scholarly journals The Use of Biologic Modifiers as a Bridge to Hematopoietic Cell Transplantation in Primary Immune Regulatory Disorders

2021 ◽  
Vol 12 ◽  
Author(s):  
Danielle E. Arnold ◽  
Deepak Chellapandian ◽  
Jennifer W. Leiding
Keyword(s):  
Hematopoietic Cell Transplantation ◽  
Clinical Manifestations ◽  
Hematopoietic Cell ◽  
Cell Transplant ◽  
Hematopoietic Cell Transplant ◽  
Inborn Errors ◽  
Immune Mediated ◽  
Definitive Therapy ◽  
Regulatory Disorders

Recently, primary immune regulatory disorders have been described as a subset of inborn errors of immunity that are dominated by immune mediated pathology. As the pathophysiology of disease is elucidated, use of biologic modifiers have been increasingly used successfully to treat disease mediated clinical manifestations. Hematopoietic cell transplant (HCT) has also provided definitive therapy in several PIRDs. Although biologic modifiers have been largely successful at treating disease related manifestations, data are lacking regarding long term efficacy, safety, and their use as a bridge to HCT. This review highlights biologic modifiers in the treatment of several PIRDs and there use as a therapeutic bridge to HCT.

scholarly journals ALLOGENEIC HEMATOPOIETIC CELL TRANSPLANTATION IN THE TREATMENT OF CHRONIC LYMPHOCITIC LEUKEMIA : WHY AND WHEN ?

2010 ◽  
Vol 2 (2) ◽  
pp. e2010018 ◽  
Author(s):  
Maria L. Delioukina ◽  
Stephen J. Forman
Keyword(s):  
Cell Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
Hematologic Malignancy ◽  
Lymphocytic Leukemia ◽  
Hematopoietic Cell ◽  
New Drugs ◽  
Cell Transplant ◽  
Hematopoietic Cell Transplant ◽  
Purine Analogues

Chronic lymphocytic leukemia (CLL) is the most common hematologic malignancy in adults with an incidence rate of 4.2 per 100,000 per year. CLL frequently takes an indolent course, with some patients not requiring treatment for years, yet is incurable by currently available chemo- and immuno-therapeutic modalities. Despite high initial response rates, particularly to purine analogues, patients invariably relapse and subsequently develop resistance to therapy. The traditional “watchful waiting” approach to CLL is being challenged by data showing that treatments used early in the disease course impact long-term overall and progression-free survivals . The only curative treatment for CLL currently, is allogeneic hematopoeietic cell transplantation (alloHCT). In contrast to autologous transplant, myeloablative alloHCT for CLL patients generates durable remissions with promising survival plateaus; however, significant transplant related mortality (TRM) is also observed (25-50%) . At present the fact remains that for poor-risk CLL, alloHCT is the only treatment with the potential of providing long-term disease control. Future combinations with emerging low-toxicity therapies may further enhance the curative potential of allogeniec hematopoietic cell transplant. New drugs can also potentially enable refractory patients to attain response as a bridge to more effective stem cell transplantation.

Neuropsychologic changes from before transplantation to 1 year in patients receiving myeloablative allogeneic hematopoietic cell transplant

Blood ◽  
2004 ◽  
Vol 104 (10) ◽  
pp. 3386-3392 ◽  
Author(s):  
Karen L. Syrjala ◽  
Sureyya Dikmen ◽  
Shelby L. Langer ◽  
Sari Roth-Roemer ◽  
Janet R. Abrams
Keyword(s):  
Hematopoietic Cell Transplantation ◽  
Population Based ◽  
Hematopoietic Cell ◽  
Cell Transplant ◽  
Neurocognitive Performance ◽  
Long Term Effects ◽  
Hematopoietic Cell Transplant ◽  
Allogeneic Hematopoietic Cell Transplant ◽  
Neuropsychologic Testing

Abstract Research indicates that myeloablative hematopoietic cell transplantation (HCT) impairs neurocognitive function. However, prospective studies on long-term effects are lacking. This longitudinal study examined neurocognitive changes over the first year in 142 adult recipients of allogeneic HC transplants who received neuropsychologic testing before transplantation and again after 80 days and 1 year. Age-, sex-, and education-adjusted population-based standardized scores were used for normative comparisons. Performance on all tests declined from before transplantation to 80 days (P < .05) and improved by 1 year (P < .05), returning to pretransplantation levels on all tests except for grip strength and motor dexterity. Although verbal fluency and memory recovered by 1 year, both were below norms at all 3 testing times (P < .01). Logistic regressions indicated that patients without chemotherapy, other than hydroxyurea, previous to HCT and patients not receiving chronic graft-versus-host disease (GVHD) medication at 1 year had lower risk of impaired function (P < .05). In conclusion, HCT was associated with significant generalized decline in neurocognitive performance at 80 days, with subsequent recovery to pretransplantation levels by 1 year for most survivors, except on motor tasks. Results indicate that long-term cognitive decrements, as distinct from motor disabilities, infrequently derive directly from HCT. (Blood. 2004;104:3386-3392)

scholarly journals Inadequate survivorship care after allogeneic hematopoietic cell transplantation: A retrospective chart review

F1000Research ◽  
2018 ◽  
Vol 7 ◽  
pp. 1389 ◽  
Author(s):  
Sunn Sunn Thaw ◽  
Shernan Holtan ◽  
Qing Cao ◽  
Michael Franklin ◽  
Nyan Paye ◽  
...  
Keyword(s):  
Hematopoietic Cell Transplantation ◽  
Retrospective Chart Review ◽  
Hematopoietic Cell ◽  
Cell Transplant ◽  
Survivorship Care ◽  
Hematopoietic Cell Transplant ◽  
Organ Systems ◽  
Care Guidelines

Background: Hematopoietic cell transplant (HCT) survivors are at risk of developing long-term complications. Guidelines for survivorship care of HCT recipients were published in 2012; however, the degree to which these guidelines are incorporated into clinical practice is unknown. The purpose of this study was to determine whether providers utilize the 2012 guidelines and analyze whether survivorship-focused providers, provider gender, or provider year of practice influenced adherence to these guidelines. Methods: Adult allogeneic HCT recipient’s medical records were reviewed at the University of Minnesota between 2010 and 2012; only patients who survived without relapse to their 2-year follow-up visit after HCT were included. A semi-quantitative scoring system was developed providing 1 point for each of the 13 organ systems assessed by the 2012 survivorship care guidelines. Data was collected on history, clinical exam, laboratory tests, preventive measures, and counseling. The primary endpoint was the overall score for adherence to the survivorship care guidelines. Wilcoxon rank-sum tests for continuous and Chi-square tests for categorical factors were used to compare the overall score between provider groups (survivorship-focused providers vs others), provider gender, and provider year of practice (≥10 years vs <10 years). Results: Fifteen providers (9 male, 3 survivorship-focused, 7 with <10 years of practice) provided follow-up care to 77 HCT survivors. Survivorship-focused providers had a higher median overall score than other providers (median 10 vs 8, p<0.01).  Female providers had a higher median overall score than male providers (median 9.0 vs 8, p<0.01). There was no difference in median overall score based on provider year of experience (p=0.43). Conclusions: In conclusion, survivorship-focused providers were more likely to achieve long-term screening recommendations. However, even within this group, adherence to the 2012 screening and preventive practice guidelines was incomplete.  Further efforts to automate and standardize the survivorship assessments in HCT survivors are necessary.

scholarly journals Management of Herpesvirus Infections in Hematopoietic Cell Transplant Recipients

2021 ◽  
Vol 2 (1) ◽  
pp. 8-21
Author(s):  
Jan Styczynski
Keyword(s):  
Cell Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
Cellular Therapy ◽  
Hematopoietic Cell ◽  
Host Cells ◽  
Preventive Strategy ◽  
Cell Transplant ◽  
Hematopoietic Cell Transplant ◽  
Screening Assay ◽  
Specific Level

Following primary infection, herpesviruses establish latency in infected individuals in the host cells and may reactivate upon external stimuli and during periods of immunosuppression. The objective of this paper was to the present current strategies on preventive and therapeutic management of infections with herpesviruses in recipients of hematopoietic cell transplantation. Strategies of antiviral management include prophylaxis, pre-emptive treatment and targeted treatment. Empirical therapy is not used in antiviral strategies. Prophylaxis can be done at universal (preventive strategy) and specific level. Universal prophylaxis includes non-pharmacologic methods of prevention of infection or reactivation. Risk-adapted specific prophylaxis includes use of specific antivirals or cellular therapy or other specific methods in order to prevent specific infection, in high-risk groups. Pre-emptive therapy means use of therapeutic approaches in asymptomatic infection, detected by a screening assay. Targeted therapy is used in established specific viral end-organ infections. The following sections of the paper refer to prophylaxis and treatment strategies, respectively, against CMV, EBV, HSV, VZV, HHV-6, HHV-7, and HHV-8 after allogeneic hematopoietic cell transplantation.

scholarly journals The Hematopoietic Cell Transplant Comorbidity Index predicts survival after allogeneic transplant for nonmalignant diseases

Blood ◽  
2019 ◽  
Vol 133 (7) ◽  
pp. 754-762 ◽  
Author(s):  
Monica S. Thakar ◽  
Larisa Broglie ◽  
Brent Logan ◽  
Andrew Artz ◽  
Nancy Bunin ◽  
...  
Keyword(s):  
Hematopoietic Cell Transplantation ◽  
Cox Regression ◽  
Bone Marrow Failure ◽  
Hematopoietic Cell ◽  
Cell Transplant ◽  
Research Database ◽  
Hematopoietic Cell Transplant ◽  
Risk Of Death ◽  
Increased Risk ◽  
Comorbidity Index

Abstract Despite improvements, mortality after allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases remains a significant problem. We evaluated whether pre-HCT conditions defined by the HCT Comorbidity Index (HCT-CI) predict probability of posttransplant survival. Using the Center for International Blood and Marrow Transplant Research database, we identified 4083 patients with nonmalignant diseases transplanted between 2007 and 2014. Primary outcome was overall survival (OS) using the Kaplan-Meier method. Hazard ratios (HRs) were estimated by multivariable Cox regression models. Increasing HCT-CI scores translated to decreased 2-year OS of 82.7%, 80.3%, 74%, and 55.8% for patients with HCT-CI scores of 0, 1 to 2, 3 to 4, and ≥5, respectively, regardless of conditioning intensity. HCT-CI scores of 1 to 2 did not differ relative to scores of 0 (HR, 1.12 [95% CI, 0.93-1.34]), but HCT-CI of 3 to 4 and ≥5 posed significantly greater risks of mortality (HR, 1.33 [95% CI, 1.09-1.63]; and HR, 2.31 [95% CI, 1.79-2.96], respectively). The effect of HCT-CI differed by disease indication. Patients with acquired aplastic anemia, primary immune deficiencies, and congenital bone marrow failure syndromes with scores ≥3 had increased risk of death after HCT. However, higher HCT-CI scores among hemoglobinopathy patients did not increase mortality risk. In conclusion, this is the largest study to date reporting on patients with nonmalignant diseases demonstrating HCT-CI scores ≥3 that had inferior survival after HCT, except for patients with hemoglobinopathies. Our findings suggest that using the HCT-CI score, in addition to disease-specific factors, could be useful when developing treatment plans for nonmalignant diseases.

scholarly journals Lung Function and Long-Term Complications after Allogeneic Hematopoietic Cell Transplant

2010 ◽  
Vol 16 (1) ◽  
pp. 53-61 ◽  
Author(s):  
Eric C. Walter ◽  
Mauricio Orozco-Levi ◽  
Alba Ramirez-Sarmiento ◽  
Afonso Vigorito ◽  
Paulo V. Campregher ◽  
...  
Keyword(s):  
Lung Function ◽  
Hematopoietic Cell ◽  
Cell Transplant ◽  
Hematopoietic Cell Transplant ◽  
Allogeneic Hematopoietic Cell Transplant
Sign in / Sign up

Export Citation Format

Share Document