Comparison of Two Methods of Transfusion for Stroke Prevention in Sickle Cell

NCT02561312 | Completed

• 1 other identifier: 15-084
• Study Type: observational
• Target: 9
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Anemia, Sickle Cell; Sickling Disorder Due to Hemoglobin S

Outcome Measures

Primary Outcomes (1)

• Hemoglobin S, baseline hemoglobin/hematocrit,

  Lab parameters pre-infusion for each method of transfusion

  Pre Infusion, lab collected monthly for one year thru study completion

Secondary Outcomes (1)

• Hemoglobin S, end of transfusion hemoglobin/hematocrit, blood volume, alloantibodies,

  Post Infusion, lab collected monthly for one year thru study completion

Other Outcomes (2)

• Nursing Time score

  Monthly at end of each transfusion for one year thru study completion

• Patient Satisfaction Questionnaire

  At end of 6 month period and at 12 months (after RMPET and ST)

Study Arms (2)

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.

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.

Other: Simple Transfusion

Interventions

Rapid manual partial exchange transfusion OTHER

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.

RMPET

Simple Transfusion OTHER

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.

Simple Transfusion

Eligibility Criteria

• Age: 3 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

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.

You may qualify if:

• 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%

You may not qualify if:

• 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.

Sponsors & Collaborators

• Chattanooga-Hamilton County Hospital Authority: lead

Study Sites (1)

Chidlren's Hospital at Erlanger

Chattanooga, Tennessee, 37403, United States

Location

Related Publications (9)

• 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.

  PMID: 11953734 BACKGROUND

• Hulbert 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.

  PMID: 20940417 BACKGROUND

• Ware 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.

  PMID: 22318199 BACKGROUND

• Adams 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.

  PMID: 9647873 BACKGROUND

• Enninful-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.

  PMID: 20434165 BACKGROUND

• McCarville 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.

  PMID: 18085672 BACKGROUND

• McCavit 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.

  PMID: 23151905 BACKGROUND

• Adams 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.

  PMID: 16382063 BACKGROUND

• Casella 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.

  PMID: 20201689 BACKGROUND

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Jennifer Keates, MD

  Children's Hospital at Erlanger

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CROSSOVER
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Pediatric Oncologist / Hematologist

Study Record Dates

• First Submitted: September 23, 2015
• First Posted: September 28, 2015
• Study Start: September 1, 2015
• Primary Completion: September 30, 2016
• Study Completion: September 30, 2017
• Last Updated: July 27, 2018

Record last verified: 2018-07

Locations

US (1)