scholarly journals Comprehensive Long Term Evaluation of End-Organ Function in Pediatric Patients Undergoing Matched Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease

2013 ◽  
Vol 19 (2) ◽  
pp. S245-S246
Author(s):  
Mari Hashitate Dallas ◽  
David Shook ◽  
Christine Mary Hartford ◽  
Ashok Srinivasan ◽  
Brandon Matthew Triplett ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Hematopoietic Cell Transplantation ◽  
Cell Disease ◽  
Organ Function ◽  
Term Evaluation ◽  
Matched Sibling

Early Identification of Barriers and Facilitators to Self-Management Behaviors in Pediatric Patients With Sickle Cell Disease to Minimize Hematopoietic Cell Transplantation Complications

2018 ◽  
Vol 35 (3) ◽  
pp. 199-209 ◽  
Author(s):  
Ginny L. Schulz ◽  
Rebecca H. Foster ◽  
Valerie Kennedy Lang ◽  
Alison Towerman ◽  
Shalini Shenoy ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Barriers And Facilitators ◽  
Self Management ◽  
Cell Disease ◽  
Management Behaviors

Hematopoietic cell transplantation (HCT) is an elective, curative treatment option for patients with sickle cell disease (SCD). Transplant requires extensive self-management behaviors to be successful. The purpose of this study was to describe potential barriers and facilitators to self-management in a group of pediatric patients with SCD prior to HCT and their medical outcomes post-HCT. A multiple case study approach was used to describe 4 pairs of transplant recipients grouped by age, donor type, and donor source. Each pair included a case with minimal and increased post-HCT complications. Complications included readmissions, graft-versus-host disease, systemic infections, and survival in the first year post-HCT. Variables were retrospectively collected and content analyzed to identify barriers and facilitators within and across pairs using existing self-management frameworks. While higher risk transplants experienced more complications, 3 of the 4 cases with increased complications had a larger number of modifiable barriers identified compared with those experiencing minimal complications. At least one modifiable barrier and multiple facilitators were identified in all cases. A standardized psychosocial assessment process with an established plan to mitigate barriers and promote facilitators to self-management is essential to optimize outcomes in patients with SCD undergoing elective HCT.

scholarly journals Stable Long-Term Donor Engraftment following Reduced-Intensity Hematopoietic Cell Transplantation for Sickle Cell Disease

2008 ◽  
Vol 14 (11) ◽  
pp. 1270-1278 ◽  
Author(s):  
Lakshmanan Krishnamurti ◽  
Sandhya Kharbanda ◽  
Melinda A. Biernacki ◽  
Wandi Zhang ◽  
K. Scott Baker ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Cell Disease ◽  
Reduced Intensity

Matched Sibling Donor Hematopoietic Cell Transplantation for Sickle Cell Disease Using a Reduced Intensity Conditioning Regimen Can Lead To Stable Long Term Engraftment.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 3172-3172 ◽  
Author(s):  
Lakshmanan Krishnamurti ◽  
Catherine Wu ◽  
K. Scott Baker ◽  
John E. Wagner
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Conditioning Regimen ◽  
Cell Disease ◽  
Reduced Intensity Conditioning ◽  
Sibling Donor ◽  
Matched Sibling ◽  
Reduced Intensity

Abstract Hematopoietic Cell Transplantation (HCT) from sibling donors has been demonstrated to cure sickle cell disease. Concerns about high regimen related toxicity particularly in older patients and in those with advanced organ damage and the potential for late sequelae such as chronic graft-versus-host disease (GVHD) and infertility have limited the applicability of HCT. HCT following a reduced intensity conditioning (RIC) regimen has the potential for reducing toxicity and making this curative therapy more acceptable and applicable to this group of patients. We report preliminary results on the first five patients enrolled on a pilot study to evaluate the safety and efficacy of HCT following a RIC regimen for patients with high risk sickle cell disease. All patients received bone marrow from a matched sibling donor. The conditioning regimen consisted of Busulfan 2 mg/kg orally q12 hr x 2 days (0.8mg/kg IV q 6 hr x 2 days for patient#3, 4, 5), Fludarabine 35 mg/m2/dose IV daily x 5 days, anti-thymocyte globulin 30 mg/kg/dose IV daily x 5 days and total lymphoid irradiation 500 cGy with shielding of the liver, lungs, heart, and gonads. GVHD prophylaxis consisted of cyclosporine A and Mycophenolate mofetil. Clinical characteristics, outcomes and donor chimerism in peripheral blood genomic DNA and of subjects are summarized in the Table.1 The preparative regimen was well tolerated with no serious infections or mucositis in any patient. No patient has had recurrence of previous sickle cell related symptoms. Lineage-specific chimerism analysis (patients # 3, 4, 5), reveal predominance of donor erythropoiesis (Table 2). These findings indicate that HCT for sickle cell disease following a RIC regimen is well tolerated and can lead to stable long term engraftment. Clinical Characteristics of patients with sickle cell disease undergoing HCT from a matched sibling donor following a reduced intensity conditioning regimen PIN. Age, Indication Follow-up (days) Regimen Related Toxicity ANC<500(days) GVHD Acute or Chronic % Engraftment Day 100 % Engraftment Day180 % Engraftment Day 365 1. 8 yrs. Stroke, allosensitization 2100 None 7 None 89 100 100 2. 8 yrs. Repeated ACS 1800 None 8 Grad II Skin 75 81 81 3. 6yrs. Repeated ACS 750 None 9 None 75 85 81 4. 8 years Repeated ACS 295 None 14 None 79 71 - 5.18 yrs. Stroke, allosensitization 288 Mild 13 None 100 100 - Lineage Specific Chimerism PIN, Genotype Donor Genotype % donor erythroid day 100 % donor erythroid day 180 % Donor Lymphoid day 100 % Donor Lymphoid day 180 % Hemoglobin S Day 100 % Hemoglobin S Day 180 3. Hb SS Sickle trait 100 100 30 35 30 34 4. HbS/β Thalassemia β Thalassemia trait 100 81 33 58 4 3 5. HbSS Sickle trait 100 100 100 100 32 34

scholarly journals Hematopoietic cell transplantation for sickle cell disease: updates and future directions

Hematology ◽  
2021 ◽  
Vol 2021 (1) ◽  
pp. 181-189
Author(s):  
Lakshmanan Krishnamurti
Keyword(s):  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Ex Vivo ◽  
Autologous Transplantation ◽  
Hematopoietic Cell ◽  
Cell Disease ◽  
The Impact

Abstract Excellent outcomes in hematopoietic cell transplantation (HCT) from HLA-identical siblings, improvements in conditioning regimens, novel graft-versus-host disease prophylaxis, and the availability of alternative donors have all contributed to the increased applicability and acceptability of HCT for sickle cell disease (SCD). In young children with symptomatic SCD with an available HLA-identical related donor, HCT should be carefully considered. HCT from alternative donors is typically undertaken only in patients with severe symptoms, causing or likely to cause organ damage, and in the context of clinical trials. Patients undergoing HCT for SCD require careful counseling and preparation. They require careful monitoring of unique organ toxicities and complications during HCT. Patients must be prospectively followed for a prolonged time to determine the long-term outcomes and late effects of HCT for SCD. Thus, there is a need for a universal, longitudinal clinical registry to follow patients after HCT for SCD in conjunction with individuals who do not receive HCT to compare outcomes. Antibody-based conditioning and ex-vivo umbilical cord blood expansion are likely to improve the availability and acceptability of HCT. In addition, new disease-modifying drugs and the emerging option of the autologous transplantation of gene-modified hematopoietic progenitor cells are likely to expand the available therapeutic options and make decision-making by patients, physicians, and caregivers even more complicated. Future efforts must also focus on determining the impact of socioeconomic status on access to and outcomes of HCT and the long-term impact of HCT on patients, families, and society.

Decision-making in pediatric hematopoeitic cell transplantation

2018 ◽  
Author(s):  
◽  
Ginny L. Schulz
Keyword(s):  
Decision Making ◽  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Family Members ◽  
Real Life ◽  
Decisional Conflict ◽  
Cell Disease ◽  
Decision Choice

[ACCESS RESTRICTED TO THE UNIVERSITY OF MISSOURI AT AUTHOR'S REQUEST.] Hematopoietic cell transplantation (HCT) is a treatment option utilized to cure many childhood disorders, with cancer, sickle cell disease (SCD), and aplastic anemia being a few. The decision of whether or not to pursue HCT involves uncertainty and risk. Pediatric patients with SCD and their families undergo a decision-making process distinct from patients with childhood cancer. Patients with sickle cell disease (SCD) realize an awareness of unpredictable disease progression and conflict when considering HCT, while patients with cancer perceive an urgency for survival. We applied Connor's Framework of Decisional Conflict and a multiple-case study approach to describe the level and source of decisional conflict in patients with SCD and their families in a real-life decision choice of HCT. The contribution of the pediatric patient in the decision was also described in our four families. Observation, survey, and interview methods allowed us to gain an in-depth understanding of their conflict. Through pattern-matching and cross-case synthesis, we found that the level and sources of conflict among 11 participants varied within and across family members. Some experienced levels that resulted in a delayed decision or feeling unsure. The patients and their family members ultimately navigated their uncertainties together to come to consensus on whether to proceed with HCT. Future studies should implement individualized interventions from a family research perspective to decrease the decisional conflict this population faces.

scholarly journals How I treat sickle cell disease with hematopoietic cell transplantation

Blood ◽  
2019 ◽  
Vol 134 (25) ◽  
pp. 2249-2260 ◽  
Author(s):  
Elizabeth O. Stenger ◽  
Shalini Shenoy ◽  
Lakshmanan Krishnamurti
Keyword(s):  
Decision Making ◽  
Stem Cell ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Cell Disease ◽  
Hematopoietic Stem

Hematopoietic stem cell transplantation can be curative for sickle cell disease, but decision-making is often complex. This How I Treat provides a perspective on how to negotiate this process for an individual patient.

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