual reality as an adjunct to standard care for patients with sickle cell disease experiencing vaso-occlusive crises.
HSCT is currently the only curative option for SCD but less than 20% of SCD patients have a MD donor available. So far, all curative approaches beyond a MSD HSCT at young age are non-satisfactory. With the lack of a suitable donor for the vast majority of patients, the major question of this trial is, if a haploidentical αß/CD19+ T-cell depleted HSCT can be a valid alternative to a MSD HSCT. The main challenge in non-malignant diseases is to offer a safe and GvHD-free HSCT without rejection.
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.
The goal of this study is to evaluate the effects of transauricular vagus and trigeminal nerve stimulation on the treatment of pain in children with Sickle Cell Disease. * The primary objective is to determine the feasibility of using a tAN device to treat pain in pediatric participants with SCD by assessing the completion rate, stimulation tolerability, and acceptability of the intervention measures. * The secondary objective is to assess the preliminary efficacy of using a tAN device as a remedy for pain in pediatric SCD subjects by analyzing inflammatory serum biomarker levels, the Functional Status Scale (FSS), the Wong-Baker FACES questionnaire, tracking analgesic consumption, and other assessments.
Brief Summary: \* A short description of the clinical study, including a brief statement of the clinical study's hypothesis, written in language intended for the lay public. Limit: 5000 characters. Severe forms of sickle cell syndrome are characterized by the occurrence of repeated vaso-occlusive crises (CVO), early complications and a high morbidity and mortality in these patients. Intensified management is then required, with the introduction of hydroxyurea treatment and then, if it proves ineffective, a transfusion program or even a haematopoietic stem cell allograft. These latter treatments present significant risks of adverse effects for the patient (haemochromatosis, erythrocyte alloimmunisation for the transfusion program, risk of GVH, chemotherapy-related toxicity, MVO for the allograft). Hydroxyurea (HU) is the first treatment based on the specific pathophysiology of sickle cell disease. It is the first line of therapeutic intensification for adult patients and children (age ≥ 2 years) with major sickle cell disease. By mainly increasing the percentage of fetal haemoglobin (HbF), HU decreases the frequency of CVO, complications, hospitalizations and prolongs the life expectancy of patients. The initial dose of HU, recommended by the ANSM, is 15 mg/kg/d once daily. However, the optimal dose cannot be predicted at the start of treatment, which is why a dosage adjustment is essential. The usual dose is between 15 and 35 mg/kg per day. Typically, the dose is increased every 3 months until a mild myelosuppression tolerated by the patient is reached, indicating that the maximum tolerated dose (MTD) has been reached. When the dose of HU has reached the MTD, the ratio of clinical (reduced frequency of vaso-occlusive attacks) and biological (better % of HbF) benefits to risk (toxicity) is optimal for the patient. The disadvantages of this practice are that: * dose escalation can be long (9-12 months) * clinicians may be reluctant to escalate HU to MTD * patients are treated sub-optimally during the therapeutic adaptation period. Recent work has shown that it is beneficial for the patient to adjust the initial dose using a pharmacological therapeutic approach in addition to monitoring haematological tolerance. Thus, by customizing the dose of HU using an area under the curve (AUC) measurement at the initial intake of HU at a standardized dose (20 mg/kg/day), the MTD would be achieved in a faster time frame of 6-9 months. The primary objective of our trial is to identify the methodology that will most effectively decrease the time to reach the MTD (therapeutic target). The immediate benefit will be a reduction in CVO which is the major clinical problem and leads to a risk of complications in sickle cell disease.
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
Short description of the protocol intended for the lay public. Include a brief statement of the study hypothesis (Limit : 5000 characters) The sickle cells anemia is a monogenic disease linked to the presence of Hemoglobin S due to a mutation in the Hemoglobin Beta chain. The lack of circulating oxygen induces a polymerization of the Hemoglobin S which change the red cell conformation into sickle. Those cells interact and causes vaso-occlusive crisis (CVO). The MEOPA is a medical gas used as an antalgic and a sedative especially in sickle cells disease patients. The nitrous oxide, oxide the cobalt ion in the vitamin B12 which inactivate it irreversibly creating a functional deficiency. During the metabolism of vitamin B12, homocysteine is transformed in methionine which is used in to form the myelin sheath and helped in producing DNA. Numerous studies already shown that the longer the exposition to MEOPA is the greater the functional deficiency of vitamin B12 occur. A few studies shown a symptomatic deficiency of vitamin B12 due to the exposition of MEOPA in sickle cells patient but there is no explanation on the necessary amount of exposure or if some patients are more at risk. When there is a deficiency of vitamin B12 the symptoms can go from a simple orthostatic hypotension to a combined spinal sclerosis. The participation to the study will be proposed to every patient hospitalized for a CVO in the follow up of the emergency room visit or directly in pediatric reanimation. During a usual blood test, a small amount of blood (4mL) will be collected in addition to dose the Vitamin B12, the vitamin B9, the homocysteine, and the methionine. A small amount of urine will also be collected to dose the methylmalonic acid, all those elements are a part of the metabolism of B12 vitamin. The same sample will be taken on the day of departure of the hospital. During the hospitalization the pain management, a daily neurological exam, and the exposition to the MEOPA will be assessed meticulously. An appointment will take place at 7 days and at one month after the hospital departure to evaluate the possible neurological defect. Each patient can only be included once.
Feasibility and reliability of ultrasound in the inpatient hematology setting.
The purpose of this research is to determine whether a new stress management and mindfulness program can improve quality of life in individuals with sickle cell disease. The program is based on a number of empirically supported treatments including techniques from cognitive-behavioral therapy like overcoming motivational barriers to self-care, learning to see things as accurately as possible, practicing assertive communication, relaxation exercises like deep slow breathing, mindfulness meditation, and other stress management strategies.
The goal of this randomized control clinical trial is to learn if virtual reality can be used to treat sickle cell pain in children. The main questions it aims to answer are: Does virtual reality reduce pain severity during a child's hospital stay for a vaso-occlusive pain crisis? Does virtual reality decrease the daily use of opiates? Researchers will compare standard therapy to the use of standard therapy plus a daily virtual reality experience to see if virtual reality works to treat sickle cell pain. All patients will: \- Be asked to fill out a pain assessment survey three times daily for up to 3 days If randomized to intervention arm, patients will: * Participate in an immersive virtual reality experience once daily for up to 3 days * Fill out a survey twice daily to monitor for side effects from virtual reality experience * Fill out a satisfaction survey once during the study period
In a hybrid type I effectiveness-implementation trial, our three-center research teams aim to examine whether empowering adults with sickle cell disease (SCD) with patient-facing SCD-specific guidelines through an mHealth application with booklets will decrease acute healthcare utilization and be cost-effective over booklets with the guidelines alone. Our team, head will test our hypotheses with the following aims: Aim 1: evaluate the effectiveness of the patient-facing guidelines mHealth app + booklet intervention to decrease acute healthcare utilization (hospitalizations, emergency room visits, and day hospital visits) in adults with SCD over the standard care in a randomized controlled trial, Aim 2: evaluate the implementation outcomes of the mHealth app + booklet using the capability, opportunity, and motivation-behavior (COM-B) and reach, effectiveness, adoption, implementation, and maintenance (RE-AIM) frameworks, and Aim 3: evaluate the cost-effectiveness of patient-facing mHealth app + booklets vs. standard care in adults with SCD. is hybrid effectiveness-implementation trial design, according to the COM-B and RE-AIM frameworks with a mixed-methods approach, will give valuable insights into the effects, facilitators, and barriers to the implementation that will influence the effects of the patient-facing guidelines intervention.
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 injection in treating sickle cell disease.
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft failure and to aid in immune reconstitution following hematopoietic stem cell transplantation.
Sickle cell disease is a chronic disease characterized by multiple vaso-occlusive complications. The basic treatment for patients with a vaso-occlusive crisis (VOC) is based on adequate hydration, oxygen therapy and pain control. Loco Regional Anesthesia (LRA) is one of the major treatments in resuscitation anesthesia for both anesthesia and analgesia. LRA allows effective and almost immediate pain control by blocking nerve afferents in a given area. LRA could decrease the inflammatory response during crises and accelerate resolution of the crisis.
45 trials · Recruiting