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Fertility challenges for women with sickle cell disease.
Ghafuri DL, Stimpson SJ, Day ME, James A, DeBaun MR, Sharma D
(2017) Expert Rev Hematol 10: 891-901
MeSH Terms: Anemia, Sickle Cell, Blood Transfusion, Chronic Pain, Female, Fertility, Fertility Preservation, Genetic Therapy, Hematopoietic Stem Cell Transplantation, Humans, Hydroxyurea, Infertility, Pregnancy, Primary Ovarian Insufficiency, Reproductive Health, Transplantation Conditioning
Show Abstract · Added November 9, 2018
INTRODUCTION - Sickle cell disease (SCD) represents one of the most common monogenic blood disorders worldwide, with an incidence of over 300,000 newborns affected per year. Reproductive challenges for men and women with SCD have been previously reviewed; however, evidence-based strategies to prevent and manage infertility and increase fecundity are lacking in women with SCD, which is one of the most important factors for quality of life. Areas covered: This review article summarizes the known risk factors for infertility, low fecundity, and premature menopause related to SCD. Expert commentary: Women with SCD have unique risk factors that may impact their ability to conceive, including chronic inflammation, oxidative stress, transfusion-related hemochromatosis, and ovarian sickling, causing ischemia and reperfusion injury to the ovary. Contraception is strongly recommended while on hydroxyurea therapy during reproductive years and discontinuing hydroxyurea for family planning and during pregnancy based on teratogenicity in animal studies. Hematopoietic stem cell transplantation (HSCT), the only curative therapy, sometimes involves conditioning regimens containing alkylating agents and total body irradiation that contribute to infertility and premature ovarian failure. Prior to HSCT or gene therapy, we strongly recommend referral to a reproductive endocrinologist to discuss fertility preservation and surrogacy options for all women with SCD.
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MeSH Terms
Reduced-Intensity Conditioning with Fludarabine, Cyclophosphamide, and Rituximab Is Associated with Improved Outcomes Compared with Fludarabine and Busulfan after Allogeneic Stem Cell Transplantation for B Cell Malignancies.
Kennedy VE, Savani BN, Greer JP, Kassim AA, Engelhardt BG, Goodman SA, Sengsayadeth S, Chinratanalab W, Jagasia M
(2016) Biol Blood Marrow Transplant 22: 1801-1807
MeSH Terms: Adult, Aged, Antineoplastic Combined Chemotherapy Protocols, Busulfan, Calcineurin Inhibitors, Cyclophosphamide, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Humans, Leukemia, B-Cell, Methotrexate, Middle Aged, Prognosis, Retrospective Studies, Rituximab, Survival Analysis, Transplantation Conditioning, Transplantation, Homologous, Vidarabine
Show Abstract · Added July 28, 2016
Reduced-intensity conditioning (RIC) has been used increasingly for allogeneic hematopoietic cell transplantation to minimize transplant-related mortality while maintaining the graft-versus-tumor effect. In B cell lymphoid malignancies, reduced-intensity regimens containing rituximab, an antiCD20 antibody, have been associated with favorable survival; however, the long-term outcomes of rituximab-containing versus nonrituximab-containing regimens for allogeneic hematopoietic cell transplantation in B cell lymphoid malignancies remain to be determined. We retrospectively analyzed 94 patients who received an allogeneic transplant for a B cell lymphoid malignancy. Of these, 33 received RIC with fludarabine, cyclophosphamide, and rituximab (FCR) and graft-versus-host disease (GVHD) prophylaxis with a calcineurin inhibitor and mini-methotrexate, and 61 received RIC with fludarabine and busulfan (FluBu) and GVHD prophylaxis with a calcineurin inhibitor and mycophenolate mofetil. The 2-year overall survival was superior in patients who received FCR versus FluBu (72.7% versus 54.1%, P = .031), and in multivariable analysis adjusted for Disease Risk Index and donor type, only the conditioning regimen (FluBu versus FCR: HR, 2.06; 95% CI, 1.04 to 4.08; P = .037) and Disease Risk Index (low versus intermediate/high: HR, .38; 95% CI, .17 to .86; P = .02) were independent predictors of overall survival. The 2-year cumulative incidence of chronic GVHD was lower in patients who received FCR versus FluBu (24.2% versus 51.7%, P = .01). There was no difference in rate of relapse/progression or acute GVHD. Our results demonstrate that the use of RIC with FCR and GVHD prophylaxis with a calcineurin inhibitor and mini-methotrexate is associated with decreased chronic GVHD and improved overall survival.
Copyright © 2016 The American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
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19 MeSH Terms
The outcome of allogeneic hematopoietic cell transplantation for children with FMS-like tyrosine kinase 3 internal tandem duplication-positive acute myelogenous leukemia.
Schechter T, Gassas A, Chen H, Pollard J, Meshinchi S, Zaidman I, Hitzler J, Abdelhaleem M, Ho R, Domm J, Woolfrey A, Frangoul H
(2015) Biol Blood Marrow Transplant 21: 172-5
MeSH Terms: Adolescent, Child, Child, Preschool, Chromosome Duplication, Female, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Humans, Immunosuppressive Agents, Leukemia, Myeloid, Acute, Male, Myeloablative Agonists, Recurrence, Retrospective Studies, Siblings, Survival Analysis, Transplantation Conditioning, Transplantation, Homologous, Treatment Outcome, Unrelated Donors, Whole-Body Irradiation, Young Adult, fms-Like Tyrosine Kinase 3
Show Abstract · Added February 25, 2015
FMS-like tyrosine kinase 3 (FLT3) internal tandem duplication (ITD) is a somatic mutation associated with poor outcome when treated with chemotherapy alone. In children, hematopoietic stem cell transplantation (HSCT) is recommended, but very limited data on outcome are reported. We determined the outcome of 29 children with FLT3/ITD-positive acute myelogenous leukemia (AML) who underwent allogeneic HSCT in 4 pediatric centers. Eleven patients (38%) received matched related donor hematopoietic stem cells and 18 (62%) received alternative donors. Eighteen patients (62%) received total body irradiation (TBI)-based regimens. No patients experienced transplantation-related mortality. Eleven patients (38%) experienced relapsed disease. The cumulative incidence of relapse at 2 years was 34.7% (95% confidence interval [CI], 20.4% to 54.9%). Two-year disease-free survival (DFS) and overall survival (OS) were 65.3% (95% CI, 45.1% to 79.6%) and 82.2% (95% CI, 58.5% to 91.3%), respectively. There was no difference in the DFS of patients who received transplants from related donors versus the DFS of those who received transplants from alternative donors (hazard ratio [HR], 2.64; 95% CI, .79 to 8.76; P = .10), using univariate analysis. Patients with higher FLT3/ITD ratio at diagnosis had significantly worse DFS (HR, 1.42; 95% CI, 1.04 to 1.93; P = .03). The use of TBI in the preparative regimen was associated with superior DFS (HR, .29; 95% CI, .08 to .99; P = .04) and OS (HR, .07; 95% CI, .01 to .62; P = .002). We conclude that allogeneic HSCT improves DFS and OS in children with FLT3/ITD-positive AML compared with what has been reported in those treated with chemotherapy alone.
Copyright © 2015 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
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23 MeSH Terms
Allotransplantation for patients age ≥40 years with non-Hodgkin lymphoma: encouraging progression-free survival.
McClune BL, Ahn KW, Wang HL, Antin JH, Artz AS, Cahn JY, Deol A, Freytes CO, Hamadani M, Holmberg LA, Jagasia MH, Jakubowski AA, Kharfan-Dabaja MA, Lazarus HM, Miller AM, Olsson R, Pedersen TL, Pidala J, Pulsipher MA, Rowe JM, Saber W, van Besien KW, Waller EK, Aljurf MD, Akpek G, Bacher U, Chao NJ, Chen YB, Cooper BW, Dehn J, de Lima MJ, Hsu JW, Lewis ID, Marks DI, McGuirk J, Cairo MS, Schouten HC, Szer J, Ramanathan M, Savani BN, Seftel M, Socie G, Vij R, Warlick ED, Weisdorf DJ
(2014) Biol Blood Marrow Transplant 20: 960-8
MeSH Terms: Adult, Age Factors, Disease-Free Survival, Female, Hematopoietic Stem Cell Transplantation, Humans, Lymphoma, Non-Hodgkin, Male, Middle Aged, Survival Analysis, Transplantation Conditioning, Transplantation, Homologous
Show Abstract · Added March 20, 2014
Non-Hodgkin lymphoma (NHL) disproportionately affects older patients, who do not often undergo allogeneic hematopoietic cell transplantation (HCT). We analyzed Center for International Blood and Marrow Transplant Research data on 1248 patients age ≥40 years receiving reduced-intensity conditioning (RIC) or nonmyeloablative (NMA) conditioning HCT for aggressive (n = 668) or indolent (n = 580) NHL. Aggressive lymphoma was more frequent in the oldest cohort 49% for age 40 to 54 versus 57% for age 55 to 64 versus 67% for age ≥65; P = .0008). Fewer patients aged ≥65 had previous autografting (26% versus 24% versus 9%; P = .002). Rates of relapse, acute and chronic GVHD, and nonrelapse mortality (NRM) at 1 year post-HCT were similar in the 3 age cohorts (22% [95% confidence interval (CI), 19% to 26%] for age 40 to 54, 27% [95% CI, 23% to 31%] for age 55 to 64, and 34% [95% CI, 24% to 44%] for age ≥65. Progression-free survival (PFS) and overall survival (OS) at 3 years was slightly lower in the older cohorts (OS: 54% [95% CI, 50% to 58%] for age 40 to 54; 40% [95% CI, 36% to 44%] for age 55 to 64, and 39% [95% CI, 28% to 50%] for age ≥65; P < .0001). Multivariate analysis revealed no significant effect of age on the incidence of acute or chronic GVHD or relapse. Age ≥55 years, Karnofsky Performance Status <80, and HLA mismatch adversely affected NRM, PFS, and OS. Disease status at HCT, but not histological subtype, was associated with worse NRM, relapse, PFS, and OS. Even for patients age ≥55 years, OS still approached 40% at 3 years, suggesting that HCT affects long-term remission and remains underused in qualified older patients with NHL.
Copyright © 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
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12 MeSH Terms
Late cardiovascular complications after hematopoietic cell transplantation.
Chow EJ, Wong K, Lee SJ, Cushing-Haugen KL, Flowers ME, Friedman DL, Leisenring WM, Martin PJ, Mueller BA, Baker KS
(2014) Biol Blood Marrow Transplant 20: 794-800
MeSH Terms: Adult, Cardiovascular Diseases, Case-Control Studies, Female, Hematopoietic Stem Cell Transplantation, Humans, Male, Middle Aged, Survival Analysis, Transplantation Conditioning, Treatment Outcome, Young Adult
Show Abstract · Added March 27, 2014
The authors sought to better understand the combined effects of pretransplant, transplant, and post-transplant factors in determining risks of serious cardiovascular disease after hematopoietic cell transplantation (HCT). Hospitalizations and deaths associated with serious cardiovascular outcomes were identified among 1379 Washington State residents who received HCT (57% allogeneic and 43% autologous) at a single center from 1985 to 2005, survived ≥ 2 years, and followed through 2008. Using a nested case-cohort design, relationships (hazard ratios [HRs]) between potential risk factors and outcomes were examined among affected survivors and a randomly selected subcohort (N = 509). After 7.0 years of median follow-up (range, 2.0 to 23.7), the 10-year cumulative incidence of ischemic heart disease, cardiomyopathy, stroke, and all-cause cardiovascular death was 3.8%, 6.0%, 3.5%, and 3.7%, respectively. In multivariable analysis, increased pretransplant anthracycline was associated with cardiomyopathy. Active chronic graft-versus-host disease was associated with cardiovascular death (HR, 4.0; 95% confidence interval, 1.1 to 14.7); risk was otherwise similar between autologous versus allogeneic HCT recipients. Independent of therapeutic exposures, pretransplant smoking, hypertension, dyslipidemia, diabetes, and obesity conferred additional risk of all outcomes except stroke (HR ≥ 1.5 for each additional risk factor, P < .03). Hypertension and dyslipidemia at 1 year with persistence of these conditions 2 or more years after HCT also were associated with independent risks of multiple outcomes. HCT survivors with preexisting or newly developed and persistent cardiovascular risk factors remain at greater risk of subsequent serious cardiovascular disease compared with other survivors, independent of chemo- and radiotherapy exposures. These survivors should receive appropriate follow-up and be considered for primary intervention.
Copyright © 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
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12 MeSH Terms
Investigator feedback about the 2005 NIH diagnostic and scoring criteria for chronic GVHD.
Inamoto Y, Jagasia M, Wood WA, Pidala J, Palmer J, Khera N, Weisdorf D, Carpenter PA, Flowers ME, Jacobsohn D, Martin PJ, Lee SJ, Pavletic SZ, Chronic GVHD Consortium
(2014) Bone Marrow Transplant 49: 532-8
MeSH Terms: Chronic Disease, Data Collection, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Humans, Severity of Illness Index, Surveys and Questionnaires, Transplantation Conditioning, Transplantation, Homologous, United States
Show Abstract · Added March 20, 2014
The 2005 National Institutes of Health (NIH) consensus criteria for chronic GVHD have set standards for reporting. Many questions, however, have arisen regarding their implementation and utilization. To identify perceived areas of controversy, we conducted an international survey on diagnosis and scoring of chronic GVHD. Agreement was observed for 50-83% of the 72 questions in 7 topic areas. There was agreement on the need for modifying criteria in six situations: two or more distinctive manifestations should be enough to diagnose chronic GVHD; symptoms that are not due to chronic GVHD should be scored differently; active disease and fixed deficits should be distinguished; a minimum threshold body surface area of hidebound skin involvement should be required for a skin score of 3; asymptomatic oral lichenoid changes should be considered a score 1; and lung biopsy should be unnecessary to diagnose chronic GVHD in a patient with bronchiolitis obliterans as the only manifestation. The survey also identified 26 points of controversy. Whenever possible, studies should be conducted to confirm the appropriateness of any revisions. In cases where data are not available, clarification of the NIH recommendations by consensus is necessary. This survey should inform future research in the field and revisions of the current consensus criteria.
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10 MeSH Terms
Avascular necrosis of bone after allogeneic hematopoietic cell transplantation in children and adolescents.
Li X, Brazauskas R, Wang Z, Al-Seraihy A, Baker KS, Cahn JY, Frangoul HA, Gajewski JL, Hale GA, Hsu JW, Kamble RT, Lazarus HM, Marks DI, Maziarz RT, Savani BN, Shah AJ, Shah N, Sorror ML, Wood WA, Majhail NS
(2014) Biol Blood Marrow Transplant 20: 587-92
MeSH Terms: Adolescent, Bone and Bones, Case-Control Studies, Child, Chronic Disease, Female, Graft vs Host Disease, Hematologic Neoplasms, Hematopoietic Stem Cell Transplantation, Humans, Immunosuppressive Agents, Male, Myeloablative Agonists, Osteonecrosis, Risk Factors, Severity of Illness Index, Sex Factors, Time Factors, Transplantation Conditioning, Transplantation, Homologous, Young Adult
Show Abstract · Added March 27, 2014
We conducted a nested case-control study within a cohort of 6244 patients to assess risk factors for avascular necrosis (AVN) of bone in children and adolescents after allogeneic transplantation. Eligible patients were ≤21 years of age, received their first allogeneic transplant between 1990 and 2008 in the United States, and had survived ≥ 6 months from transplantation. Overall, 160 patients with AVN and 478 control subjects matched by year of transplant, length of follow-up and transplant center were identified. Patients and control subjects were confirmed via central review of radiology, pathology, and/or surgical procedure reports. Median time from transplant to diagnosis of AVN was 14 months. On conditional logistic regression, increasing age at transplant (≥5 years), female gender, and chronic graft-versus-host disease (GVHD) were significantly associated with increased risks of AVN. Compared with patients receiving myeloablative regimens for malignant diseases, lower risks of AVN were seen in patients with nonmalignant diseases and those who had received reduced-intensity conditioning regimens for malignant diseases. Children at high risk for AVN include those within the age group where rapid bone growth occurs as well as those who experience exposure to myeloablative conditioning regimens and immunosuppression after hematopoietic cell transplantation for the treatment of GVHD. More research is needed to determine whether screening strategies specifically for patients at high risk for developing AVN with early interventions may mitigate the morbidity associated with this complication.
Copyright © 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
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21 MeSH Terms
Allogeneic hematopoietic stem cell transplantation for infants with idiopathic myelofibrosis.
Hussein AA, Hamadah T, Domm J, Al-Zaben A, Frangoul H
(2013) Pediatr Transplant 17: 815-9
MeSH Terms: Blood Platelets, Busulfan, Child, Preschool, Cohort Studies, Cyclophosphamide, Female, Follow-Up Studies, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Humans, Immunosuppressive Agents, Infant, Male, Neutrophils, Primary Myelofibrosis, Stem Cells, Transplantation Conditioning, Treatment Outcome
Show Abstract · Added March 7, 2014
IMF is a rare disease in children that can present during infancy and has a protracted course. The only known curative approach for this disease in adult patients is allogeneic HSCT. There are very few reports describing the long-term outcome of young children following stem cell transplantation for IMF. We report on eight patients less than two yr of age with IMF that did not resolve with supportive care measures. All patients underwent myeloablative conditioning regimen with busulfan and cyclophosphamide ± ATG followed by HSCT from matched related (n = 6) or unrelated donor (n = 2). All patients achieved neutrophil and platelet engraftment. Four patients had grade II-III acute GVHD, and chronic GVHD developed in five patients (three mild and two severe). At a median follow-up of eight and a half yr (0.7-9), all patients are alive with complete resolution of their hematologic manifestations. At the last follow-up, all patients had normal endocrine function except for one patient who developed hypothyroidism. To date, this is the largest cohort of young children with IMF treated successfully with HSCT, with the longest duration of follow-up. In conclusion, our study showed that HSCT is a curative option for infants with IMF.
© 2013 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.
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18 MeSH Terms
Six-month freedom from treatment failure is an important end point for acute GVHD clinical trials.
Sengsayadeth S, Savani BN, Jagasia M, Goodman S, Greer JP, Chen H, Chinratanalab W, Kassim AA, Engelhardt BG
(2014) Bone Marrow Transplant 49: 236-40
MeSH Terms: Acute Disease, Adult, Aged, Female, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Humans, Male, Middle Aged, Survival Analysis, Transplantation Conditioning, Transplantation, Homologous, Treatment Failure, Treatment Outcome
Show Abstract · Added March 20, 2014
We studied the American Society for Blood and Marrow Transplantation (ASBMT) 6-month (m) freedom from treatment failure (FFTF) as a predictor of survival for patients with acute GVHD (aGVHD) requiring treatment. Adult patients undergoing allogeneic hematopoietic cell transplant (HCT) from February 2007 to March 2009 who were enrolled in a prospective biomarker clinical trial and developed aGVHD requiring systemic corticosteroids by day +100 were included (N=44). Six-month FFTF was defined as per the ASBMT guidelines (absence of death, malignancy relapse/progression or systemic immunosuppression change within 6 months of starting steroids and before chronic GVHD development). aGVHD was treated with systemic corticosteroids in 44 patients. Day 28 response after steroid initiation (complete response+very good partial response+partial response) occurred in 38 (87%) patients, but only 28 (64%) HCT recipients met the 6-m FFTF end point. Day 28 response predicted 6-m FFTF. Achieving 6-m FFTF was associated with improved 2-year (y) OS (81% vs 48%; P=0.03) and decreased 2-y non-relapse mortality (8% vs 49%; P=0.01). In multivariate analysis, 6-m FFTF continued to predict improved OS (hazard ratio, 0.27; P=0.03). The 6-m FFTF end point measures fixed outcomes, predicts long-term therapeutic success and could be less prone to measurement error than aGVHD clinical response at day 28.
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14 MeSH Terms
Incidence and risk factors for hypogammaglobulinemia in pediatric patients following allo-SCT.
Frangoul H, Min E, Wang W, Chandrasekhar R, Calder C, Evans M, Manes B, Bruce K, Brown V, Ho R, Domm J
(2013) Bone Marrow Transplant 48: 1456-9
MeSH Terms: Adolescent, Adult, Agammaglobulinemia, Child, Child, Preschool, Female, Hematopoietic Stem Cell Transplantation, Humans, Incidence, Infant, Infant, Newborn, Male, Prospective Studies, Risk Factors, Transplantation Conditioning, Transplantation, Autologous, Young Adult
Show Abstract · Added March 5, 2014
We evaluated the incidence and risk factors for hypogammaglobulinemia after allogeneic hematopoietic SCT (HSCT) in pediatric patients. Ig levels were measured pre-transplant, every 2 weeks until day 100 and then monthly post SCT in 185 patients undergoing myeloablative HSCT. Median age was 9 years; 142 (77%) had malignant disease and 114 (62%) received stem cells from an unrelated source. Hypogammaglobulinemia (IgG <500 mg/dL) developed in 143 (77%) of the patients at a median of 56 days (range 15-339) post SCT. The cumulative incidence of hypogammaglobulinemia at 1 year was higher among patients who developed acute GVHD (97% vs 54%, P<0.001), and for those receiving stem cells from an unrelated source (94% vs 51%, P<0.001). The cumulative incidence of TRM was significantly higher for patients with hypogammaglobulinemia (P=0.026). In multivariable analysis, lower pre-transplant IgG level (P<0.001), younger age (P=0.012), diagnosis of malignant disease (P<0.001), receiving unrelated SCT (P<0.001) and development of acute GVHD (P<0.001) were all significantly associated with higher risk of hypogammaglobulinemia post HSCT. We conclude that hypogammaglobulinemia is common, following allogeneic HSCT in pediatric patients, especially in those with malignant diseases, those who receive an unrelated transplant or patients who develop GVHD.
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17 MeSH Terms