Prophylactic Transfusion for Sickle Cell Disease in Pregnancy

(ProTIP Trial)

Overseen By Ross Fasano, MD

Age: 18+

Sex: Female

Trial Phase: Phase 4

Sponsor: Emory University

Disqualifiers: Chronic transfusion therapy, DHTR, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 8-10 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether regular blood transfusions (prophylactic transfusion) can reduce complications during pregnancy for women with sickle cell disease (SCD). SCD often causes painful episodes and other health issues, and the study aims to determine if transfusions can lower these risks compared to standard care. Participants will receive either standard care or regular blood transfusions throughout their pregnancy and up to two months postpartum. Women who are currently pregnant (6 to 20 weeks along) and have sickle cell disease may be suitable for this trial. As a Phase 4 trial, this study involves an FDA-approved treatment and aims to understand how it can benefit more patients.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What is the safety track record for prophylactic transfusion?

Research has shown that red blood cell transfusions for pregnant women with sickle cell disease (SCD) are a well-researched treatment. These transfusions may improve health outcomes for both mother and baby. However, specific information about their safety and benefits during pregnancy remains limited.

Blood transfusions are generally considered safe, though they can cause side effects. Some individuals might experience mild reactions, such as a fever or an allergic response. Serious issues, like infections or iron overload, are rare. Using carefully matched blood reduces these risks.

Since transfusions are commonly used to manage SCD in pregnancy, they are usually well-tolerated. However, it is important to weigh the potential risks and benefits of any treatment. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Researchers are excited about prophylactic red blood cell transfusions for sickle cell disease in pregnancy because this approach aims to maintain hemoglobin S (HbS) levels below 30%, which could potentially reduce complications during pregnancy. Unlike the standard care, which often involves managing symptoms and complications as they arise, this proactive method could help prevent complications before they occur. By regularly transfusing red blood cells between 6 and 20 weeks of gestation and maintaining low HbS levels, the treatment could help ensure healthier pregnancies and better outcomes for both mother and baby.

What evidence suggests that prophylactic transfusion might be an effective treatment for sickle cell disease in pregnancy?

Research has shown that regular blood transfusions for pregnant women with sickle cell disease (SCD) can improve health outcomes for both mother and baby. In this trial, participants in the Red Blood Cell (RBC) Transfusion arm will receive blood transfusions between 6 and 20 weeks of gestation, repeated at 3-6 week intervals, to maintain HbS levels below 30%. These transfusions help reduce serious complications related to SCD during pregnancy. Although experts do not fully agree on whether it prevents all pregnancy complications, it is commonly used as a treatment for pregnant women with SCD. Overall, evidence suggests that these transfusions can help manage SCD during pregnancy, potentially leading to better health for both mother and baby. ^{1 2 3 4 6}

Who Is on the Research Team?

Ross Fasano, MD

Principal Investigator

Emory University

Are You a Good Fit for This Trial?

This trial is for pregnant women with Sickle Cell Disease. It aims to see if regular blood transfusions during pregnancy and two months post-partum can reduce complications compared to standard care. Women must be expecting and diagnosed with SCD, but specific inclusion and exclusion criteria are not provided.

Inclusion Criteria

I understand the study's risks and agree to participate.

I am female.

I have been diagnosed with sickle cell disease.

Exclusion Criteria

Prior history of DHTR with hyperhemolysis

I was on regular blood transfusions before becoming pregnant.

Known current triplet pregnancy

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive repeated red blood cell transfusions or standard care from 6 to 20 weeks of gestation, repeated at 3-6 week intervals

8-10 months

Follow-up

Participants are monitored for safety and effectiveness after treatment, including hospital admissions, emergency visits, and SCD-related complications

8 weeks post-partum

What Are the Treatments Tested in This Trial?

Interventions

• Prophylactic Transfusion

Trial Overview The study tests whether prophylactic red blood cell transfusions can lower the risk of acute SCD episodes or pregnancy-related issues in women with SCD. Participants will either receive regular transfusions or standard care throughout their pregnancy until two months after giving birth.

How Is the Trial Designed?

Group I: Standard of Care Experimental Treatment 1 Intervention

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.

Group II: Red Blood Cell (RBC) Transfusion Experimental Treatment 1 Intervention

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%

Who Is Running the Clinical Trial?

Emory University

Lead Sponsor

Trials

1,735

Recruited

2,605,000+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39252331/

The role of prophylactic transfusion on the maternal and ... The present meta-analysis indicated that prophylactic blood transfusion in pregnant women with sickle cell disease may improve maternal and fetal outcomes.

2. journals.lww.com journals.lww.com/md-journal/fulltext/2024/09060/the_role_of_prophylactic_transfusion_on_the.118.aspx

The role of prophylactic transfusion on the maternal and... The present meta-analysis indicated that prophylactic blood transfusion in pregnant women with sickle cell disease may improve maternal and fetal outcomes.

3. sciencedirect.com sciencedirect.com/science/article/pii/S2473952924003987

Prophylactic exchange transfusion in sickle cell disease ... Exchange transfusion is used for SCD complications, but there is inadequate evidence of its safety and benefit in pregnancy. •. The TAPS2 trial ...

4. ashpublications.org ashpublications.org/hematology/article/2023/1/640/506395/Managing-pregnancy-in-patients-with-sickle-cell

Managing pregnancy in patients with sickle cell disease from ... We describe here a case-by-case approach to the management of pregnancy in women with SCD based on the medical and transfusion history of each patient.

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11427169/

Prophylactic Red Cell Transfusions for Sickle Cell Disease ... ... Sickle Cell Disease ... prophylactic red cell transfusions significantly reduce major SCD pregnancy complications and improve fetal outcomes.

6. ajog.org ajog.org/article/S0002-9378%2823%2900767-6/fulltext

Society for Maternal-Fetal Medicine Consult Series #68 Pregnant individuals with sickle cell disease have an increased risk of maternal and perinatal morbidity and mortality.