NCT00495638

Pulmonary Hypertension, Hypoxia and Sickle Cell Disease

Completed Last updated 16 December 2019

What this trial tests

trial in Chuvash Polycythemia in 29 participants. Completed in 2 December 2014.

Timeline

28 June 2007

2 December 2014

Quick facts

Lead sponsor	National Heart, Lung, and Blood Institute (NHLBI)
Status	Completed
Study type	OBSERVATIONAL
Enrollment	29
Start date	28 June 2007
Estimated completion	2 December 2014
Sites	7 locations across Russia, United States

Conditions studied

• Chuvash Polycythemia — all drugs for Chuvash Polycythemia →
• Cerebrovascular Disease — all drugs for Cerebrovascular Disease →
• Pulmonary Hypertension — all drugs for Pulmonary Hypertension →
• Sickle Cell Anemia — all drugs for Sickle Cell Anemia →

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Who can join

Adults 3 to 20, any sex, with Chuvash Polycythemia or Cerebrovascular Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The study will look at the risk factors for pulmonary hypertension (high blood pressure in the lungs) in children and adolescents with sickle cell anemia (SCA) and examine the role of hypoxia (oxygen shortage) in the disease. In patients with SCA, red blood cells become sickle-shaped and tend to form clumps that get stuck in blood vessels, blocking blood flow to the limbs and organs. Blocked blood vessels can cause pain, serious infections, and organ damage. Many patients with SCA also develop pulmonary hypertension.

Children and adolescents with SCA or Chuvash polycythemia (another blood disorder that carries an increased risk for pulmonary hypertension) may be eligible for this study.

Participants undergo the following procedures at the beginning (baseline) and end of the study:

* History, physical examination and blood tests .
* Echocardiography (ultrasound study of heart function).
* Transcranial doppler (brain ultrasound study to measure brain blood flow).
* Lung function tests.
* 6-minute walk (measure of the distance covered in 6 minutes of walking).

In addition, patients are followed by telephone or by clinic visits every 6 months for a review of their medical history and medications. A physical examination is also done at 12 months.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Markers of severe vaso-occlusive painful episode frequency in children and adolescents with sickle cell anemia.
   Darbari DS, Onyekwere O, Nouraie M, Minniti CP, et al · · 2012 · cited 78× · PMID 21890147 · DOI 10.1016/j.jpeds.2011.07.018
2. Meta-analysis of 2040 sickle cell anemia patients: BCL11A and HBS1L-MYB are the major modifiers of HbF in African Americans.
   Bae HT, Baldwin CT, Sebastiani P, Telen MJ, et al · · 2012 · cited 66× · PMID 22936743 · DOI 10.1182/blood-2012-06-432849
3. Prospective echocardiography assessment of pulmonary hypertension and its potential etiologies in children with sickle cell disease.
   Dham N, Ensing G, Minniti C, Campbell A, et al · · 2009 · cited 44× · PMID 19699350 · DOI 10.1016/j.amjcard.2009.04.034
4. Tricuspid regurgitation velocity and other biomarkers of mortality in children, adolescents and young adults with sickle cell disease in the United States: The PUSH study.
   Nouraie M, Darbari DS, Rana S, Minniti CP, et al · · 2020 · cited 26× · PMID 32243618 · DOI 10.1002/ajh.25799
5. Clinical correlates of acute pulmonary events in children and adolescents with sickle cell disease.
   Paul R, Minniti CP, Nouraie M, Luchtman-Jones L, et al · · 2013 · cited 24× · PMID 23560516 · DOI 10.1111/ejh.12118
6. Bayesian methods for multivariate modeling of pleiotropic SNP associations and genetic risk prediction.
   Hartley SW, Monti S, Liu CT, Steinberg MH, et al · · 2012 · cited 24× · PMID 22973300 · DOI 10.3389/fgene.2012.00176
7. Pulmonary artery pressure and iron deficiency in patients with upregulation of hypoxia sensing due to homozygous VHL(R200W) mutation (Chuvash polycythemia).
   Sable CA, Aliyu ZY, Dham N, Nouraie M, et al · · 2012 · cited 22× · PMID 21993671 · DOI 10.3324/haematol.2011.051839
8. Predictors of osteoclast activity in patients with sickle cell disease.
   Nouraie M, Cheng K, Niu X, Moore-King E, et al · · 2011 · cited 13× · PMID 21546502 · DOI 10.3324/haematol.2011.042499