Prophylactic Transfusion In Pregnant in Women With Sickle Cell Disease
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Number of participants with pregnancy related complications
研究概览
简要总结
The goal of this study is to determine if there is a positive effect of prophylactic red blood cell (RBC) transfusion of leukoreduced, ABO, Rh (D/Cc/Ee) and Kell matched blood compared to standard of care on the number of episodes of acute sickle cell disease (SCD) manifestations or pregnancy-related complications requiring acute health care encounters (acute care/ER/Hospital visits) or resulting in death over the entirety of pregnancy until 2 months post-partum in women with SCD.
RBC transfusion is the only disease-modifying therapy for pregnant women with SCD, and it is considered a standard treatment option however, there exists no consensus on the role of transfusion therapy in preventing SCD-related pregnancy complications.
Participants will be randomly assigned to repeated red blood cell transfusions or the standard of care. Participants will be on study for about 8-10 months (Pregnancy through 2 months post-partum).
详细描述
Sickle cell disease (SCD) is a common genetic disorder that results from the homozygous presence of abnormal β-globin chains (hemoglobin Hb SS, Hb SC, HbSβ thalassemia). SCD causes red blood cells to become rigid, leading to various acute and chronic manifestations: chronic hemolytic anemia, poor growth, acute vaso-occlusive pain crises (VOC), priapism (in men) acute ischemic stroke, acute chest syndrome (ACS), and chronic organ damage within the spleen, kidneys, liver, lungs and heart.
High rates of both maternal and fetal morbidity and mortality complicate pregnancy in patients affected by SCD. SCD pregnancies have been linked to higher rates of obstetrical complications, including preeclampsia, venous thromboembolism, intrauterine growth restriction, preterm delivery, and small-for-gestational-age infants. In addition, sickle-related maternal complications are common during pregnancy. More than 50% of women with SCD have a VOC in the antenatal period (76% for HbSS vs 27% for Hb SC). ACS in pregnancy is seen in 7% to 20% of women with HbSS and approximately 5% in women with HbSC. Prophylactic transfusion therapy has established benefits in stroke prevention and preoperative optimization in SCD patients, but its use in pregnancy has not been established. Because hydroxyurea (HU) may be teratogenic, RBC transfusion is the only disease-modifying therapy available for pregnant women with SCD.
The decision to put a pregnant woman with SCD on chronic transfusion therapy is entirely based on provider preference and patient willingness. RBC transfusions are considered a standard treatment option for pregnant women with SCD and transfusion therapy widely used, however there exists no consensus among providers on the role of chronic transfusion therapy in preventing SCD-related pregnancy complications and no prospective randomized controlled study investigating the role of prophylactic transfusion for prevention of both maternal and fetal morbidity has been done.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Female
- 接受健康志愿者
- 是
入选标准
- •Diagnosis of SCD of any genotype (i.e., HbSS, HbSC, HbSβ thalassemia)
- •18 Years and older
- •Currently pregnant at 6 weeks through 20 weeks of gestation.
- •Ability to understand the purposes and risks of the study and willingly give informed consent.
- •For participants with private health insurance, insurance pre-approval for blood transfusions
排除标准
- •Currently on chronic transfusion therapy before pregnancy
- •Prior history of DHTR with hyperhemolysis
- •Red cell antibody history, which would prevent the provision of adequate red cell units to support chronic transfusions.
- •Unable or unwilling to receive blood transfusion for social, religious, or clinical reasons
- •Known current triplet pregnancy
- •Current diagnosis of major medical or psychiatric comorbidity, which in the randomizing clinician's opinion renders them unable to enter a clinical trial.
研究组 & 干预措施
Red Blood Cell (RBC) Transfusion
Participants will receive a blood transfusion between 6 and 20 weeks of gestation. It will be repeated at 3-6 week intervals, aiming to maintain HbS <30%
干预措施: Prophylactic Transfusion Intervention group: Transfusion (Biological)
Standard of Care
Patients randomized to the control group will receive standard care for SCD alone. As part of the standard of care, women with SCD who become pregnant and who are on hydroxyurea (HU) will have the HU suspended by their primary SCD provider.
干预措施: Control group (Other)
结局指标
主要结局
Number of participants with pregnancy related complications
时间窗: Baseline (enrollment) up to 8 weeks post-partum
Number of participants who will develop pregnancy-related complications (pre-eclampsia, venous thromboembolism, infection). Collected from medical records
Maternal death
时间窗: From randomization up to 8 weeks post-partum
Number of women who die at any gestational age during pregnancy or within 8 weeks after delivery from any cause arising from the pregnancy or its management, but not from incidental or accidental causes, divided by the total number of participants.
Number of maternal emergency department (ED)/Acute care visits
时间窗: Baseline (enrollment) up to 8 weeks post-partum
Collected from medical records
Number of SCD-related complications per group
时间窗: Baseline (enrollment) up to 8 weeks post-partum
Sickle cell complications (vaso-occlusive crisis (VOC) / acute chest syndrome (ACS) / cerebral stroke) collected from medical records
Hospital admissions rate
时间窗: Baseline (enrollment) up to 8 weeks post-partum
Number of participants who require hospital admission during study participation divided by the total number of participants. Collected from medical records
次要结局
- Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me)(2, 4, 6, 8 and 10 months post randomization.)
- Infant birth weight(At delivery up to 42 weeks of pregnancy)
- Number of neonatal intensive care unit/critical care admissions(After neonate's delivery up to 1 week post-partum)
- Number of participants with transfusion reactions(From enrollment up to 8 weeks post-partum)
- Preterm delivery(Up to delivery (36 weeks of pregnancy))
- Fetal demise/stillbirth(From enrollment up to 42 weeks of pregnancy)
- Number of participants with an increase in iron overload (serum ferritin)(From enrollment up to 8 weeks post-partum)
- Patient reported outcomes measurement information system (PROMIS)(2, 4, 6, 8 and 10 months post randomization.)
- APGAR Scores(1 and 5 minutes after neonate's delivery)
- Perinatal death(From 28 weeks of pregnancy up to 1 week post-partum)
- Number of participants with new RBC alloantibodies(From enrollment up to 8 weeks post-partum)
研究者
Ross Fasano
Associate Professor
Emory University