Clinical trial NCT01629017: Long Term Effects On Recipients of Hematopoietic Stem Cell Transplantation
Official title: Longitudinal Evaluation of Recipients of Hematopoietic Stem Cell Transplantation (HSCT)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Blood and Marrow Transplant (BMT)
Lead sponsor: Stanford University
Sponsor class: OTHER
First posted: 2012-06-27
Enrollment: 800
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Stanford Blood and Marrow Transplant clinic.

Brief summary: This project allows for the systematic collection and analysis of long-term follow-up clinical parameters in children who have received a stem cell transplant. The data collected will assist in determining appropriate intervention and treatment plans for patients enrolled on this study. In addition, future patients may benefit by having the ability to anticipate problems and develop methods of prevention or early intervention.

Detailed description: Primary Objective: Implement long-term follow-up guidelines for children, adolescents and young adults who are survivors of HSCT while establishing a database which captures the survivor's lifetime care.

Secondary Objectives:

* Collect evidence-based data through a structured framework of periodic physical examination, laboratory and medical tests to monitor late effects in our HSCT survivors.
* Establish standards of care for late effects of therapy post HSCT for malignant and non-malignant diagnoses.
* Provide early identification and intervention towards recognized problems, as well as consistent education to foster a healthy lifestyle to all survivors.
* Collect blood and tissue samples to study immune reconstitution of all immune cell types and learn more about immune reconstitution and its affect on post-HSCT complications and disease free survival.

Eligibility criteria: Inclusion Criteria:

* Participant is scheduled to receive OR has received a hematopoietic stem cell transplant (HSCT) for any condition.
* Participant may have had a HSCT in another institution than Lucile Packard Children's Hospital (LPCH) yet has transferred follow-up long-term care to our institution and is being seen through the late-effects clinic from the Pediatric HSCT program.
* Participant AND/OR parent/legally authorized representative is able to provide written informed consent and assent (as applicable) for participation.

Exclusion Criteria:

* Participant has relapsed from a malignant diagnosis post HSCT and is not being worked-up for a new HSCT.
* Participant or patient's authorized guardian is unable to provide consent and assent

Primary outcomes:
measure: Collection of data and tissue samples with the goal of establishing a systematic follow-up care for HSCT recipients and a comprehensive database which can capture the particularities of survivors' clinical status through their life span.
timeFrame: This research study is expected to continue as long as we continue to perform clinical trials for children with diseases that involve the bone marrow or are affected by bone marrow production.
Secondary outcomes:
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford University School of Medicine
state: California
Study officials:
affiliation: Stanford University
name: Ami Shah
role: PRINCIPAL_INVESTIGATOR
References: