Sickle Cell Transplantation Evaluation of Long-term and Late Effects Registry (STELLAR) to compare long-term outcomes after hematopoietic cell transplantation to those in siblings without sickle cell disease and in nontransplanted individuals with sickle cell disease: Design and feasibility study

BACKGROUND: There are sparse data on the long-term and late effects of hematopoietic cell transplantation (HCT) for sickle cell disease (SCD). OBJECTIVE: This study aims to establish an international registry of long-term outcomes post-HCT for SCD and demonstrate the feasibility of recruitment at a single site in the United States. METHODS: The Sickle Cell Transplantation Evaluation of Long-Term and Late Effects Registry (STELLAR) was designed to enroll patients with SCD ≥1 year post-HCT, their siblings without SCD, and nontransplanted controls with SCD to collect web-based participant self-reports of health status and practices by using the Bone Marrow Transplant Survivor Study (BMTSS) surveys, health-related quality of life (HRQOL) using the Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Profile-25 or Pediatric Profile-29 survey, chronic graft-versus-host disease (cGVHD) using the symptom scale survey, daily pain using an electronic pain diary, the economic impact of HCT using the financial hardship survey, sexual function using the PROMIS Sexual Function SexFSv2.0 survey, and economic productivity using the American Time Use Survey (ATUS). We also piloted retrieval of clinical data previously submitted to the Center for International Blood and Marrow Transplant Research (CIBMTR); recorded demographics, height, weight, blood pressure, waist and hip circumferences, timed up and go (TUG) test, and handgrip test; and obtained blood for metabolic screening, gonadal function, fertility potential, and biorepository of plasma, serum, RNA, and DNA. RESULTS: Of 100 eligible post-HCT patients, we enrolled 72 (72%) participants aged 9-38 (median 17) years. We also enrolled 19 siblings aged 5-32 (median 10) years and 28 nontransplanted controls with SCD aged 4-46 (median 22) years. Of the total 119 participants, 73 (61%) completed 85 sets of surveys and 41 (35%) contributed samples to the biorepository. We completed ATUS interviews of 28 (24%) participants. We successfully piloted retrieval of data submitted to the CIBMTR and expanded recruitment to multiple sites in the United States, Canada, the United Kingdom, and Nigeria. CONCLUSIONS: It is feasible to recruit subjects and conduct study procedures for STELLAR in order to determine the long-term and late effects of HCT for SCD. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/36780

Quantification of variation in biological input materials and its effect on manufacture and process control

Currently cellular therapies, such as hematopoietic stem cell transplantation (HSCT), are produced at a small scale on a case-by-case basis, usually in a clinical or near-clinical setting. Meeting the demand for future cellular therapies will require a robust and scalable manufacturing process that is either designed around or controls the variation associated with biological starting materials. Understanding variation requires both a measure of the allowable variation (that does not negatively affect patient outcome) and the achievable variation (with current technology). The prevalence of HSCT makes it an ideal case study to prepare for more complex biological manufacturing with more challenging regulatory classifications

The EBMT Handbook

This Open Access edition of the European Society for Blood and Marrow Transplantation (EBMT) handbook addresses the latest developments and innovations in hematopoietic stem cell transplantation and cellular therapy. Consisting of 93 chapters, it has been written by 175 leading experts in the field. Discussing all types of stem cell and bone marrow transplantation, including haplo-identical stem cell and cord blood transplantation, it also covers the indications for transplantation, the management of early and late complications as well as the new and rapidly evolving field of cellular therapies. This book provides an unparalleled description of current practices to enhance readers’ knowledge and practice skills

Blood As a Biological Drug : Scientific, Legal, and Policy Issues in the Regulation of Placental and Umbilical Cord Stem Cell Transplantation

Not all blood cells are created equal. Some are born, carry out their appointed tasks-red blood cells oxygenating the blood, white blood cells fighting infection-and die. But an elusive subset have special properties: they are the progenitors of all the many types of peripheral (circulatory) blood cells, and as such, they have the potential to reconstitute an entire blood supply. Known as hematopoietic stem cells, these blood cells reproduce indefinitely. For patients with leukemia or other blood diseases, the transplantation of hematopoietic stem cells from another person\u27s bone marrow can provide the gift of life

Cord Blood Collection in Pregnant Women for Stem Cell Research

Umbilical cord blood (UCB) stem cells form commonly banked types of human tissue. Confusion remains about sources of stem cells and their use. Birth is a once in a lifetime opportunity with 74 million births per year in the world and four million occur in the United States. Cord blood contains hematopoietic stem cells and pluripotent mesenchymal cells (Moise, 2005). There is a surge of interest in the clinical use and research investigation of umbilical cord blood for transplantation and regenerative medicine. Clinicians need increased awareness and education of options for private versus public donation and banking of cord blood at birth to present to pregnant patients. The aims of this research were to describe decision-making readiness and communication with mothers and their partners during her pregnancy using the Transtheoretical Model (Prochaska, J.O. and DiClemente, C.C, 1982). Describe the relationship between age, pregnant women\u27s level of parity, gestational age, highest level of education, marital status, race, religion, economic status, and plan of payment for prenatal care to their sources of knowledge, their beliefs and knowledge regarding cord blood transplantation. Perceptions of pregnant women\u27s cord blood use and their understanding of the difference between public and private cord blood storage banks and use. The partner\u27s perceptions of cord blood use and their understanding of the difference between public and private cord blood storage banks and use. The voluntary sample consisted of 30 participants of convenience from two obstetrical clinics from a local clinic. The design was a quantitative method staging an algorithm for Cord Blood Donation using a Decision of Readiness Scale along with notification of significant other. The Cord Blood Survey measured sources of knowledge, beliefs, and knowledge of cord blood transplantation. The Statistical Package for the Social Sciences (SPWS 18.0) was used to perform an analysis of data. Descriptive statistics were used to summarize the demographic variables and to identify central tendency, variability and percentages of key variables. One-way ANOVA and t-test were computed on the quantitative questions. Qualitative measures from randomly selected post interviews of 1/3 of the sample were completed. The barriers to understanding what umbilical cord blood donation options and how pregnant women make decisions regarding umbilical cord blood donations are explored. The implications for nursing research are: overcoming the barriers to understanding the difference between private and public banking and the ramifications of each option; add to nursing knowledge by understanding when and how women make decisions about umbilical cord blood donation; and future studies could investigate ethical implications of umbilical cord blood donation and best nursing practice guidelines for approaching pregnant women and their partners on umbilical cord blood donation options