Survival Benefit Of Transplantation Research Articles

(58) - Outcomes and Survival Benefit of Lung Transplantation for Cystic Fibrosis: Single-Center Experience of 309 Consecutive Cases

(58) - Outcomes and Survival Benefit of Lung Transplantation for Cystic Fibrosis: Single-Center Experience of 309 Consecutive Cases

(429) - Is Bad Donor in Bad Recipient Leading To Bad Outcomes? Potential Utility of Scores To Achieve Optimal Donor-Recipient Match

(429) - Is Bad Donor in Bad Recipient Leading To Bad Outcomes? Potential Utility of Scores To Achieve Optimal Donor-Recipient Match

P203 Lung transplantation and survival in idiopathic pulmonary fibrosis - an Irish perspective

Introduction and ObjectivesIdiopathic pulmonary fibrosis (IPF) is a chronic, fibrosing interstitial pneumonia of unknown aetiology. Its clinical course is unpredictable but invariably leads to progressive respiratory failure and death. Median...

Assessing the Causal Effect of Organ Transplantation on the Distribution of Residual Lifetime

Because the number of patients waiting for organ transplants exceeds the number of organs available, a better understanding of how transplantation affects the distribution of residual lifetime is needed to improve organ allocation. However, there has been little work to assess the survival benefit of transplantation from a causal perspective. Previous methods developed to estimate the causal effects of treatment in the presence of time-varying confounders have assumed that treatment assignment was independent across patients, which is not true for organ transplantation. We develop a version of G-estimation that accounts for the fact that treatment assignment is not independent across individuals to estimate the parameters of a structural nested failure time model. We derive the asymptotic properties of our estimator and confirm through simulation studies that our method leads to valid inference of the effect of transplantation on the distribution of residual lifetime. We demonstrate our method on the survival benefit of lung transplantation using data from the United Network for Organ Sharing.

The Survival Benefit of Kidney Transplantation in Obese Patients

Obese patients have a decreased risk of death on dialysis but an increased risk of death after transplantation, and may derive a lower survival benefit from transplantation. Using data from the United States between 1995 and 2007 and multivariate non-proportional hazards analyses we determined the relative risk of death in transplant recipients grouped by body mass index (BMI) compared to wait-listed candidates with the same BMI (n = 208 498). One year after transplantation the survival benefit of transplantation varied by BMI: Standard criteria donor transplantation was associated with a 48% reduction in the risk of death in patients with BMI ≥ 40 kg/m2 but a ≥66% reduction in patients with BMI < 40 kg/m2. Living donor transplantation was associated with ≥66% reduction in the risk of death in all BMI groups. In sub-group analyses, transplantation from any donor source was associated with a survival benefit in obese patients ≥50 years, and diabetic patients, but a survival benefit was not demonstrated in Black patients with BMI ≥ 40 kg/m2. Although most obese patients selected for transplantation derive a survival benefit, the benefit is lower when BMI is ≥40 kg/m2, and uncertain in Black patients with BMI ≥ 40 kg/m2.

Survival Benefit of Lung Transplant for Cystic Fibrosis since Lung Allocation Score Implementation

The survival benefit of lung transplantation (LT) in adult patients with cystic fibrosis (CF) is debated. We sought to assess the survival benefit of LT in adult patients with CF. We used data from the United Network for Organ Sharing Registry to identify adult patients with CF on a wait list for LT in the United States between 2005 and 2009. Survival times while on the wait list and after LT were modeled by use of a Cox model that incorporated transplantation status as a time-dependent covariate. Evolution in lung allocation score (LAS) while on the wait list was used as a surrogate for disease severity. We fitted a model for the joint distribution of survival and longitudinal disease process (LAS over time). A total of 704 adult patients with CF were registered on a wait list during the study period. The cumulative incidence of LT was 39.3% (95% confidence interval, 35.6-42.9%) at 3 months and 64.7% (61.0-68.4%) at 12 months, whereas the incidence of death while on the wait list at the same times was 8.5% (6.4-10.6%) and 12.9% (10.3-15.5%), respectively. Survival after LT was 96.5% (94.7-98.2%) at 3 months; 88.4% (85.1-91.8%) at 12 months; and 67.8% (59.9-76.8%) at 3 years. LT conferred a 69% reduction in the instantaneous risk of death (51-80%). The interaction between LAS and LT was significant: the higher the LAS, the greater the survival benefit of LT (P < 0.001). LT confers a survival benefit for adult patients with CF.

Survival Benefit of Lung Transplantation in Japan: Extreme Donor Shortage and the Potential Role of Living Transplantation

Purpose Cadaveric lung transplantation (LTx) in Japan has multiple limitations including 1) extreme donor shortage (only 44 cadaveric brain-death donors with 37 cadaveric LTx cases across Japan in 2011); 2) limited recipient age below 55 for bilateral and 60 for single LTx; 3) priority of LTx determined solely by waiting time on the list. Thus, living LTx could be the only life-saving option for those who cannot wait for cadaveric LTx. We examined the survival benefit of LTx (either cadaveric or living) among those who were listed for cadaveric LTx in Japan. Methods and Materials The relative risks of death on the waiting list and death after LTx were evaluated using multivariate Cox models. Reception of LTx was treated as censored on the list. Since the initiation of the current Japanese listing system in 1998, patients who were listed by July 31, 2012 were included in the study. Results 617 patients were listed and 174 received LTx (139 cadaveric, 35 living). Average waiting time for cadaveric LTx on the list was 966±676 (10-4507) days. In multivariate Cox regression models, idiopathic pulmonary fibrosis (IPF) showed the most significant risk of death on waiting list (n=115; hazard ratio (HR), 3.53; confidence interval (CI), 2.34-5.31; reference, PAH), while the risk of death after LTx (n=23, including cadaveric (n=15) and living (n=8)) was moderate (HR, 0.85; CI, 0.36-2.01; ref, PAH). Treating the conditions of being on cadacveric-LTx list and being post-LTx as time-dependent covariates, the estimated survival benefit of LTx was most significant in IPF (HR, 0.26; CI, 0.097-0.746). This trend persisted in subanalyses of living and cadaveric LTx. Conclusions IPF showed the most significant survival benefit by LTx. This is explained by their poor prognosis without LTx and reasonable outcome after LTx. Adult-to-adult living LTx, which appears suitable for relatively old IPF patients with small thorax, remains the only life-saving option for many IPF patients who cannot wait for cadaveric LTx.

La répartition des greffons cardiaques

Until 2004, French rules for cardiac graft allocation were aimed at ensuring uniform organ distribution on the national scale, while logically giving priority to local teams. The allocation was center-based, with transplant MDs choosing the most suitable recipient from the local waiting list. In 2004, two national priority lists were added to the French cardiac graft allocation system. High Emergency 1 (HE1) is for patients with unstable hemodynamic status (on inotropes or ECMO), while High Emergency 2 (HE2) is for patients who develop complications during long-term circulatory support. Data from the French Biomedicines Agency show significantly poorer survival for HE1 patients than for HE2 patients. However, this should not lead to the conclusion that HE2 is a better strategy, as mortality during long-term circulatory support is not taken into account, and it is well known that the survival benefit of transplantation is significantly better in HE1 patients. Furthermore, the risk related to the quality of the cardiac graft must be taken into account. Indeed, the decision to transplant or not to transplant an HE1 patient with a given graft will impact not only the survival of the patient concerned, but also the waiting time of the other patients on the list, independently of allocation rules. This is also true for the criteria used to place a patient on the waiting list. Each cardiac transplantation team considers the level of risk before adding a patient to the waiting list, and this will impact not only the individual candidate but also the entire waiting list. Thus, even if allocation rules aim to provide all patients with the same chance of being transplanted across the entire country, the medical decision to accept a cardiac graft and to register a patient on the waiting list will make this process somewhat heterogeneous. This allows cardiac transplantation to be adapted to each individual case, independently of the rules. However, a multidisciplinary decision process is necessary to ensure fairness.

Distinctive disease subgroups according to differentiation stages in adult patients with T‐cell acute lymphoblastic leukemia

The categorization of T-cell acute lymphoblastic leukemia (T-ALL) into four subgroups according to the degree of thymic differentiation was proposed in 1995, and this categorization scheme has been described in the World Health Organization classification 4th edition with minor changes. The aim of this study is to explore the clinical significance of leukemia cell differentiation stages in patients with T-ALL. We analyzed 36 adult T-ALL patients, including six patients (17%) in pro-T stage, 16 (44%) in pre-T stage, three (8%) in cortical-T stage, and 11 (31%) in medullary-T stage. Pro-T and pre-T stages were arbitrarily clustered as the immature group, and cortical-T and medullary-T stages as the mature group. Patients in the immature group had unique presentations, including lower lactate dehydrogenase levels, lower frequency of mediastinal tumor, and higher expression of myeloid antigens than the mature group. There was no difference in the treatment strategies between both groups. Although patients in the immature group had a lower complete remission (CR) rate when compared with the mature group (45% vs. 79%, respectively; P=0.04), the 3-yr overall survival (OS) for both groups was comparable (27% vs. 38%, respectively; P=0.66). Such discrepancy between the CR rates and the OS could be partially explained by survival benefit of allogeneic transplantation observed in the immature group but not in the mature group. These findings indicate that T-ALL patients can be categorized into two biologically distinctive subgroups according to the differentiation stages, and this stratification might enable prospective identification of patients with poor chemotherapy response.

Abstracts for the V SLIMP - IV LASRI Meeting 2013

Survival benefit (SB) for first liver transplantation (LT) is favorable at Model for End-Stage Liver Disease (MELD) ≥15. Herein, we identify the MELD threshold for SB from repeat liver transplantation (ReLT) by recipient hepatitis C virus (HCV) status and donor risk index (DRI). We analyzed lab MELD scores in new United Network for Organ Sharing registrants for ReLT from March 2002 to January 2010. Risk of ReLT graft failure ≤1 year versus waitlist mortality was calculated using Cox regression, adjusting for recipient characteristics. Of 3057 ReLT candidates, 54% had HCV and 606 died while listed. There were 1985 ReLT recipients, 52% had HCV and 567 ReLT graft failures by 1 year. Unadjusted waitlist mortality and post-ReLT graft failure rates were 416 (95% confidence interval [CI] 384–450) and 375 (95% CI 345–407) per 1000 patient-years, respectively. Waitlist mortality was higher with increasing waitlist MELD (p < 0.001). The MELD for SB from ReLT overall was 21 (21 in non-HCV and 24 in HCV patients). MELD for SB varied by DRI in HCV patients (MELD 21, 24 and 27 for low, medium and high DRI, respectively) but did not vary for non-HCV patients. Compared to first LT, ReLT requires a higher MELD threshold to achieve an SB resulting in a narrower therapeutic window to optimize the utility of scarce liver grafts.

Model for end-stage liver disease-sodium and survival benefit in liver transplantation

There are currently no studies calculating the survival benefit of liver transplantation (LT) according to model for end-stage liver disease-sodium (MELD-Na) and based on the competing risk (CR) method. We enrolled consecutive adult patients with chronic end-stage liver disease entering the waiting list (WL) for primary LT (WL group=337) and undergoing LT (LT group=220) in the period 2006-2009. Two independent multivariable regressions (WL and LT models) were created to measure the prognostic power of MELD-Na with respect to MELD. For the WL model, both Cox and CR multivariable analyses were performed. Estimates were finally included in a Markov model to calculate 3-year survival benefit. WL Cox model: MELD-Na (P<0.0001) and MELD (P<0.0001) significantly predicted survival. WL CR model: MELD-Na (P=0.0045) and MELD (P=0.0109) significantly predicted survival. LT Cox model: MELD-Na (P=0.7608) and MELD score (P=0.9413) had not correlation with survival. Benefit model: MELD and MELD-Na had an overlapping significant impact on 3-year survival benefit; CR method determined a significant decrease in 3-year life expectancy (LE) estimations. MELD-Na and MELD scores similarly predicted 3-year LT survival benefit, but the gain in LE is significantly lower when a CR method is adopted.

Potential impact of sorafenib on the survival benefit of liver transplantation for hepatocellular carcinoma

Liver transplantation (LT) theoretically provides the best treatment for patients with hepatocellular carcinoma (HCC) since it removes both the tumour and the underlying cirrhosis [ [1] Roayaie S. Schwartz J.D. Sung M.W. et al. Recurrence of hepatocellular carcinoma after liver transplant: patterns and prognosis. Liver Transpl. 2004; 10: 534-540 Crossref PubMed Scopus (363) Google Scholar ].

Are Patients with Child’s A Cirrhosis and Hepatocellular Carcinoma Appropriate Candidates for Liver Transplantation?

We aimed to estimate the survival benefit derived from transplantation in patients with stage II hepatocellular carcinoma (HCC) and Child's A cirrhosis, defined as the mean lifetime with transplantation minus the mean lifetime with treatments other than transplantation. We calculated the posttransplantation survival of all adult, first-time, deceased-donor, liver transplant recipients in the United States since the introduction of the Model for End-Stage Liver Disease based priority system in February 2002 (n = 36,791). We estimated the posttreatment survival of patients with Child's A cirrhosis and stage II HCC treated by radiofrequency ablation (RFA) ± transarterial chemoembolization (TACE) or surgical resection by conducting a systematic review of the medical literature. In patients with Child's A cirrhosis and stage II HCC, the estimated median survival benefit of liver transplantation compared to RFA ± TACE was 1.5 months at 3 years (range -3.5 to 5.6) and 5.7 months at 5 years (range 0.7-11.4), whereas compared to surgical resection it was 0.7 months at 3 years (range -2.9 to 3) and 2.8 months at 5 years (range -4.4 to 5.7). Liver transplantation in patients with stage II HCC and Child's A cirrhosis results in a very low survival benefit and may not constitute optimal use of scarce liver donor organs.

T-40 Alphafetoprotein and transplant survival benefit for patients with hepatocellular carcinoma: a multicentre cohort study

T-40 Alphafetoprotein and transplant survival benefit for patients with hepatocellular carcinoma: a multicentre cohort study

A Decision Analysis of Reduced-Intensity Conditioning Allogeneic Hematopoietic Stem Cell Transplantation for Older Patients with De-Novo Myelodysplastic Syndrome (MDS): Early Transplantation Offers Survival Benefit in Higher-Risk MDS

Abstract 115Reduced-intensity conditioning (RIC) allogeneic hematopoietic stem cell transplantation is a curative approach for older patients with myelodysplastic syndrome (MDS), but is associated with early mortality risk. We previously used decision modeling to define the role of myeloablative transplantation in younger MDS patients aged <60 years, stratified by IPSS risk (Cutler et al, Blood 2004). We thereafter undertook a similar analysis to define the role of RIC transplantation in older MDS patients.We constructed a Markov decision model in de-novo MDS patients aged 60–70 years to evaluate life expectancy after a strategy of early T-replete RIC transplantation versus standard-of-care non-transplantation approaches. Patients with chronic myelomonocytic leukemia, isolated 5q-, unclassifiable and therapy-related MDS were excluded. Patients undergoing transplantation with anti-thymocyte globulin or alemtuzumab-based conditioning or HLA mismatched or umbilical cord donors were also excluded. We undertook adjustments for quality-of-life (QoL) using standard utility estimates for different MDS and transplantation states (0.84: RBC transfusion-independent low/intermediate-1 MDS; 0.6: RBC transfusion-dependent low/intermediate-1 MDS; 0.53: intermediate-2/high MDS; 0.92: post transplantation-overall; 0.6: post transplantation-GVHD)We evaluated survival of 92 patients after HLA matched RIC transplantation (Dana-Farber, Fred Hutchinson and CIBMTR datasets) stratified by IPSS risk state, versus survival with: best supportive care for 183 non-anemic low/intermediate-1 IPSS patients (IMRAW and Pavia datasets); hematopoietic growth factors for 78 anemic low/intermediate-1 IPSS patients (Nordic MDS Group and GFM datasets); and hypomethylating agents for 160 intermediate-2/high IPSS risk patients (Celgene AZA-001, GFM compassionate use azacytidine and M. D. Anderson decitabine datasets).For older patients with low/intermediate-1 IPSS risk, early RIC transplantation impaired life expectancy compared to non-transplantation approaches (Table). Excluding patients transplanted beyond 12 months from diagnosis did not alter the conclusion. QoL adjustment narrowed the gap in quality adjusted life expectancy (QALE), but sensitivity analysis did not support RIC transplantation as the preferred strategy across a broad range of possible utility estimates (0.5–1), indicating that MDS-associated morbidity (e.g. RBC transfusion-dependence) does not favor early transplantation unless its QoL impact is substantial (state utility <0.4).For older patients with intermediate-2/high IPSS risk, early RIC transplantation improved life expectancy compared with hypomethylating agents (Table). Excluding patients transplanted beyond 12 months from diagnosis did not alter the conclusion. However, transplantation is associated with early mortality such that the life expectancy benefit was apparent only after modeling survival beyond 5 years. Importantly, QoL adjusted survival benefit of transplantation was apparent both at 5 years and beyond, highlighting the morbidity of higher-risk MDS. In sensitivity analyses, QoL adjustment across the range of plausible transplantation-associated utility estimates (0.5–1) did not change the conclusion of QALE benefit.We conclude that for de-novo MDS patients aged 60–70 years with low/intermediate-1 disease, early transplantation is not the preferred strategy unless MDS-associated QoL impairment is substantial. For intermediate-2/high IPSS risk, early RIC transplantation offers a life expectancy benefit, with quality adjusted survival benefit detectable earlier.TableEarly RIC transplantationNo early RIC transplantationLow/intermediate-1 IPSSOverall Life Expectancy (months)3877QALE: Transfusion-independent MDS morbidity (months)3565QALE: Transfusion-dependent MDS morbidity (months)3546Intermediate-2/high IPSSOverall Life Expectancy (months)3628QALE: Higher-risk MDS morbidity (months)3315QALE: GVHD morbidity (months)2215 Disclosures:Fenaux:Celgene: Honoraria, Research Funding. Hellstrom-Lindberg:Celgene: Consultancy, Research Funding. Gale:celgene: Employment; unitedhealthcare: Consultancy; oxford health plans: Consultancy. Beach:celgene: Employment. Greenberg:novartis: Consultancy, Research Funding; glaxosmithkline: Research Funding; onconova: Research Funding; amgen: Consultancy, Research Funding.

Impact of liver transplantation on the survival of patients treated for hepatorenal syndrome type 1*

The development of hepatorenal syndrome type 1 (HRS1) is associated with a poor prognosis. Liver transplantation improves this prognosis, but the degree of the improvement is unclear. Most patients receive vasoconstrictors such as terlipressin before transplantation, and this may affect the posttransplant outcomes. We examined a cohort of patients with access to liver transplantation from our previously published study of terlipressin plus albumin versus albumin alone in the treatment of HRS1. The purpose of this analysis was the quantification of the survival benefits of liver transplantation for patients with HRS1. Ninety-nine patients were randomized to terlipressin or placebo. Thirty-five patients (35%) received a liver transplant. Among those receiving terlipressin plus albumin, the 180-day survival rates were 100% for transplant patients and 34% for nontransplant patients; among those receiving only albumin, the rates were 94% for transplant patients and 17% for nontransplant patients. The survival rate was significantly better for those achieving a reversal of hepatorenal syndrome (HRS) versus those not achieving a reversal (47% versus 4%, P < 0.001), but it was significantly lower for the responders versus those undergoing liver transplantation (97%). We conclude that the use of terlipressin plus albumin has no significant impact on posttransplant survival. Liver transplantation offers a clear survival benefit to HRS1 patients regardless of the therapy that they receive or the success or failure of HRS reversal. The most likely benefit of terlipressin in patients undergoing liver transplantation for HRS1 is improved pretransplant renal function, and this should make the posttransplant management of this difficult group of patients easier. For patients not undergoing transplantation, HRS reversal with terlipressin and/or albumin improves survival.

Liver transplant recipient survival benefit with living donation in the model for endstage liver disease allocation era

Receipt of a living donor liver transplant (LDLT) has been associated with improved survival compared with waiting for a deceased donor liver transplant (DDLT). However, the survival benefit of liver transplant has been questioned for candidates with Model for Endstage Liver Disease (MELD) scores <15, and the survival advantage of LDLT has not been demonstrated during the MELD allocation era, especially for low MELD patients. Transplant candidates enrolled in the Adult-to-Adult Living Donor Liver Transplantation Cohort Study after February 28, 2002 were followed for a median of 4.6 years. Starting at the time of presentation of the first potential living donor, mortality for LDLT recipients was compared to mortality for patients who remained on the waiting list or received DDLT (no LDLT group) according to categories of MELD score (<15 or ≥ 15) and diagnosis of hepatocellular carcinoma (HCC). Of 868 potential LDLT recipients (453 with MELD <15; 415 with MELD ≥ 15 at entry), 712 underwent transplantation (406 LDLT; 306 DDLT), 83 died without transplant, and 73 were alive without transplant at last follow-up. Overall, LDLT recipients had 56% lower mortality (hazard ratio [HR] = 0.44, 95% confidence interval [CI] 0.32-0.60; P < 0.0001). Among candidates without HCC, mortality benefit was seen both with MELD <15 (HR = 0.39; P = 0.0003) and MELD ≥ 15 (HR = 0.42; P = 0.0006). Among candidates with HCC, a benefit of LDLT was not seen for MELD <15 (HR = 0.82, P = 0.65) but was seen for MELD ≥ 15 (HR = 0.29, P = 0.043). Across the range of MELD scores, patients without HCC derived a significant survival benefit when undergoing LDLT rather than waiting for DDLT in the MELD liver allocation era. Low MELD candidates with HCC may not benefit from LDLT.

Peak Oxygen Uptake Correlates With Survival Without Clinical Deterioration in Ambulatory Children With Dilated Cardiomyopathy

Children stable at home with dilated cardiomyopathy remain at risk of death; there is evidence of survival benefit for transplantation out to 4 years postoperatively. The limited supply of donor organs makes risk stratification imperative, but although cardiopulmonary exercise test is well established as a powerful tool in adults with heart failure, no published studies have linked oxygen uptake to prognosis in children. Between 2001 and 2009, using cardiopulmonary exercise test and echocardiography, we studied 82 children (mean age, 13.5±2.3 years) with dilated cardiomyopathy. All were ambulatory, outpatients, and >120 cm in height. All children completed a symptom-limited maximal exercise test. Resting left ventricular shortening fraction was 20±9%; peak heart rate was 87±13% of predicted; peak oxygen uptake (VO(2)) was 67±22% of predicted; and ventilatory efficiency was 32±8. Follow-up was available for 100% of the children, and was a mean of 32.3±7.5 months. Eighteen patients reached the defined clinical end point of death or listing for urgent heart transplantation. On univariate analysis, left ventricular shortening fraction, peak heart rate, peak VO(2), peak systolic blood pressure, and ventilatory efficiency were all associated with adverse outcome. On multivariable Cox analysis, only peak VO(2) (P=0.003) was associated with the study end point. Patients with a peak VO(2) ≤62% of predicted had a higher 24-month event rate (50.6% versus 4.4%; hazard ratio, 10.78). We have demonstrated that a cardiopulmonary exercise test is feasible in ambulatory children with dilated cardiomyopathy who are >120 cm height and for the first time have linked peak VO(2) with outcome in children.

Age is an important predictor of kidney transplantation outcome

Donor and recipient age may have an impact on the renal transplant outcome. Kidney transplantation from older donors may result in a worse outcome, and the survival benefit of kidney transplantation compared with dialysis may be reduced. The aim of this study was to evaluate the impact of donor and recipient age on kidney transplant outcome. Two hundred and twenty-three recipients of kidney transplants performed at our institution between 2002 and 2007 were analysed. The role of donor and recipient age matching on survival rate were investigated performing the Kaplan-Meier survival time analysis by decades, considering the donor's age of 60 and 70 years. The Cox proportional hazard uni- and multivariate regressions were also performed. Finally, Kaplan-Meier survival time analysis was performed to assess survival rates of patients transplanted stratified by donor age compared with wait-listed renal transplant candidates. Elderly recipients had a significant lower graft and patient survival as well as a significantly higher risk of graft loss and patient death. Recipients younger and older than 65 years of age were at higher risk of graft loss if they received grafts from donors>65 years [hazard ratio (HR)=2.59, 95% confidence interval (CI): 1.12-6 and HR=5.65, 95% CI: 2.31-13.79, respectively]. Elderly recipients displayed a worse survival compared with transplant candidates on the waiting list. Age is an important predictor of kidney transplantation outcome. Kidney transplantation does not offer a significant survival benefit in the intermediate term, compared to the waiting list, to elderly recipients transplanted with grafts from older donors. However, it cannot be excluded that it is still possible that there is a long-term benefit of transplantation over dialysis in this group of patients.

Survival benefit of lung transplantation in individuals with severe α1-anti-trypsin deficiency (PiZZ) and emphysema

The objective of lung transplantation (LTx) is to prolong life, but the survival benefit for patients with severe α(1)-anti-trypsin deficiency (PiZZ) and emphysema is still unclear. The aim of this study was to assess whether PiZZ patients who have undergone lung transplantation (the lung transplant group, TxG) do better than patients who have continued on the usual medical therapy (the non-transplant group, NTxG). Between 1990, when the first patient received a lung transplant in Sweden, until June 2010, a total of 83 PiZZ patients with severe emphysema underwent transplantation. Seventy appropriate controls were identified from the Swedish National AAT Deficiency Registry. Each control was matched with a patient who had received a lung transplant, for age, gender, smoking history (number of pack-years) and lung function at the time of transplantation. Both controls and lung transplant patients had low spirometric values with a mean FEV(1) of 23 ± 6% and 22 ± 9% of predicted value, respectively (not a statistically significant difference). Of the 83 transplant patients, 62 (75%) underwent single-lung transplantation (SLTx). During follow-up, 37 (45%) deaths occurred in the TxG and 45 (64%) in the NTxG. In the TxG, the estimated median survival time was 11 years (95% confidence interval [CI] 9 to 14 years), compared with 5 years (95% CI 4 to 6 years) for the NTxG (p = 0.006). The most common cause of death was pulmonary infection among the transplant patients (38%) and respiratory failure (60%) among the controls. Lung transplantation significantly improves long-term survival of patients with severe α(1)-anti-trypsin deficiency (PiZZ) and emphysema.