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4.
Bone Marrow Transplant ; 51(9): 1173-9, 2016 Sep.
Article in English | MEDLINE | ID: mdl-27159181

ABSTRACT

Much research into the impact of hematopoietic cell transplantation (HCT) on recipients' symptoms, functioning and health-related quality of life uses diverse patient-reported outcome (PRO) measures. Robust conclusions regarding PROs in HCT patients are constrained by methodological issues, including the use of multiple different and noncomparable assessment measures. We reviewed 114 publications addressing PROs in HCT patients. Although three multi-item measures were most frequently used (FACT-BMT, n=28; European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-C30, n=26; and SF-36, n=26), 25 additional measures were used in more than one study. Another 50 measures were used in single studies. Over 50% of studies used more than one measure. We recommend that the field agrees upon a set of measures to address the core domains important to patients, to reduce heterogeneity and allow comparisons across studies and between different populations. Measures should be available in a free and easily accessible manner internationally. We discuss the relative benefits of the National Institutes of Health-supported Patient-Reported Outcomes Measurement Information System (PROMIS) system to achieve these goals. To further address these issues, the Blood and Marrow Transplant Clinical Trials Network has recently created a task force to implement PROMIS measures alongside traditional PRO measures in future clinical trials. Robust comparisons between measures in this setting may allow for the development of a standard for HCT patients.


Subject(s)
Hematopoietic Stem Cell Transplantation/standards , Outcome Assessment, Health Care/standards , Patient Reported Outcome Measures , Quality Indicators, Health Care/standards , Humans , Reproducibility of Results , Treatment Outcome
5.
Bone Marrow Transplant ; 49(8): 1016-21, 2014 Aug.
Article in English | MEDLINE | ID: mdl-24777183

ABSTRACT

Patient registries, frequently referred to as outcome registries, are 'organized systems' that use observational study methods to collect uniform data. Registries are used to evaluate specified outcomes for a population defined by a particular disease, condition or exposure that serves one or more predetermined scientific, clinical or policy purposes. Outcome registries were established very early in the development of hematopoietic SCT (HSCT). Currently, myriads of national and international HSCT registries collect information about HSCT activities and outcomes. These registries have contributed significantly to determining trends, patterns, treatment practices and outcomes. There are many different HSCT registries, each with different aims and goals; some are led by professional organizations, others by government authorities, health care providers or third parties. Some registries simply assess activity and others study outcomes. These registries are complementary and are gradually developing interoperability with each other to expand future collaborative research activities. A key development in the last few years was the incorporation of recommendations into the World Health Organization guiding principles on cell, tissue and organ transplantation. The data collection and analysis should be an integral part of therapy and an obligation rather than a choice for transplant programs. This article examines challenges in ensuring data quality and functions of outcome registries, using HSCT registries as an example. It applies to all HSCT-related data, but is predominantly focused on HSCT registries of professional organizations.


Subject(s)
Hematopoietic Stem Cell Transplantation , Registries , Humans , Multicenter Studies as Topic
6.
Bone Marrow Transplant ; 48(2): 220-5, 2013 Feb.
Article in English | MEDLINE | ID: mdl-22773129

ABSTRACT

The number of allogeneic hematopoietic SCTs performed globally each year continues to increase, paralleled by an increased demand for donors of therapeutic cells. Donor characteristics and collection procedures have undergone major changes during recent decades, and further changes are foreseen. Information on short- and long-term donor outcomes is of crucial importance to ensure maximal donor safety and availability. Current data, predominantly from unrelated donors, give reliable information on the frequent early events associated with donation-most of them of mild-to-moderate intensity. Information on the type and relative risk of serious adverse reactions is more limited. Moreover, only few data exist on long-term donor outcome. On the basis of this need, recommendations for a minimum data set for prospective donor follow-up were developed in a workshop with the participation of an international group of investigators actively involved in allogeneic stem cell donation under the auspices of and approved by the Worldwide Network for Blood and Marrow Transplantation. Establishment of a standardized global follow-up for both, related and unrelated, donors will enable monitoring of the short- and long-term safety profiles of hematopoietic cell donation and form a solid basis for future donor selection and counseling.


Subject(s)
Hematopoietic Stem Cell Transplantation/methods , Hematopoietic Stem Cells/cytology , Tissue Donors , Adolescent , Adult , Donor Selection , Humans , Middle Aged , Transplantation, Homologous , Young Adult
7.
Transpl Infect Dis ; 14(5): 468-78, 2012 Oct.
Article in English | MEDLINE | ID: mdl-22548788

ABSTRACT

Limited data exist on allogeneic transplant outcomes in recipients receiving hematopoietic cells from donors with prior or current hepatitis B (HBV) or C virus (HCV) infection (seropositive donors), or for recipients with prior or current HBV or HCV infection (seropositive recipients). Transplant outcomes are reported for 416 recipients from 121 centers, who received a human leukocyte antigen-identical related-donor allogeneic transplant for hematologic malignancies between 1995 and 2003. Of these, 33 seronegative recipients received grafts from seropositive donors and 128 recipients were seropositive. The remaining 256 patients served as controls. With comparable median follow-up (cases, 5.9 years; controls, 6.7 years), the incidence of treatment-related mortality, survival, graft-versus-host disease, and hepatic toxicity, appears similar in all cohorts. The frequencies of hepatic toxicities as well as causes of death between cases and controls were similar. Prior exposure to HBV or HCV in either the donor or the recipient should not be considered an absolute contraindication to transplant.


Subject(s)
Hematopoietic Stem Cell Transplantation/mortality , Hepatitis B/mortality , Hepatitis C/mortality , Transplantation, Homologous/mortality , Adolescent , Adult , Child , Child, Preschool , Female , Hepacivirus , Hepatitis B/epidemiology , Hepatitis B/virology , Hepatitis B virus , Hepatitis C/epidemiology , Hepatitis C/virology , Humans , Incidence , Infant , Male , Middle Aged , Tissue Donors , Transplantation , Young Adult
8.
Bone Marrow Transplant ; 47(6): 810-6, 2012 Jun.
Article in English | MEDLINE | ID: mdl-21986636

ABSTRACT

Allogeneic hematopoietic SCT is an effective treatment in accelerated (AP) or blast phase (BP) CML. Imatinib (IM) has transient but significant activity in advanced phases of CML, which may permit early allografting for responding patients. To identify prognostic factors in allograft recipients previously treated with IM, we analyzed 449 allogeneic hematopoietic SCTs performed from 1999 to 2004 in advanced-phase CML, using the data reported to the Center for International Blood and Marrow Transplant Research. CML patients in second chronic phase (CP2, n=184), AP (n=185) and BP (n=80) received HLA-identical sibling (27%), related (3%), or matched or mismatched unrelated donor (70%), peripheral blood (47%) or BM (53%) hematopoietic SCT after myeloablative (78%) or non-myeloablative (22%) conditioning. In all, 52% in CP2, 49% in AP and 46% in BP received IM before hematopoietic SCT. Disease-free survival was 35-40% for CP2, 26-27% for AP and 8-11% for BP. Cumulative incidence of acute and chronic GVHD and TRM were not affected by the stages of CML or pre-hematopoietic SCT IM exposure. Multivariate analyses showed that conventional prognostic indicators remain the strongest determinants of transplant outcomes. In conclusion, there are no new prognostic indicators of the outcomes of allogeneic hematopoietic SCT for advanced-phase CML in the IM era.


Subject(s)
Hematopoietic Stem Cell Transplantation , Leukemia, Myelogenous, Chronic, BCR-ABL Positive/mortality , Leukemia, Myelogenous, Chronic, BCR-ABL Positive/therapy , Piperazines/administration & dosage , Protein Kinase Inhibitors/administration & dosage , Pyrimidines/administration & dosage , Transplantation Conditioning , Adolescent , Adult , Aged , Benzamides , Child , Disease-Free Survival , Female , Humans , Imatinib Mesylate , Male , Middle Aged , Retrospective Studies , Siblings , Survival Rate , Transplantation, Homologous
9.
Bone Marrow Transplant ; 46(1): 70-6, 2011 Jan.
Article in English | MEDLINE | ID: mdl-20436518

ABSTRACT

A retrospective analysis was conducted to examine factors affecting early mortality after myeloablative, single-unit cord blood transplantation (CBT) for hematological malignancies in adolescents and adults. Data were collected from the three main CBT registries pooling 514 records of unrelated, single, unmanipulated, first myeloablative allogeneic CBTs conducted in North America or Europe from 1995 to 2005, with an HLA match ≥ 4/6 loci, in patients aged 12-55. Overall 100-day, 180-day and 1-year survival (Kaplan-Meier method) were 56, 46 and 37%, respectively, with no significant heterogeneity across registries. Multivariate analysis showed cell dose < 2.5 × 107/kg (odds ratio (OR) 2.76, P < 0.0001), older age (P = 0.002), advanced disease (P = 0.02), positive CMV sero-status (OR 1.37 P = 0.11), female gender (OR 1.43, P = 0.07) and limited CBT center experience (< 10 records contributed, OR 2.08, P = 0.0003) to be associated with higher 100-day mortality. A multivariate model predictive of 1-year mortality included similar prognostic factors except female gender. Transplant year did not appear as a significant independent predictor. This is the first analysis to pool records from three major CBT registries in the United States and Europe. In spite of some differences in practice patterns, survival was remarkably homogeneous. The resulting model may contribute to better understanding factors affecting CBT outcomes.


Subject(s)
Cord Blood Stem Cell Transplantation/mortality , Hematologic Neoplasms/therapy , Myeloablative Agonists/therapeutic use , Transplantation Conditioning , Adolescent , Adult , Aging , Child , Cord Blood Stem Cell Transplantation/adverse effects , Cord Blood Stem Cell Transplantation/methods , Cytomegalovirus Infections/complications , Europe , Female , Hematologic Neoplasms/complications , Hematologic Neoplasms/diagnosis , Humans , Male , Middle Aged , Myeloablative Agonists/adverse effects , Neoplasm Staging , North America , Prognosis , Registries , Reproducibility of Results , Retrospective Studies , Survival Analysis , Transplantation Conditioning/adverse effects , Young Adult
10.
Bone Marrow Transplant ; 45(6): 1068-76, 2010 Jun.
Article in English | MEDLINE | ID: mdl-19915634

ABSTRACT

GVHD is partly mediated by host APCs that activate donor T cells. Extracorporeal photopheresis (ECP) can modulate APC function and benefit some patients with GVHD. We report the results of a study using ECP administered before a standard myeloablative preparative regimen intended to prevent GVHD. Grades II-IV acute GVHD developed in 9 (30%) of 30 recipients of HLA-matched related transplants and 13 (41%) of 32 recipients of HLA-matched unrelated or HLA-mismatched related donor transplants. Actuarial estimates of overall survival (OS) at day 100 and 1-year post transplant were 89% (95% CI, 78-94%) and 77% (95% CI, 64-86%), respectively. There were no unexpected adverse effects of ECP. Historical controls receiving similar conditioning and GVHD prophylaxis regimens but no ECP were identified from the database of the Center for International Blood and Marrow Transplant Research and multivariate analysis indicated a lower risk of grades II-IV acute GVHD in patients receiving ECP (P=0.04). Adjusted OS at 1 year was 83% in the ECP study group and 67% in the historical control group (relative risk 0.44; 95% CI, 0.24-0.80) (P=0.007). These preliminary data may indicate a potential survival advantage with ECP for transplant recipients undergoing standard myeloablative hematopoietic cell transplantation.


Subject(s)
Graft vs Host Disease/prevention & control , Hematopoietic Stem Cell Transplantation/adverse effects , Photopheresis/methods , Transplantation Conditioning/adverse effects , Acute Disease , Adolescent , Adult , Female , HLA Antigens , Hematologic Neoplasms/complications , Hematologic Neoplasms/therapy , Hematopoietic Stem Cell Transplantation/methods , Humans , Male , Middle Aged , Myeloablative Agonists/adverse effects , Survival Rate , Transplantation Conditioning/methods , Transplantation, Homologous , Treatment Outcome , Young Adult
11.
Bone Marrow Transplant ; 42(3): 201-5, 2008 Aug.
Article in English | MEDLINE | ID: mdl-18490913

ABSTRACT

Donor leukocyte infusions (DLI) can reverse relapse of hematologic malignancy following allogeneic hematopoietic stem cell transplant (HSCT) in some cases. Little is known regarding the effectiveness of DLI in children who relapse after HSCT. We report outcomes of 49 children who received DLI for relapse after allogeneic transplant. Prognosis was particularly poor (0/14 responses) for patients relapsing within 6 months from transplant. DLI rarely induced remission when given as sole therapy for marrow relapse. One-year disease-free survival was 30% (6/20) in patients who received DLI as consolidation following chemotherapy. The development of GVHD grades 1-2 was associated with superior 3-year survival than patients who developed GVHD grades 3-4 (P<0.002). To determine the benefit of DLI, 45 children who received DLI for relapse (four children without matches were excluded) were compared to 1229 children with similar characteristics whose relapse was not treated with DLI. There was no difference in survival (P=0.30) once adjustments were made to account for the time from relapse to DLI. Although a few children achieved durable remissions when DLI was used as part of a post-relapse treatment strategy, DLI was unsuccessful in the majority of cases. Strategies may be better directed at preempting post transplant relapse.


Subject(s)
Hematologic Neoplasms/surgery , Hematologic Neoplasms/therapy , Leukocyte Transfusion , Stem Cell Transplantation/methods , Acute Disease , Child , Combined Modality Therapy , Disease-Free Survival , Female , Graft vs Host Disease/epidemiology , Graft vs Tumor Effect , Humans , Leukemia/surgery , Leukemia/therapy , Leukemia, Myelogenous, Chronic, BCR-ABL Positive/surgery , Leukemia, Myelogenous, Chronic, BCR-ABL Positive/therapy , Male , Recurrence , Tissue Donors , Transplantation, Homologous
12.
Bone Marrow Transplant ; 41(7): 635-42, 2008 Apr.
Article in English | MEDLINE | ID: mdl-18084335

ABSTRACT

For adults with high-risk or recurrent ALL who lack a suitable sibling donor, the decision between autologous (Auto) and unrelated donor (URD) hematopoietic stem cell transplantation (HSCT) is difficult due to variable risks of relapse and treatment-related mortality (TRM). We analysed data from two transplant registries to determine outcomes between Auto and URD HSCT for 260 adult ALL patients in first (CR1) or second (CR2) CR. All patients received a myeloablative conditioning regimen. The median follow-up was 77 (range 12-170) months. TRM at 1 year post transplant was significantly higher with URD HSCT; however, there were minimal differences in TRM according to disease status. Relapse was higher with Auto HSCT and was increased in patients transplanted in CR2. Five-year leukemia-free (37 vs 39%) and overall survival (OS) rates (38 vs 39%) were similar for Auto HSCT vs URD HSCT in CR1. There were trends favoring URD HSCT in CR2. The long-term follow-up in this analysis demonstrated that either Auto or URD HSCT could result in long-term leukaemia-free survival and OS for adult ALL patients. The optimal time (CR1 vs CR2) and technique to perform HSCT remains an important clinical question for adult ALL patients.


Subject(s)
Bone Marrow Transplantation/methods , Neoplasm Recurrence, Local , Precursor Cell Lymphoblastic Leukemia-Lymphoma/therapy , Registries , Adolescent , Adult , Disease-Free Survival , Female , Graft Survival , Hematopoietic Stem Cell Transplantation/methods , Humans , Kaplan-Meier Estimate , Male , Middle Aged , Retrospective Studies , Transplantation, Autologous , Transplantation, Homologous , Treatment Outcome
13.
Bone Marrow Transplant ; 34(8): 721-7, 2004 Oct.
Article in English | MEDLINE | ID: mdl-15322568

ABSTRACT

Treatment options for persons with leukemia relapsing after allogeneic transplantation are limited. We analyzed the outcome of 279 patients with acute and chronic leukemia, who relapsed after HLA-identical sibling transplantation and received a second allogeneic transplant. The influence of potential risk factors on treatment-related mortality (TRM), relapse, treatment failure (relapse or death) and overall survival after second transplantation were assessed using proportional-hazards regression. The cumulative incidences (95% confidence interval) of relapse and TRM at 5 years were 42 (36-48)% and 30 (24-36)%, respectively. The 5-year probabilities of both overall and leukemia-free survival were 28 (23-34)%. In multivariate analyses, risks of treatment failure and mortality were lower in younger patients (< or =20 years) and patients who relapsed after 6 months from first transplantation. Risks of relapse were lower in patients who relapsed after 6 months from first transplantation and in complete remission prior to second transplantation. Risks of relapse were higher after reduced-intensity conditioning regimens. Any potential advantage of using a different matched related donor for a second transplantation is not supported by these data. Although age, disease status and conditioning regimen are important, duration of remission after first transplantation appear to be the most important determinant of outcome.


Subject(s)
Bone Marrow Transplantation/methods , Leukemia/therapy , Adolescent , Adult , Bone Marrow Transplantation/adverse effects , Child , Family , Female , Graft vs Host Disease , Hematopoietic Stem Cells/cytology , Histocompatibility Testing , Humans , Leukemia, Myelogenous, Chronic, BCR-ABL Positive/therapy , Leukemia, Myeloid, Acute/therapy , Linear Models , Male , Multivariate Analysis , Neutrophils/metabolism , Platelet Count , Precursor Cell Lymphoblastic Leukemia-Lymphoma/therapy , Proportional Hazards Models , Recurrence , Retrospective Studies , Risk , Risk Factors , Sex Factors , Siblings , Stem Cell Transplantation/adverse effects , Stem Cell Transplantation/methods , Time Factors , Transplantation, Homologous , Treatment Outcome
14.
Bone Marrow Transplant ; 32(2): 151-5, 2003 Jul.
Article in English | MEDLINE | ID: mdl-12838279

ABSTRACT

Metastatic breast cancer has been a common indication for autologous hematopoietic stem cell transplantation (HSCT). Previous reports indicate 3-year survival and progression-free survival (PFS) rates after autotransplant to be about 30 and 15%, respectively. Most deaths are from recurrent disease. One potential cause for high relapse rates is graft contamination with tumor. We describe 14 women with metastatic breast cancer transplanted between 1991 and 1998 with hematopoietic cells from identical twins. Median age was 41 y (range 34-50). Most women (12 of 14) were treated with mastectomy, and all received anthracycline-based regimens in their pretransplant course; nine women also received a taxane, seven radiotherapy and three hormonal therapy. Four women were in complete remission (one CR, three CRU) at transplant, five were in partial remission, two had stable disease and two had progressive disease. Eight women have died, one of treatment-related causes and seven of progressive breast cancer. Three-year survival was 48% (21-71%) and 3-year PFS was 21% (5-45%). Although the number of patients is small, outcomes for women transplanted with syngeneic grafts are similar to those of women receiving autologous grafts. This suggests that residual cancer in the patient is the major contributor to relapse after transplantation for breast cancer.


Subject(s)
Breast Neoplasms/therapy , Hematopoietic Stem Cell Transplantation/methods , Adult , Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Breast Neoplasms/mortality , Breast Neoplasms/pathology , Female , Hematopoietic Stem Cell Transplantation/mortality , Humans , Mastectomy , Middle Aged , Neoplasm Metastasis/therapy , Recurrence , Remission Induction/methods , Retrospective Studies , Survival Analysis , Transplantation, Isogeneic , Treatment Outcome , Twins, Monozygotic
15.
Bone Marrow Transplant ; 31(6): 417-21, 2003 Mar.
Article in English | MEDLINE | ID: mdl-12665834

ABSTRACT

Center effects are differences in outcome among treatment centers that cannot be explained by identifiable differences in patients treated or specific treatments applied and are presumed to result from differences in the ways health care is delivered. This paper will briefly review studies of association between treatment center factors and clinical outcomes in general medicine and surgery and look more closely at studies involving hematopoietic stem cell transplantation. We will also attempt to identify conceptual domains to study further the processes and mechanisms that may be associated with better outcomes.


Subject(s)
Hematopoietic Stem Cell Transplantation/mortality , Hospital Mortality/trends , Hospitals/statistics & numerical data , Humans , United States
16.
Exp Hematol ; 29(11): 1336-46, 2001 Nov.
Article in English | MEDLINE | ID: mdl-11698130

ABSTRACT

BACKGROUND: Autologous bone marrow transplantation is an important therapy for patients with acute myelogenous leukemia (AML). However, leukemia in the graft may contribute to posttransplant relapse. Treatment of the graft with 4-hydroperoxycyclophosphamide (4HC) is sometimes used to decrease numbers of infused leukemia cells (4HC purging). No large controlled trials evaluating efficacy and toxicity of 4HC purging are reported. METHODS: We studied 294 patients reported to the Autologous Blood and Marrow Registry receiving either a 4HC-purged (n = 211) or unpurged (n = 83) autograft for AML in first (n = 209) or second (n = 85) remission. Analyses were restricted to patients transplanted less than 6 months after achieving remission. Using Cox proportional hazards regression, we compared time to treatment failure (death or relapse, inverse of leukemia-free survival) after 4HC-purged vs unpurged transplants while controlling for important prognostic factors. RESULTS: Median duration of posttransplant neutropenia was 40 (range, 10-200) days after 4HC-purged transplants and 29 (9-97) days after unpurged transplants (p < 0.01). Transplant-related mortality was similar in the two groups. In multivariate analysis, patients receiving 4HC-purged transplants had lower risks of treatment failure than those receiving unpurged transplants (relative risk, 0.69, p = 0.12 in the first posttransplant year; relative risk, 0.28, p < 0.0001 thereafter). Adjusted three-year probabilities of leukemia-free survival (95% confidence interval) were 56% (47-64%) and 31% (18-45%) after 4HC-purged and unpurged transplants in first remission, respectively. Corresponding probabilities in second remission were 39% (25-53%) and 10% (1-29%). CONCLUSION: Grafts purged with 4HC are associated with higher leukemia-free survival after autologous bone marrow transplants for AML.


Subject(s)
Bone Marrow Purging/methods , Bone Marrow Transplantation/methods , Cyclophosphamide/analogs & derivatives , Leukemia, Myeloid/therapy , Acute Disease , Adolescent , Adult , Americas/epidemiology , Bone Marrow Transplantation/mortality , Child , Child, Preschool , Disease-Free Survival , Female , Graft Survival , Humans , Infant , Leukemia, Myeloid/mortality , Leukemia, Myeloid/pathology , Life Tables , Male , Middle Aged , Prognosis , Proportional Hazards Models , Registries , Remission Induction , Retrospective Studies , Risk , Survival Analysis , Transplantation, Autologous , Treatment Outcome
17.
Blood ; 98(12): 3205-11, 2001 Dec 01.
Article in English | MEDLINE | ID: mdl-11719355

ABSTRACT

Various therapeutic options are available for patients with chronic myelogenous leukemia. Allogeneic stem cell transplantation, though often curative, is associated with high nonrelapse mortality and long-term morbidity, particularly when cells from unrelated donors are used. Many physicians and patients opt for a trial of interferon-alpha (IFN)-based therapy first, reserving transplantation for patients with inadequate response or intolerance to IFN. Data were analyzed on 740 patients receiving unrelated donor transplants for chronic myelogenous leukemia in first chronic phase provided by the International Bone Marrow Transplant Registry and the National Marrow Donor Program to see whether IFN pretreatment compromised transplantation outcome. A total of 489 (66%) had received IFN prior to transplantation; 251 (34%) had not. Disease characteristics in the 2 groups were similar at diagnosis but at the time of transplantation, hematologic parameters and weight were lower in IFN patients and the interval between diagnosis and transplantation was longer. After adjustment for baseline covariates, no effect of IFN exposure was found on overall survival, leukemia-free survival, nonrelapse mortality, engraftment, relapse, or acute or chronic graft-versus-host disease. Evaluation of effects based on duration of therapy and time off IFN prior to transplantation was limited by missing data and confounding with IFN intolerance and disease responsiveness. In conclusion, no evidence was found for an independent adverse effect of IFN pretreatment on the outcome of subsequent unrelated donor transplantation.


Subject(s)
Hematopoietic Stem Cell Transplantation , Interferons/therapeutic use , Leukemia, Myelogenous, Chronic, BCR-ABL Positive/therapy , Treatment Outcome , Acute Disease , Adolescent , Adult , Antibodies, Viral/blood , Chronic Disease , Cytomegalovirus/immunology , Disease-Free Survival , Female , Graft vs Host Disease/epidemiology , Humans , Interferons/administration & dosage , Leukemia, Myelogenous, Chronic, BCR-ABL Positive/mortality , Male , Middle Aged , Recurrence , Registries , Survival Rate , Time Factors
18.
Oncology (Williston Park) ; 15(5): 649-59; discussion 663-4, 666, 2001 May.
Article in English | MEDLINE | ID: mdl-11396358

ABSTRACT

About 50,000 hematopoietic stem cell transplantations are performed yearly, primarily for malignancies. Use of this therapy increased dramatically over the past 30 years due to its proven and potential efficacy in diverse diseases, better understanding of appropriate timing of transplantation and patient selection, and greater availability of allogeneic donors. The International Bone Marrow Transplant Registry (IBMTR) and the Autologous Blood and Marrow Transplant Registry (ABMTR) collect data on consecutive allogeneic and autologous transplants, respectively, in more than 400 participating centers worldwide. The IBMTR/ABMTR database contains information on more than 120,000 transplant recipients. Among 11,347 patients transplanted in 101 IBMTR/ABMTR research centers in North America during 1995-1997, 66% received autologous transplants, 24% related-donor transplants, and 10% unrelated-donor transplants. More than 90% of transplantations were for malignant disease, with more than half of these done in patients with advanced disease. Of the recipients, 70% were younger than 50 years. Posttransplant survivals varied substantially by disease, transplant type, recipient age, and disease status at transplantation. IBMTR/ABMTR data provide an important tool for assessing transplant use and outcome, identifying prognostic factors for transplant outcomes, evaluating new transplant therapies, comparing transplant and nontransplant therapies, evaluating late transplant complications, and planning prospective phase II and III clinical trials.


Subject(s)
Hematopoietic Stem Cell Transplantation , Registries , Bone Marrow Transplantation/methods , Bone Marrow Transplantation/mortality , Data Collection/statistics & numerical data , HLA Antigens/analysis , HLA Antigens/therapeutic use , Hematologic Neoplasms/therapy , Hematopoietic Stem Cell Transplantation/methods , Hematopoietic Stem Cell Transplantation/mortality , Humans , Multicenter Studies as Topic , Survival Analysis , Time , Transplantation Conditioning/methods , Transplantation Conditioning/mortality , Transplantation, Autologous , Transplantation, Homologous , Treatment Outcome
19.
Stat Med ; 20(12): 1871-85, 2001 Jun 30.
Article in English | MEDLINE | ID: mdl-11406847

ABSTRACT

Multi-state models have proved versatile and useful in the statistical analysis of the complicated course of events after bone marrow transplantation. Working from data from the International Bone Marrow Transplant Registry, we show that summary probability calculations may be useful to explore hypothetical scenarios where some transition intensities are set by the researcher. A multi-state Markov process model is specified with six states: the initial state 0; acute; chronic and both acute and chronic graft-versus-host disease A, C and AC; relapse R and death in remission D. Transition rates between the states are estimated using Nelson-Aalen estimators and Cox regression models and combined to transition probability estimators using Aalen-Johansen product integration. Besides the estimated transition probabilities to D and R we explore hypothetical probabilities obtained by artificially changing certain transition intensities, with the general purposes of getting summary views of the development for actual patients 'in this world' and of exploring the intrinsic information from real patients about consequences of various changed conditions.


Subject(s)
Bone Marrow Transplantation/immunology , Graft vs Host Disease/etiology , Models, Biological , Proportional Hazards Models , Humans , Leukemia/therapy , Markov Chains , Predictive Value of Tests , Recurrence , Treatment Outcome
20.
Bone Marrow Transplant ; 27(7): 689-92, 2001 Apr.
Article in English | MEDLINE | ID: mdl-11360107

ABSTRACT

Donation-related data for 1488 allogeneic peripheral blood stem cell (PBSC) transplants reported to the International Bone Marrow Transplant Registry (IBMTR) or the European Blood and Marrow Transplant Group (EBMT) by 152 teams worldwide between 1994 and 1998 were reviewed. In 1998, 26% of allografts registered with the IBMTR were collected from blood. Median age of PBSC donors was 38 years (range <1-76), and 55% were male. Of 1486 donor-recipient pairs evaluable for HLA compatibility, 1322 (89%) were HLA-identical siblings. Recombinant human granulocyte colony-stimulating factor (G-CSF) was employed to mobilize PBSCs in almost all (99%) cases. One hundred and seventy (20%) of 828 evaluable PBSC donors had a central catheter placed for leukapheresis. Eighty-five percent of 1321 evaluable PBSC grafts were collected with one or two leukaphereses. There were 15 reported donation-related adverse events (1% of evaluable donors). Complications were catheter-related in five. No donation-related fatalities were reported. These data suggest that PBSC donation is becoming more prevalent worldwide. It appears to have a safety profile comparable to marrow harvesting, although experience with the latter is much more extensive.


Subject(s)
Blood Donors/statistics & numerical data , Hematopoietic Stem Cells , Adolescent , Adult , Aged , Catheterization, Central Venous/adverse effects , Child , Child, Preschool , Databases, Factual , Female , Hematopoietic Stem Cell Transplantation/adverse effects , Hematopoietic Stem Cell Transplantation/statistics & numerical data , Humans , Infant , Male , Middle Aged , Registries/statistics & numerical data , Safety
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