Assessment of Bleeding Severity in Children with Immune Thrombocytopenia (ITP)

Study ID
STU 072011-091

Cancer Related
No

Healthy Volunteers
No

Study Sites

Contact
Anna Winborn
214-456-8185
anna.winborn@childrens.com

Principal Investigator
Adolfo Flores

Summary

Group a: all patients who present for inpatient or outpatient medical care with newly diagnosed or established iTP seen by the Pediatric Hematology service at Children's Medical Center are eligible for participation in this study. Particular emphasis will be placed on children with iTP who are newly diagnosed and/or are having symptomatic relapse of their thrombocytopenic state and have a platelet count less than 50,000 per mm3. Subjects will undergo a simultaneous bleeding score assessment according to five different published scoring instruments (Buchanan and Holtkamp 1984, Bolton-Maggs and Moon 1997, Buchanan and adix 2002, World Health organization 2006, Page et al 2007, see appendix B) on the basis of recent history and physical examination. Moreover, subjects will be evaluated with our new bleeding assessment tool based on hemorrhagic manifestations the child may have experienced within the past 48 hours.
Group B: new or established iTP patients with platelet counts equal to or less than 10,000 per mm3 or moderate to severe bleeding according to the Buchanan and adix bleeding instrument, will have all of the bleeding score measurements as described above in addition to undergoing more specific bleeding assessments. These include a urinalysis with microscopy to examine for red blood cells and RBC casts, fecal occult blood testing using a stool guaiac test and non-contrast magnetic resonance imaging of the brain to detect sub-clinical intracranial bleeding. The implementation of these specific bleeding assessments will be administered regardless of the child's presenting symptom(s) at diagnosis or relapse in an effort to characterize clinically important occult bleeding sites. if a patient with a platelet count [Less Than] 10,000/mm3 and/or moderate to severe bleeding presents to our clinic past the 7 day window period of enrollment, they may still participate in this investigation solely under the MRi component of this study. They will not receive any other tests to characterize occult hemorrhage.
Specific treatment will not be dictated by this study although standard of care recommendations will help guide management decisions. no investigational drugs will be administered as part of this study. Patient identifier information being collected is strictly for future contact by the research team and any patient information used for research will be de-identified prior to analysis or sharing of data with non-research personnel. The identification and enrollment of all participants is described in the protocol.

Sub-Group B, MRi Component: in the event that an eligible subject for participation in this study (meets platelet count criteria and/or has moderate to severe bleeding according to the Buchanan and adix bleeding scale) but is seen in our clinics past the 7 day window period for enrollment, they may still be enlisted to participate as part of an MRi sub-group component. Subjects enrolled to participate in this sub-group will receive a bleeding score as previously described, as well as undergo a non-contrast brain MRi within 3 to 6 months from symptomatic relapse or diagnosis of their iTP to characterize subclinical brain micro-bleeding. This will be the only test that these subjects will undertake. Results will not be disclosed to the patient or to the patient's family or primary treating hematologist as this test is not standard of care.

Participant Eligibility

1) Children >= 12 weeks and <= 18 years of age with newly diagnosed or established ITP seen in the Center for Cancer and Blood Disorders at Children[Single Quote]s Medical Center will be potential research subjects. [Some such patients will also be enrolled in the institution[Single Quote]s prospective ITP cohort study that began during 2010 (IRB # STU082010-073)].

2) Particular emphasis will be placed on children with ITP who are newly diagnosed, established patients who are having symptomatic relapse of their thrombocytopenic state (defined as a decrease in platelet count that causes moderate to severe bleeding according to the Buchanan and Adix bleeding instrument, or administration of therapy in order to achieve hemostasis), and children with chronic ITP all with platelet counts less than 50,000 per mm3 (and in some cases less than 10,000 per mm3).

3) Children who meet eligibility criteria but are seen in our clinic after the 7 day window period for enrollment are still able to participate solely under the MRI subgroup component of this investigation.