Comparison of Outcomes and Quality-of-Life Measures Following Haploidentical Vs. Matched Related/Unrelated Donor Allogeneic Hematopoietic Cell Transplantation

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 16-17
Author(s):  
Sanghee Hong ◽  
Lisa Rybicki ◽  
Linda McLellan ◽  
Jane Dabney ◽  
Aaron T. Gerds ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cell Transplantation ◽  
Research Funding ◽  
Hematopoietic Cell Transplantation ◽  
Well Being ◽  
Hematopoietic Cell ◽  
Unrelated Donor ◽  
Post Transplant ◽  
The Mean

Background: Haploidentical (haplo) transplantation using post-transplant cyclophosphamide (Cy) based GVHD prophylaxis is a well-established alternative donor approach for allogeneic hematopoietic cell transplantation (HCT). Although outcomes from haplo HCT have been compared to matched related (MRD) and unrelated donor (MUD) allo HCT, quality-of-life (QOL) recovery between these transplant modalities has not been described. We hypothesized that post-transplant QOL is similar between Haplo and MRD/MUD grafts. A single-institution, retrospective cohort study was therefore conducted to compare outcomes including QOL between these groups. Methods: From May 2014 through December 2019, 90 haplo, 102 MRD and 229 MUD adult 1st allo HCTs were performed. Myeloablative conditioning (MAC) for haplo grafts (n=35) included fludarabine (Flu)/ total body irradiation (TBI) and reduced-intensity conditioning (RIC) haplo transplants (n=55) included Flu/Cy/TBI. QOL was assessed prior to transplant and at days +100 and +180 by the FACT-BMT which consisted of the following subdomains: "Physical well-being," "Social well-being," "Emotional well-being," "Functional well-being," "Additional concerns," and sum of all these subdomains as "Total score." Outcomes were estimated and compared with the Kaplan-Meier and log-rank test or cumulative incidence and Gray test. FACT-BMT scores were compared with repeated measures analysis of variance. Results: Patient and transplant characteristics were comparable among the donor sources except for higher median age at HCT in MUD transplants, and haplo grafts were more commonly used non-Caucasians, used RIC regimens and bone marrow grafts (Table 1). Median transplant hospital length of stay was longer for haplo vs. MRD vs. MUD grafts (31 vs.25 vs. 25 days, p<0.001) with longer median time until neutrophil and platelet recovery for haplo compared to MRD and MUD grafts (20 vs. 14 vs. 15 days, p<0.001, and 31 vs. 21 vs. 22 days, p<0.001, respectively). MAC haplo HCT had a higher incidence of CMV (p=0.05) and other infections (p=0.003), but no difference in other outcomes compared to MRD/MUD grafts, including all of the QOL domains. The mean Total scores from baseline to day +180 for haplo HCT were 157-157, MRD 153-166, and MUD HCT 152-163. RIC haplo HCT was associated with higher incidences of infections (p<0.001) and relapse mortality (p=0.04), but lower NRM (p=0.008) and no differences in other outcomes. When comparing RIC haplo to MUD transplants there was significantly better Emotional well-being (p=0.008) and Functional well-being (p=0.011) at day+180; respective mean emotional well-being score for haplo 19-23 and MUD 16-19; mean functional well-being score for haplo 19-24 and MRD 19-19 (Figures 1a and 1b). No difference for the other QOL measures were found; the mean Total scores from baseline to day +180 for haplo HCT were 155-180, and MUD HCT were 150-158 (p=0.25). Discussion: Among MAC recipients, we observed no differences in total FACT QOL scores and individual domains among haplo, MRD and MUD HCT recipients through 6 months post-HCT. After RIC HCT, haplo recipients reported better Emotional and Functional well-being compared to those receiving a MUD graft, although Total FACT score and other QOL domains were comparable through 6 months. Future strategies to lessen infectious complications and relapse may further improve QOL outcomes. Interrogation of QOL among disease specific groups may also elucidate whether additional benefits exist between these different transplant modalities. Disclosures Gerds: Sierra Oncology: Research Funding; Gilead Sciences: Research Funding; Celgene: Consultancy, Research Funding; Imago Biosciences: Research Funding; Roche/Genentech: Research Funding; Incyte Corporation: Consultancy, Research Funding; CTI Biopharma: Consultancy, Research Funding; Apexx Oncology: Consultancy; AstraZeneca/MedImmune: Consultancy; Pfizer: Research Funding. Hamilton:Syndax Pharmaceuticals: Consultancy, Honoraria. Majhail:Nkarta Therapeutics: Honoraria; Incyte: Honoraria; Mallinckrodt: Honoraria; Anthem, Inc.: Consultancy.

scholarly journals Long-term recovery after hematopoietic cell transplantation: predictors of quality-of-life concerns

Blood ◽  
2010 ◽  
Vol 115 (12) ◽  
pp. 2508-2519 ◽  
Author(s):  
F. Lennie Wong ◽  
Liton Francisco ◽  
Kayo Togawa ◽  
Alysia Bosworth ◽  
Mitzi Gonzales ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cell Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
Chronic Gvhd ◽  
Well Being ◽  
Primary Diagnosis ◽  
Hematopoietic Cell ◽  
Prospective Longitudinal Study ◽  
Prospective Longitudinal

This prospective longitudinal study examined the quality of life (QOL) after hematopoietic cell transplantation (HCT) and identified risk factors of poor QOL in 312 adult autologous and allogeneic HCT patients. Physical, psychological, social, and spiritual well-being was assessed before HCT, 6 months, and 1, 2, and 3 years after HCT. For all HCT patients, physical QOL was stable from before to after HCT (P > .05); psychologic (P < .001), social (P < .001), and spiritual (P = .03) QOL improved at 6 months. Study noncompleters (because of illness or death) had worse QOL. Allogeneic patients reported worse physical and psychologic well-being (P < .05). Older patients reported worse physical but better social well-being regardless of HCT type (P < .05). Two or more domains were affected by race/ethnicity, household income, and education in autologous patients, and by body mass index (BMI), decline in BMI, primary diagnosis, and chronic graft-versus-host disease (GVHD) in allogeneic patients (P < .05). At 3 years, 74% of HCT patients were employed full or part time. Older autologous patients with lower pre-HCT income were less likely to work (P < .05); allogeneic patients with chronic GVHD were less likely to work (P = .002). Multidisciplinary efforts to identify and support vulnerable subgroups after HCT need to be developed.

scholarly journals Comparison of the Impact of Pre-Transplant Co-Morbidity Scores on Allogeneic Hematopoietic Stem Cell Transplant Outcomes

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 4670-4670
Author(s):  
Mohammed A. Marei ◽  
Eshetu G Atenafu ◽  
Arjun Law ◽  
Wilson Lam ◽  
Rajat Kumar ◽  
...  
Keyword(s):  
Cell Transplantation ◽  
Research Funding ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Cell Transplant ◽  
Post Transplant ◽  
Transplant Outcomes ◽  
C Statistic ◽  
Co Morbidity ◽  
The Impact

Abstract Introduction: Allogeneic hematopoietic cell transplantation (allo-HCT) is potentially curative for the treatment of various hematological diseases, in part due to the effect of conditioning chemotherapy, and in part due to graft-versus-malignancy effect. However, alloHCT is associated with significant morbidity and mortality. Multiple co-morbidity indices have been published in the literature for the purpose of pre-transplant risk assessment. The purpose of the presented study is to assess a number of these pre-transplant scores on a single-center transplant population and to determine the score with improved risk stratification ability using concordance statistics. Methods: We investigated the impact of the prospectively collected Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) on post-transplant outcomes for 243 recipients of allo-HCT performed between August 2014 and October 2016 at the Princess Margaret Cancer Center (Toronto, Canada), and compared this score to other pre-transplant scores including the age-adjusted HCT-CI, PAM score (Pre-transplant Assessment of Mortality Score) and the Disease Risk Index (DRI). Partitioning of the HCT-CI, HCT-CI/age and PAM scores into three groups was performed based on maximum significant differences on univariate analysis for overall survival (OS). Concordance statistics were used to compare the stratification power of the scores. Statistical analyses were performed using SAS version 9.4 (SAS Institute, Inc, Cary, NC). Results: The median age at transplant is 56 years, patients were transplanted for AML (53%), ALL (7.5%), MDS (13.5%), MPN (14%), NHL/CLL (8.5%) and (3.5%) AA. Donors were matched related in 37%, unrelated in 59% and haploidentical in 3% of the patients. Reduced intensity conditioning chemotherapy was used in 132 patients (54%), 153 patients (63%) received in-vivo T-cell depletion by using Campath or ATG, both donor and recipient were CMV negative in 48 (20%) of the patients. DRI was high in 67 (29%), intermediate in 145 (62%) and low in 22 (9%) of patients. HCT-CI was 0 in 90 (37%), 1 in 49(21%) and ≥2 in 103 (43%) of patients. HCT-CI/age was 0 in 22 (10%), 1 in 72 (30%) and ≥2 in 148 (62%). PAM score was 1-17 in 157(68%), 18-24 in 70 (30%) and 25-27 in 7 (3%) of patients. Median follow up of survivors was 28 months (range 17-44 months). OS of the entire cohort was 51% and 43% at 2 and 5 years post-transplant respectively. Cumulative incidence of relapse (CIR) was 19% at 2 years. For OS, as grouped above, the DRI did not demonstrate a significant difference between groups (p=0.77). For HCT-CI, p=0.034 (Figure 1), for HCT-CI/age p=0.02 and for the PAM score p=0.38. For OS, for the DRI, the C-statistic was 0.51 (se=0.03, 95%CI 0.45-0.57). For the PAM score, C-statistic was 0.51 (se=0.02,95%CI 0.45-0.56). For the HCT-CI age, C-statistic was 0.56 (se=0.024, 95%CI 0.51-0.61). For the HCT-CI, C-statistic was 0.56 (se 0.02, 95% CI 0.50-0.61). For CIR, the PAM score demonstrated a superior C-statistic of 0.56 (se=0.06, 95%CI 0.44-0.67) compared to the other scores. For NRM, the HCT-CI score (Figure 2, p=0.039) is superior with C-statistic 0.56 (se=0.04, 95%CI=0.49-0.63). Conclusion: Based on the above described analysis, the original HCT-CI score as described by Sorror et aldemonstrates superior prognostic stratification ability for OS and NRM in our patient cohort compared to other scores. Further investigation for the development of an optimal risk scoring system for allogeneic HCT is required. Figure 1. Figure 1. Disclosures Kim: Paladin: Consultancy; Pfizer: Consultancy; Novartis: Consultancy, Honoraria, Research Funding; BMS: Consultancy, Honoraria, Research Funding. Lipton:Novartis: Consultancy, Honoraria, Research Funding; Pfizer: Consultancy, Honoraria, Research Funding; BMS: Consultancy, Honoraria, Research Funding; Takeda: Consultancy, Honoraria, Research Funding.

scholarly journals Donor IFNL4 Genotype Is Associated with Transplant-Related Mortality after Unrelated Donor Myeloablative Hematopoietic Cell Transplantation in Patients with Acute Leukemia

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 968-968
Author(s):  
Shahinaz M Gadalla ◽  
Tao Wang ◽  
Olusegun Onabajo ◽  
Youjin Wang ◽  
Michael D. Haagenson ◽  
...  
Keyword(s):  
Acute Leukemia ◽  
Cell Transplantation ◽  
Research Funding ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Taqman Assay ◽  
Unrelated Donor ◽  
Karnofsky Performance Score ◽  
Related Mortality

Abstract Introduction. Interferon Lambda 4 gene (IFNL4) encodes IFN-λ4 protein, a new member of the type-III interferon family. IFNL4 genotype (rs368234815-dG allele), defines the genetic ability to produce IFN-λ4 and has been associated with reduced clearance of hepatitis C virus (HCV) infection. Given antiviral activity and immune modulation properties of IFN-λ4, we hypothesized that IFNL4 genotype of recipient and/or donor may modulate post-transplant survival outcomes, possibly through control of viral infections, and/or alloreactivity. Methods. From the Center for International Blood and Marrow Transplant Research (CIBMTR) database, we randomly selected 627 patients who received unrelated hematopoietic cell transplantation (HCT) for acute myeloid leukemia (AML, N=449) or acute lymphocytic leukemia (ALL, N=178). The patients had to match the following criteria: 1) HCT between 2004 and 2012, 2) available pre-HCT blood sample for the donor and recipient, 3) 8/8 HLA matching, and 3) myeloablative conditioning. IFNL4 genotyping was completed for 619 donors and 522 recipients using a custom-designed TaqMan assay for rs368234815. Multivariable Cox proportional hazard models were used for statistical analyses. Follow-up ended in November 2017. Results. Median age at HCT was 40 years (range=<1-68). Most patients (66%, N=411) were in first complete remission, had a Karnofsky Performance Score (KPS) between 90-100% (70%, N=436), and received peripheral blood stem cell grafts (70%, N=439). The median post-HCT follow-up was 68 months (range=5-122). Donor IFNL4 genotype was associated with risk of transplant-related mortality (TRM); with 5 years probabilities=19%, 27%, and 30% for donor TT/TT (n=286), TT/dG (n=267), and dG/dG (n=64) genotypes, respectively, p=0.02. The results remained significant in multivariable analysis (p=0.002); compared with patients receiving HCT from donors with TT/TT genotype, with the HR=1.59 (95% CI=1.13-2.23, p=0.007) for TT/dG donors and HR=1.95 (95% CI=1.18-3.23, p=0.009) for dG/dG donors. The data suggested that donor IFNL4 genotype may also predict risk of disease-free survival (DFS; HR=1.43, 95% CI=1.02-2.00, p=0.03), and overall survival (OS; HR=1.40 (95% CI=0.98-1.99, p=0.06) for donor dG/dG genotype (Table1). No association between recipient genotype and any survival outcome was observed (p>0.05 for OS, DFS, and TRM) Conclusions. Donor IFNL4 genotype is associated with risk of transplant-related mortality in patients with acute leukemia. The data suggest that avoiding donors with dG/dG genotype will improve HCT outcomes without limiting the potential donor pool. A validation study is needed; if confirmed, IFNL4 genotype may provide an added value to donor selection criteria. Disclosures Lee: Onyx: Research Funding; Kadmon: Research Funding; Amgen: Consultancy, Research Funding; Mallinckrodt: Honoraria; Incyte: Consultancy; Pfizer: Consultancy; Takeda: Research Funding.

scholarly journals Validation of Different Prognostic Scores in Allogeneic Hematopoietic Cell Transplantation in the Post-Transplant Cyclophosphamide Era

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3925-3925
Author(s):  
Maria Queralt Salas ◽  
Luis Gerardo Rodríguez-Lobato ◽  
María Suárez-Lledó ◽  
Nuria Martínez-Cibrian ◽  
Teresa Solano ◽  
...  
Keyword(s):  
Cell Transplantation ◽  
Research Funding ◽  
Operating Characteristic ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Prognostic Scores ◽  
Predictive Capacity ◽  
Post Transplant ◽  
Gvhd Prophylaxis

Abstract INTRODUCTION The use of post-transplantation cyclophosphamide (PTCy) for graft-versus-host disease (GvHD) prophylaxis has decreased the rates of this complication, resulting on an improvement of transplant-related toxicity and survival. Secondary to its efficacy, the use of PTCy has been almost universally integrated for allogeneic hematopoietic cell transplantation (alloHCT), independently of the selected donor source. Clinical decisions in alloHCT are supported by the use of prognostic scores for outcome prediction. However, capability of prediction by diverse scores can vary depending on their features and on the composition of the study cohort. Additionally, the continuous innovation on alloHCT techniques and practices leads to an ongoing need to update risk indices aimed at improving risk stratification of patients undergoing alloHCT. This study explores the predictive capacity of different prognostic scores routinely used in alloHCT, in a contemporaneous cohort of adults undergoing peripheral blood (PB) alloHCT using PTCy-based GvHD prophylaxis. METHODS Between 2014 and 2020, 230 consecutive adults with hematological malignancies underwent PB-alloHCT with PTCy-based GvHD prophylaxis at our Institution. Data related to Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI), Karnosfky Performance Status (KPS), Disease Risk Index (DRI), European Bone Marrow Transplantation (EBMT) score, and Endothelial Activation and Stress Index (EASIX) were collected retrospectively. Complete information was available for 216 patients. Overall survival (OS) was considered the main outcome variable. Patients were grouped into two risk groups based on the optimal cut-off value for each score. In the case of EASIX, 1.578 was the most discriminating cut-off for OS. The score discrimination for OS was measured independently for each index using the receiver operating characteristic curve (AUC) calculated using receiver operating characteristic (ROC) curves, and determined at different time-points after alloHCT. RESULTS Of the 216 patients included, the median age was 52 years (range: 18-70), acute myeloid leukemia (36.1%) was the most prevalent baseline diagnosis, 42.1% of adults underwent reduced-intensity conditioning alloHCT, 69.4% received grafts from unrelated donors, and 23.0% from haploidentical donors. With a median follow-up of 22.6 months, 24.1% patients relapsed, and 2-y OS and non-relapse mortality were 67.3% and 19.9%. DRI, HCT-CI, KPS, and EASIX successfully grouped patients into higher and lower risk strata, supporting their use for risk classification. HCT-CI [(score&gt;3 (vs 0-3): HR 2.02, p&lt;0.01], DRI [High - Very High risk (vs Low - Int): HR 2.08, p&lt;0.01], and EASIX [&gt;1.578 (vs ≤ 1.578): HR 1.73, p&lt;0.02], maintained an optimal discrimination capacity during the entire post-transplant follow-up (median AUC ranges &gt; 55%). DRI was the most accurate prognostic index during the entire post-transplant period (median AUC ranges &gt; 60%). KPS score was found to be a useful predictor of mortality up to the first year after alloHCT and with the highest prognostic accuracy at 3 months (AUC 67.09%). HCT-CI score was found to present a better discrimination capacity once elapsed 6 months after alloHCT and with a peak of prediction capacity at 2 years (AUC 60.3%). EASIX, when measured at the pre-transplant evaluation, demonstrated to have acceptable predictive ability during the entire post-transplant period (median AUC &gt; 55%), and with a peak of prediction at 3 months (AUC 62.6%). The EBMT score had the lowest predictive capacity in our analysis (Figure 1). CONCLUSION: This study validates, for the first time, the risk stratification capacity for OS of DRI, HCT-CI, KPS, and EASIX in PB-alloHCT with PCTy-based prophylaxis. Interestingly, the prediction accuracy of the prognostic scores differed depending on the time-period. This result can be taken into consideration to enhance the applicability of these scores and refine the clinical decisions taken based on the information provided from their use in routine clinical practice. Figure 1 Figure 1. Disclosures Lozano: Terumo BCT: Honoraria, Research Funding; Macopharma: Research Funding; Grifols: Honoraria. Rosinol: Janssen, Celgene, Amgen and Takeda: Honoraria. Esteve: Novartis: Consultancy, Research Funding; Astellas: Consultancy; Jazz: Consultancy; Pfizer: Consultancy; Novartis: Research Funding; Abbvie: Consultancy; Bristol Myers Squibb/Celgene: Consultancy.

scholarly journals Establishment of Subspecialized Care in Hematologic Malignancies and a Hematopoietic Cell Transplantation Program

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3580-3580
Author(s):  
Edward A. Copelan ◽  
Jonathan M. Gerber ◽  
Saad Z Usmani ◽  
Michael R. Grunwald ◽  
Nilanjan Ghosh ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Cell Transplantation ◽  
Research Funding ◽  
Hematopoietic Cell Transplantation ◽  
Hematologic Malignancies ◽  
Hematopoietic Cell ◽  
Advisory Committees ◽  
Transplantation Program ◽  
Disease Specific

Abstract Introduction Access to appropriate healthcare close to home is a national and global problem with huge geographic variation in availability of subspecialized care and specific therapies, as illustrated by hematopoietic cell transplantation rates [Gratwohl, et al. Lancet Hematol, 2015]. Disease-specific physician specialization appears to improve outcomes in hematologic malignancies [Go, et al. Mayo Clin Proc, 2015]. Charlotte is the 2nd largest city in the Southeastern United States and 17th largest in the US, yet subspecialized care in hematologic malignancies including a leukemia unit and hematopoietic cell transplantation (HCT) program were non-existent 3 years ago. The closest HCT program was a 90-minute drive from Charlotte. Many patients lacked the resources or willingness to travel to a transplant center and died of potentially curable diseases. Establishment of a transplantation program, in particular, requires a substantial upfront investment, broad infrastructure and highly specialized interdisciplinary care. Better transplant outcomes have been associated with higher numbers of procedures [Loberiza, et al. Blood, 2005], but programs established over the last decade have struggled to attract adequate numbers of patients to support the required investment. Methods In 2011 Carolinas HealthCare System (CHS), which serves as a healthcare safety net for the region, decided to develop the Levine Cancer Institute as a primary through quaternary referral and treatment center, integrated through more than 12 sites, with a key focus being a program in HCT. The Department of Hematologic Malignancies and Blood Disorders was established in September 2012. We envisioned that the development of specialized care in leukemia, lymphoma, and plasma cell disorders, as well as more complex non-malignant hematologic disorders, would improve the quality of care for patients with those diseases, attract larger volumes of patients, and serve to identify patients appropriate for HCT in a timely manner. A 16 bed hematologic malignancies unit housed in a protected environment was constructed and completed in January 2014. Results Starting with 4 general hematologists, the department has grown over 4 years to include 23 faculty members, 14 of whom provide subspecialized care in hematologic malignancies and HCT. Patient volumes have grown more than six-fold during this time. The HCT Program performed its first transplant in March 2014, with a total of 60 transplants performed in 2014 and 81 in 2015. The HCT Program is on pace to perform over 100 transplants in 2016. The program received FACT accreditation in 2016, a little more than 2 years after the first HCT was performed. The age range of patients undergoing transplantation is from 22 to 76 (median 58) years. Sixty-nine percent of transplants have been autologous and 31% allogeneic, of which 65% were from haploidentical related donors. The proportion of transplants which are allogeneic is steadily increasing. More than 90% of patients who have undergone transplantation were referred through a disease-specific section. Non-relapse mortality (NRM) at 1 year is 1.8% for autologous transplants and 9.4% for allogeneic transplants, with survival rates at 1 year of 95.6% and 80.8% respectively. Notably, there is no difference in NRM (P=0.86), relapse-free survival (P=0.85), or overall survival (P=0.47) between HLA-identical and haploidentical transplant recipients. Conclusions Three years ago, for patients in Charlotte, access to subspecialized care in hematologic malignancies and HCT required significant travel. The development and growth of a program that provides disease-specific care in hematologic malignancies has overcome this barrier and has provided a base for growth of a newly established program in HCT. These developments have elevated the quality of care in hematologic malignancies in the Charlotte area and permit patients to receive appropriate and complex care close to home. Disclosures Gerber: Janssen: Research Funding; Alexion: Membership on an entity's Board of Directors or advisory committees; Spectrum: Membership on an entity's Board of Directors or advisory committees; Seattle Genetics: Membership on an entity's Board of Directors or advisory committees. Usmani:Takeda: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Pharmacyclics: Research Funding; Novartis: Speakers Bureau; Millenium: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; BioPharma: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Sanofi: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Array: Research Funding; Amgen: Consultancy, Research Funding, Speakers Bureau; Onyx: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Britsol-Myers Squibb: Consultancy, Research Funding; Janssen: Membership on an entity's Board of Directors or advisory committees, Research Funding; Skyline: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Grunwald:Amgen: Research Funding; Incyte Corporation: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Ariad: Membership on an entity's Board of Directors or advisory committees; Forma Therapeutics: Research Funding; Medtronic: Equity Ownership; Janssen: Research Funding; Alexion: Membership on an entity's Board of Directors or advisory committees. Ghosh:Pharmacyclics LLC, an AbbVie Company: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; AbbVie: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; SGN: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Janssen: Consultancy, Honoraria, Research Funding, Speakers Bureau; Gilead: Honoraria, Speakers Bureau; Bristol Myers Squibb: Consultancy, Honoraria, Research Funding; Celgene: Consultancy, Honoraria, Research Funding, Speakers Bureau; Genentech: Research Funding; TG Therapeutics: Research Funding. Bhutani:Prothena: Research Funding; Takeda Oncology: Research Funding, Speakers Bureau; Bristol-Myers Squibb: Speakers Bureau; Onyx, an Amgen subsidiary: Speakers Bureau. Symanowski:Endocyte: Consultancy; Eli Lilly & Co: Consultancy; Ra Pharma: Consultancy; Caris Life Sciences: Consultancy. Raghavan:Gerson Lehrman: Consultancy; Caris Life Sciences: Membership on an entity's Board of Directors or advisory committees. Avalos:Seattle Genetics: Membership on an entity's Board of Directors or advisory committees.

scholarly journals Parent proxy assessment of sibling quality of life following pediatric hematopoietic cell transplantation

2019 ◽  
Vol 17 (1) ◽  
Author(s):  
David Buchbinder ◽  
Sunita K. Patel ◽  
Jacqueline N. Casillas ◽  
Diane J. Nugent ◽  
Steven Neudorf ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Physical Health ◽  
Cell Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
Short Form ◽  
Hematopoietic Cell ◽  
Family Function ◽  
The Impact ◽  
Parent Proxy

Abstract Background When a child undergoes hematopoietic cell transplantation (HCT), the impact extends to the entire family, including siblings. Assessment of the quality of life (QoL) of siblings is challenged by their general lack of availability for regular assessment by clinical providers. Thus, the use of parent proxy reporting may be useful. Our aim was to describe the QoL of siblings of HCT survivors, as reported by their parents, as well as to identify parent and family factors associated with lower sibling QoL. Methods A cross-sectional study was utilized to assess parent-reported QoL of the HCT recipient’s sibling (Short Form (SF)-10 Health Survey for Children and the Pediatric Symptom Checklist (PSC)-17). Parent QoL was assessed using the SF-12. Multivariable linear regression was used to explore hypothesized predictors of sibling QoL, including parent QoL, family impact/function (Impact on Family Scale, Family Adaptability and Cohesion Evaluation Scales, IV, and a question asking about financial problems) while adjusting for demographic and HCT characteristics. Results Ninety-seven siblings (55% males) with a mean age of 12 years (standard deviation [SD] 4 years) were assessed, representing HCT survivors, who were an average of 5 years (SD 4 years) post-HCT. Neither sibling psychosocial (mean 49.84, SD 10.70, p = 0.87) nor physical health scores (mean 51.54, SD 8.42, p = 0.08) differed from norms. Parent proxies reported behavioral/emotional problems (PSC-17 total score > 15) in 24% of siblings. While parental ratings of their own physical health (SF-12 were higher than norms (mean 53.04, SD 8.17, p = 0.0005), mental health scores were lower (mean 45.48, SD 10.45, p < 0.0001). In multivariable analysis, lower parent emotional functioning and adverse family function were associated with lower sibling QoL, as reported by parents. Conclusions While proxy-reported QoL of siblings did not differ significantly from normative data, both parent QoL and family function were associated with sibling QoL. Future research is needed to understand how siblings themselves perceive their QoL following HCT.

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