Anemia, Sickle CellNCT02561312
Comparison of Two Methods of Transfusion for Stroke Prevention in Sickle Cell
An observational study of Anemia, Sickle Cell and Sickling Disorder Due to Hemoglobin S, run by Chattanooga-Hamilton County Hospital Authority at 1 site in United States.
Trial summary
Chronic blood transfusions are essential supportive care for sickle cell patients at high risk for morbidity and mortality due to stroke. These patients, however, are at risk for iron overload. Read more about this study
- Who is it for?
- Ages 3 to 25 years
- Conditions the study is for
- Anemia, Sickle Cell, Sickling Disorder Due to Hemoglobin S
- What is being tested?
- Rapid manual partial exchange transfusion and Simple Transfusion
- Type of treatment
- Injection or infusion
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2015to 30 Sep 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: 8 chronically exchanged transfused participants at T.C. Thompson Children's Infusion Clinic with sickle cell disease (Hemoglobin SS or SBeta thalassemia) currently receiving rapid manual partial exchange transfusion.
Who may be able to join
- Participants between 3 and 25 years of age
- Diagnosis of Hemoglobin SS or SBeta thalassemia
- On chronic exchange for stroke prevention
- Performance status: Lansky play score of 100%, and if over 16 years of age, Karnofsky=100%
Who cannot join
- Participant has experienced more than one stroke and has a modified Rankin Scale of >3.
- Diagnosis of Hemoglobin SC disease
- Participants on chronic transfusion for priapism.
Trial locations and contacts
1 site in 1 country.
United States1 site
Chidlren's Hospital at Erlanger
Chattanooga, Tennessee, 37403
About this study
Chronic blood transfusions are essential supportive care for sickle cell patients at high risk for morbidity and mortality due to stroke. These patients, however, are at risk for iron overload. In the investigator's comprehensive sickle cell center, the investigators support chronic transfusion with rapid manual partial exchange transfusions (RMPET) using a single access central line port. The investigators do not have a comprehensive adult sickle cell program but upon transition of patients the patients would be provided simple transfusion (ST) in an adult ambulatory infusion setting due to nursing acuity needed for RMPET. The investigators plan to study the institution's participants currently on chronic transfusion support and compare different transfusion modalities to better understand the effects from switching from RMPET to ST. To date, there are no such comparisons within and between sickle cell patients in the literature.
Read the detailed description
II. Objective. Compare differences in RMPET versus ST.
III. Specific Aims:
- To compare key predictive hematologic factors (hematocrit, hemoglobin, hemoglobin S quantification, blood volume and alloantibodies) for relative risk of stroke utilizing two methods of blood transfusion therapy.
- To determine the nursing time to administer straight versus manual exchange transfusion therapy.
- To survey patient satisfaction for both procedures.
IV: Background/Significance:
Stroke occurs in 10% of Sickle Cell Disease (SCD) patients before the age of 20 Years. Current standard of care for secondary overt stroke prevention in patients with SCD is chronic red blood cell (RBC) transfusions. Stroke recurs in ~ 60% of patients without chronic RBC therapy and in ~ 20% of patients with chronic transfusion while maintaining a hemoglobin S percentage of less than 30%. Indefinite transfusion therapy is practiced as discontinuation after short-term or long-term prophylactic transfusions leads to recurrent overt strokes and more ensuing CNS damage, even with transition to hydroxyurea. Chronic transfusions also prevent initial stroke in high-risk patients identified by transcranial Doppler (TCD) ultrasound. The Stroke Prevention Study in Sickle Cell Disease (STOP) demonstrated a 92% stroke risk reduction among 63 of 130 children with abnormal TCD results. Rates of stroke declined significantly since implementing routine TCD screening and primary prophylactic transfusion therapy. The subsequent STOP 2 trial supports the use of chronic transfusion indefinitely because discontinuation resulted in an increased rate of abnormal TCD conversion and development of overt stroke. Discontinuing transfusions on the STOP 2 trial was also associated with a higher occurrence of silent cerebral infarcts, documented in 3 of 37 patients (8.1%) in the continued-transfusion group compared with 11 of 40 (27.5%) in the transfusion-halted group. More recent studies demonstrate that SCD patients are also at risk for silent cerebral infarcts. An association between worsening vasculopathy shown by magnetic resonance angiography and progressive overt and silent infarcts on magnetic resonance imaging has been found. More aggressive magnetic resonance imaging screening may be indicated and this could result in more patients with SCD treated with chronic transfusion.
Common chronic transfusion modalities include ST or RMPET. The goal of therapy is to reduce the hemoglobin S level either by diluting the blood (ST) or by removing and replacing the blood with non-sickle hemoglobin (RMPET). To prevent further brain injury, the goal of transfusion therapy is to lower the hemoglobin S quantification to less than 30% on a routine basis, usually monthly transfusion procedure. There are many large centers that utilize erythrocytapheresis which is considered the preferable method if available.
The investigator's study will focus on the types of RBC exchange therapy currently utilized in the investigator's Infusion Clinic at T.C. Thompson Children's Hospital. The investigators will determine which transfusion method is best for each participant for achieving the hematologic parameters of lower hemoglobin S quantification.The investigators will also measure the amount of nursing time for each procedure and which method is preferred by participants. The investigators will share the institutional observations with other institutions who may intend to switch between RMPET and ST.
- Methods:
Study Design: Prospective observational cohort study
The investigators will utilize the institution's current population of 8 eligible chronically exchanged transfused participants at the T.C. Thompson Children's Infusion Clinic. The eligible participants will be invited to participate in a cross over design study so that each participant serves as its own control. Eight participants will be randomly assigned (blinded envelope) whereby four participants will start with rapid manual partial exchange transfusion: 3 months will be spent in a wash out period, then 3 months of data collection for RMPET. This group will then be switched to simple transfusion with a wash out period of three months, then data collection for three months. The second group of four participants will start with simple transfusion and have an identical study design over 12 months (3 months of collected data during simple transfusion, a 3 month wash out period, switch to rapid manual partial exchange transfusion for three months, then 3 months of data collection for RMPET).
The investigators will optimize all therapy to achieve the post transfusion goal of <30% hemoglobin S and post transfusion Hb <12g/DL.with each transfusion performed for best practice in avoiding sickle cell complications.
- Being tested
- Rapid manual partial exchange transfusion, Simple Transfusion
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rapid manual partial exchange transfusionOther
The first four participants will receive peripheral red blood cells via rapid manual partial exchange transfusions every month for 6 months. There is a pre-study washout for 3 months then there is a 3 month test period (data collection) before the participant is transferred to ST treatment.
Simple TransfusionOther
The second group of four participants will receive peripheral red blood cells via simple transfusion every month for 6 months. There is a pre-study washout period for 3 months then there is a 3 month test period (data collection) before the participant is transferred to RMPET treatment.
| Study group | Role | What they receive |
|---|---|---|
| RMPET | For rapid manual partial exchange transfusion, participants with a weight >50kg, 500 ml of whole blood is removed from the participant via a single lumen central venous line, followed by infusion of 500 ml of saline. A 30 second wait time is utilized for equilibration to occur. A second 500 ml aliquot is removed, and then two units of packed red blood cells (PRBC) are infused. (This is customized for a patient with large red blood cell mass). For participants <50 kg, the individual exchange aliquots are adjusted to 10 ml/kg or normal saline and PRBC. Receives: Other: Rapid manual partial exchange transfusion | |
| Simple Transfusion | For simple transfusion, the volume of packed red blood cells (PRBC) to be transfused in the participant is 10-15 cc/kg. No normal saline exchange is required. All blood is transfused through a single lumen central venous line. Receives: Other: Simple Transfusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hemoglobin S, baseline hemoglobin/hematocrit,
Lab parameters pre-infusion for each method of transfusion
Measured: Pre Infusion, lab collected monthly for one year thru study completion
Secondary outcomes (1)
Hemoglobin S, end of transfusion hemoglobin/hematocrit, blood volume, alloantibodies,
Lab parameters post-infusion for each method of transfusion
Measured: Post Infusion, lab collected monthly for one year thru study completion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2015 (estimated)
- Study startSep 2015
- Primary completion30 Sep 2016
- Study completion30 Sep 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (9)Papers the sponsor has linked to this study.
- Other trials for Anemia, Sickle CellStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case crossover
- Time perspective
- Prospective
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chattanooga-Hamilton County Hospital Authority
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jennifer Keates, M.D., Chattanooga-Hamilton County Hospital Authority
- Investigators
- Jennifer Keates, MD, Principal investigator, Children's Hospital at Erlanger
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02561312
- Sponsor's protocol ID
- 15-084
- Status last verified
- Jul 2018
- First submitted
- 23 Sep 2015
- Last updated
- 27 Jul 2018
- Results posted
- No
- Data monitoring committee
- No
Publications
Scothorn DJ, Price C, Schwartz D, Terrill C, Buchanan GR, Shurney W, Sarniak I, Fallon R, Chu JY, Pegelow CH, Wang W, Casella JF, Resar LS, Berman B, Adamkiewicz T, Hsu LL, Ohene-Frempong K, Smith-Whitley K, Mahoney D, Scott JP, Woods GM, Watanabe M, Debaun MR. Risk of recurrent stroke in children with sickle cell disease receiving blood transfusion therapy for at least five years after initial stroke. J Pediatr. 2002 Mar;140(3):348-54. doi: 10.1067/mpd.2002.122498.
PubMed 11953734Hulbert ML, McKinstry RC, Lacey JL, Moran CJ, Panepinto JA, Thompson AA, Sarnaik SA, Woods GM, Casella JF, Inusa B, Howard J, Kirkham FJ, Anie KA, Mullin JE, Ichord R, Noetzel M, Yan Y, Rodeghier M, Debaun MR. Silent cerebral infarcts occur despite regular blood transfusion therapy after first strokes in children with sickle cell disease. Blood. 2011 Jan 20;117(3):772-9. doi: 10.1182/blood-2010-01-261123. Epub 2010 Oct 12.
PubMed 20940417Ware RE, Helms RW; SWiTCH Investigators. Stroke With Transfusions Changing to Hydroxyurea (SWiTCH). Blood. 2012 Apr 26;119(17):3925-32. doi: 10.1182/blood-2011-11-392340. Epub 2012 Feb 7.
PubMed 22318199Adams RJ, McKie VC, Hsu L, Files B, Vichinsky E, Pegelow C, Abboud M, Gallagher D, Kutlar A, Nichols FT, Bonds DR, Brambilla D. Prevention of a first stroke by transfusions in children with sickle cell anemia and abnormal results on transcranial Doppler ultrasonography. N Engl J Med. 1998 Jul 2;339(1):5-11. doi: 10.1056/NEJM199807023390102.
PubMed 9647873Enninful-Eghan H, Moore RH, Ichord R, Smith-Whitley K, Kwiatkowski JL. Transcranial Doppler ultrasonography and prophylactic transfusion program is effective in preventing overt stroke in children with sickle cell disease. J Pediatr. 2010 Sep;157(3):479-84. doi: 10.1016/j.jpeds.2010.03.007.
PubMed 20434165McCarville MB, Goodin GS, Fortner G, Li CS, Smeltzer MP, Adams R, Wang W. Evaluation of a comprehensive transcranial doppler screening program for children with sickle cell anemia. Pediatr Blood Cancer. 2008 Apr;50(4):818-21. doi: 10.1002/pbc.21430.
PubMed 18085672McCavit TL, Xuan L, Zhang S, Flores G, Quinn CT. National trends in incidence rates of hospitalization for stroke in children with sickle cell disease. Pediatr Blood Cancer. 2013 May;60(5):823-7. doi: 10.1002/pbc.24392. Epub 2012 Nov 14.
PubMed 23151905Adams RJ, Brambilla D; Optimizing Primary Stroke Prevention in Sickle Cell Anemia (STOP 2) Trial Investigators. Discontinuing prophylactic transfusions used to prevent stroke in sickle cell disease. N Engl J Med. 2005 Dec 29;353(26):2769-78. doi: 10.1056/NEJMoa050460.
PubMed 16382063Casella JF, King AA, Barton B, White DA, Noetzel MJ, Ichord RN, Terrill C, Hirtz D, McKinstry RC, Strouse JJ, Howard TH, Coates TD, Minniti CP, Campbell AD, Vendt BA, Lehmann H, Debaun MR. Design of the silent cerebral infarct transfusion (SIT) trial. Pediatr Hematol Oncol. 2010 Mar;27(2):69-89. doi: 10.3109/08880010903360367.
PubMed 20201689
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparison of Two Methods of Transfusion for Stroke Prevention in Sickle Cell
- ClinicalTrials.gov ID
- NCT02561312
- Acronym / sponsor ID
- 15-084
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Chattanooga-Hamilton County Hospital AuthorityUniversity, hospital or other
- Study start
- Sep 2015 (actual)
- Primary completion
- 30 Sep 2016 (actual)
- Study completion
- 30 Sep 2017 (actual)
- First posted
- 28 Sep 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Chronic blood transfusions are essential supportive care for sickle cell patients at high risk for morbidity and mortality due to stroke. These patients, however, are at risk for iron overload. In the investigator's comprehensive sickle cell center, the investigators support chronic transfusion with rapid manual partial exchange transfusions (RMPET) using a single access central line port. The investigators do not have a comprehensive adult sickle cell program but upon transition of patients the patients would be provided simple transfusion (ST) in an adult ambulatory infusion setting due to nursing acuity needed for RMPET. The investigators plan to study the institution's participants currently on chronic transfusion support and compare different transfusion modalities to better understand the effects from switching from RMPET to ST. To date, there are no such comparisons within and between sickle cell patients in the literature.
Detailed description
II. Objective. Compare differences in RMPET versus ST.
III. Specific Aims:
- To compare key predictive hematologic factors (hematocrit, hemoglobin, hemoglobin S quantification, blood volume and alloantibodies) for relative risk of stroke utilizing two methods of blood transfusion therapy.
- To determine the nursing time to administer straight versus manual exchange transfusion therapy.
- To survey patient satisfaction for both procedures.
IV: Background/Significance:
Stroke occurs in 10% of Sickle Cell Disease (SCD) patients before the age of 20 Years. Current standard of care for secondary overt stroke prevention in patients with SCD is chronic red blood cell (RBC) transfusions. Stroke recurs in ~ 60% of patients without chronic RBC therapy and in ~ 20% of patients with chronic transfusion while maintaining a hemoglobin S percentage of less than 30%. Indefinite transfusion therapy is practiced as discontinuation after short-term or long-term prophylactic transfusions leads to recurrent overt strokes and more ensuing CNS damage, even with transition to hydroxyurea. Chronic transfusions also prevent initial stroke in high-risk patients identified by transcranial Doppler (TCD) ultrasound. The Stroke Prevention Study in Sickle Cell Disease (STOP) demonstrated a 92% stroke risk reduction among 63 of 130 children with abnormal TCD results. Rates of stroke declined significantly since implementing routine TCD screening and primary prophylactic transfusion therapy. The subsequent STOP 2 trial supports the use of chronic transfusion indefinitely because discontinuation resulted in an increased rate of abnormal TCD conversion and development of overt stroke. Discontinuing transfusions on the STOP 2 trial was also associated with a higher occurrence of silent cerebral infarcts, documented in 3 of 37 patients (8.1%) in the continued-transfusion group compared with 11 of 40 (27.5%) in the transfusion-halted group. More recent studies demonstrate that SCD patients are also at risk for silent cerebral infarcts. An association between worsening vasculopathy shown by magnetic resonance angiography and progressive overt and silent infarcts on magnetic resonance imaging has been found. More aggressive magnetic resonance imaging screening may be indicated and this could result in more patients with SCD treated with chronic transfusion.
Common chronic transfusion modalities include ST or RMPET. The goal of therapy is to reduce the hemoglobin S level either by diluting the blood (ST) or by removing and replacing the blood with non-sickle hemoglobin (RMPET). To prevent further brain injury, the goal of transfusion therapy is to lower the hemoglobin S quantification to less than 30% on a routine basis, usually monthly transfusion procedure. There are many large centers that utilize erythrocytapheresis which is considered the preferable method if available.
The investigator's study will focus on the types of RBC exchange therapy currently utilized in the investigator's Infusion Clinic at T.C. Thompson Children's Hospital. The investigators will determine which transfusion method is best for each participant for achieving the hematologic parameters of lower hemoglobin S quantification.The investigators will also measure the amount of nursing time for each procedure and which method is preferred by participants. The investigators will share the institutional observations with other institutions who may intend to switch between RMPET and ST.
- Methods:
Study Design: Prospective observational cohort study
The investigators will utilize the institution's current population of 8 eligible chronically exchanged transfused participants at the T.C. Thompson Children's Infusion Clinic. The eligible participants will be invited to participate in a cross over design study so that each participant serves as its own control. Eight participants will be randomly assigned (blinded envelope) whereby four participants will start with rapid manual partial exchange transfusion: 3 months will be spent in a wash out period, then 3 months of data collection for RMPET. This group will then be switched to simple transfusion with a wash out period of three months, then data collection for three months. The second group of four participants will start with simple transfusion and have an identical study design over 12 months (3 months of collected data during simple transfusion, a 3 month wash out period, switch to rapid manual partial exchange transfusion for three months, then 3 months of data collection for RMPET).
The investigators will optimize all therapy to achieve the post transfusion goal of <30% hemoglobin S and post transfusion Hb <12g/DL.with each transfusion performed for best practice in avoiding sickle cell complications.
Study design
- Observational model
- Case crossover
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| RMPET | – | Other: Rapid manual partial exchange transfusionFor rapid manual partial exchange transfusion, participants with a weight >50kg, 500 ml of whole blood is removed from the participant via a single lumen central venous line, followed by infusion of 500 ml of saline. A 30 second wait time is utilized for equilibration to occur. A second 500 ml aliquot is removed, and then two units of packed red blood cells (PRBC) are infused. (This is customized for a patient with large red blood cell mass). For participants <50 kg, the individual exchange aliquots are adjusted to 10 ml/kg or normal saline and PRBC. |
| Simple Transfusion | – | Other: Simple TransfusionFor simple transfusion, the volume of packed red blood cells (PRBC) to be transfused in the participant is 10-15 cc/kg. No normal saline exchange is required. All blood is transfused through a single lumen central venous line. |
Who may qualify
- Ages
- 3 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- 8 chronically exchanged transfused participants at T.C. Thompson Children's Infusion Clinic with sickle cell disease (Hemoglobin SS or SBeta thalassemia) currently receiving rapid manual partial exchange transfusion.
Key inclusion criteria (4 listed)
- Participants between 3 and 25 years of age
- Diagnosis of Hemoglobin SS or SBeta thalassemia
- On chronic exchange for stroke prevention
- Performance status: Lansky play score of 100%, and if over 16 years of age, Karnofsky=100%
Key exclusion criteria (3 listed)
- Participant has experienced more than one stroke and has a modified Rankin Scale of >3.
- Diagnosis of Hemoglobin SC disease
- Participants on chronic transfusion for priapism.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Rapid manual partial exchange transfusionThe first four participants will receive peripheral red blood cells via rapid manual partial exchange transfusions every month for 6 months. There is a pre-study washout for 3 months then there is a 3 month test period (data collection) before the participant is transferred to ST treatment. | Other | RMPET |
| Simple TransfusionThe second group of four participants will receive peripheral red blood cells via simple transfusion every month for 6 months. There is a pre-study washout period for 3 months then there is a 3 month test period (data collection) before the participant is transferred to RMPET treatment. | Other | Simple Transfusion |
Primary outcome
Hemoglobin S, baseline hemoglobin/hematocrit,
Time frame: Pre Infusion, lab collected monthly for one year thru study completion
Lab parameters pre-infusion for each method of transfusion
1 secondary outcome
Hemoglobin S, end of transfusion hemoglobin/hematocrit, blood volume, alloantibodies,
Time frame: Post Infusion, lab collected monthly for one year thru study completion
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2018.
- Background: PubMed 11953734Scothorn DJ, Price C, Schwartz D, Terrill C, Buchanan GR, Shurney W, Sarniak I, Fallon R, Chu JY, Pegelow CH, Wang W, Casella JF, Resar LS, Berman B, Adamkiewicz T, Hsu LL, Ohene-Frempong K, Smith-Whitley K, Mahoney D, Scott JP, Woods GM, Watanabe M, Debaun MR. Risk of recurrent stroke in children with sickle cell disease receiving blood transfusion therapy for at least five years after initial stroke. J Pediatr. 2002 Mar;140(3):348-54. doi: 10.1067/mpd.2002.122498.
- Background: PubMed 20940417Hulbert ML, McKinstry RC, Lacey JL, Moran CJ, Panepinto JA, Thompson AA, Sarnaik SA, Woods GM, Casella JF, Inusa B, Howard J, Kirkham FJ, Anie KA, Mullin JE, Ichord R, Noetzel M, Yan Y, Rodeghier M, Debaun MR. Silent cerebral infarcts occur despite regular blood transfusion therapy after first strokes in children with sickle cell disease. Blood. 2011 Jan 20;117(3):772-9. doi: 10.1182/blood-2010-01-261123. Epub 2010 Oct 12.
- Background: PubMed 22318199Ware RE, Helms RW; SWiTCH Investigators. Stroke With Transfusions Changing to Hydroxyurea (SWiTCH). Blood. 2012 Apr 26;119(17):3925-32. doi: 10.1182/blood-2011-11-392340. Epub 2012 Feb 7.
- Background: PubMed 9647873Adams RJ, McKie VC, Hsu L, Files B, Vichinsky E, Pegelow C, Abboud M, Gallagher D, Kutlar A, Nichols FT, Bonds DR, Brambilla D. Prevention of a first stroke by transfusions in children with sickle cell anemia and abnormal results on transcranial Doppler ultrasonography. N Engl J Med. 1998 Jul 2;339(1):5-11. doi: 10.1056/NEJM199807023390102.
- Background: PubMed 20434165Enninful-Eghan H, Moore RH, Ichord R, Smith-Whitley K, Kwiatkowski JL. Transcranial Doppler ultrasonography and prophylactic transfusion program is effective in preventing overt stroke in children with sickle cell disease. J Pediatr. 2010 Sep;157(3):479-84. doi: 10.1016/j.jpeds.2010.03.007.
- Background: PubMed 18085672McCarville MB, Goodin GS, Fortner G, Li CS, Smeltzer MP, Adams R, Wang W. Evaluation of a comprehensive transcranial doppler screening program for children with sickle cell anemia. Pediatr Blood Cancer. 2008 Apr;50(4):818-21. doi: 10.1002/pbc.21430.
- Background: PubMed 23151905McCavit TL, Xuan L, Zhang S, Flores G, Quinn CT. National trends in incidence rates of hospitalization for stroke in children with sickle cell disease. Pediatr Blood Cancer. 2013 May;60(5):823-7. doi: 10.1002/pbc.24392. Epub 2012 Nov 14.
- Background: PubMed 16382063Adams RJ, Brambilla D; Optimizing Primary Stroke Prevention in Sickle Cell Anemia (STOP 2) Trial Investigators. Discontinuing prophylactic transfusions used to prevent stroke in sickle cell disease. N Engl J Med. 2005 Dec 29;353(26):2769-78. doi: 10.1056/NEJMoa050460.
- Background: PubMed 20201689Casella JF, King AA, Barton B, White DA, Noetzel MJ, Ichord RN, Terrill C, Hirtz D, McKinstry RC, Strouse JJ, Howard TH, Coates TD, Minniti CP, Campbell AD, Vendt BA, Lehmann H, Debaun MR. Design of the silent cerebral infarct transfusion (SIT) trial. Pediatr Hematol Oncol. 2010 Mar;27(2):69-89. doi: 10.3109/08880010903360367.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jennifer Keates, MD principal investigator
Children's Hospital at Erlanger
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Anemia, Sickle Cell and Sickling Disorder Due to Hemoglobin S, run by Chattanooga-Hamilton County Hospital Authority at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Jul 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hemoglobin S, baseline hemoglobin/hematocrit, (time frame: Pre Infusion, lab collected monthly for one year thru study completion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02561312, which also lists 9 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jul 2018.