Long Term Outcomes of Hematopoietic Stem Cell Transplantation in Patients with Severe Phenotype Hurler Syndrome: an International Multi-Center Study

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 1958-1958
Author(s):  
Mieke Aldenhoven ◽  
Maria Escolar ◽  
Robert Wynn ◽  
Ed Wraith ◽  
Anne O'Meara ◽  
...  
Keyword(s):  
Mixed Chimerism ◽  
Hurler Syndrome ◽  
Long Term Outcomes ◽  
Term Outcome ◽  
Hematopoietic Stem ◽  
Long Term Outcome ◽  
Donor Chimerism ◽  
Normal Enzyme

Abstract Abstract 1958 Background: Hurler syndrome (HS), the most severe phenotype in the spectrum of Mucopolysaccharidosis type I, is caused by a deficiency of the lysosomal enzyme alpha-L-iduronidase. HS is clinically characterized by a progressive and ultimately fatal multi-system deterioration with involvement of the central nervous system. At present, hematopoietic stem cell transplantation (HSCT) is the only treatment that prevents disease progression in the central nervous system and is therefore considered the treatment of choice in HS. Long-term follow-up of outcomes of HSCT for HS are sparse and risk factors for favorable long-term outcomes are still largely unknown. Therefore, an international multicenter study was initiated to describe the long-term outcomes of successfully transplanted HS patients. Methods: HS-patients transplanted between 1980 and 2007 within the leading transplantation centers in Europe and the United States were include in this study. Patient, donor, and transplantation-related variables which may influence long-term outcome were analyzed. Patients who were ‘alive and engrafted (donor-chimerism >10%)’ with a follow up of at least three years after HSCT were included. The functional outcomes assessed for the various organ systems - orthopedic, cardiac, ophthalmologic, respiratory and audiologic - were analyzed using multivariate Cox proportional hazards and logistic regression models. Results: 197 Hurler patients were included from 8 different transplant centers. This is estimated to be about 70–80% of the successfully transplanted HS patients worldwide during that time period. These patients had a median age of 16 (2–80) months at HSCT with a median follow up of 88 (36–258) months after successful HSCT. Seventy-nine % of the patients received a graft from an unaffected (non-carrier) donor. Seventy-two % of the patients achieved full (>95%)-donor-chimerism and 28% mixed-chimerism. After HSCT, normal enzyme-levels (EL; according to the local reference range) were found in 75% of the patients while 25% had EL below lower limit of normal; either due to mixed-chimerism or carrier-donorship). Multivariate analyses (table 1) showed having a “normal EL” after HSCT and younger (below the median age of 16mths) “age at transplantation” were associated with less serious orthopedic complications requiring surgical interventions; e.g. cord compression, genu-valgum surgery, carpal tunnel surgery. Genotype (double non-sense vs. any other genotype) was associated with a lower probability of requiring hip dysplasia surgery as well as with the occurrence of retinopathy. For other endpoints; e.g progression valve insufficiency, progression corneal clouding and development of retinopathy and the need for hearing aids having a normal EL as well as age at HSCT (<16mths) were predictors for better outcome. Furthermore, growth at the age of 60mths was influenced by EL (−1.93 SDS vs. −1,09 SDS; p=0.042). Conclusion: The long-term outcome of clinical manifestations in HS-patients after successful HSCT is promising although residual disease burden remains. Predictors, favorably influencing the long-term outcomes are suggested to be 1) enzyme level (normal vs. below LLN) after HSCT, 2) genotype and 3) age at HSCT. Achieving normal enzyme levels at an early age might significantly impact the prognosis of Hurler syndrome patients. Newborn screening (resulting in early HSCT), the use of non-carrier donors and achieving full-donor chimerism may be crucial in optimizing long-term outcomes. Disclosures: No relevant conflicts of interest to declare.

scholarly journals An update of the long-term outcome of patients with nonspecific pleurisy at medical thoracoscopy

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Yan-Xia Yu ◽  
Yuan Yang ◽  
Yan-Bing Wu ◽  
Xiao-Juan Wang ◽  
Li-Li Xu ◽  
...  
Keyword(s):  
Pleural Effusion ◽  
Malignant Disease ◽  
Long Term Outcomes ◽  
Benign Diseases ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Medical Thoracoscopy ◽  
One Year

Abstract Background Medical thoracoscopy (MT) is recommended in patients with undiagnosed exudative pleural effusion and offers a degree of diagnostic sensitivity for pleural malignancy. However, not all patients who undergo MT receive an exact diagnosis. Our previous investigation from 2014 summarized the long-term outcomes of these patients with nonspecific pleurisy (NSP); now, we offer updated data with the goal of refining our conclusions. Methods Between July 2005 and August 2018, MT with pleural biopsies were performed in a total of 1,254 patients with undiagnosed pleural effusions. One hundred fifty-four patients diagnosed with NSP with available follow-up data were included in the present study, and their medical records were reviewed. Results A total of 154 patients were included in this study with a mean follow-up duration of 61.5 ± 43.7 months (range: 1–180 months). No specific diagnosis was established in 67 (43.5%) of the patients. Nineteen patients (12.3%) were subsequently diagnosed with pleural malignancies. Sixty-eight patients (44.2%) were diagnosed with benign diseases. Findings of pleural nodules or plaques during MT and the recurrence of pleural effusion were associated with malignant disease. Conclusions Although most NSP patients received a diagnosis of a benign disease, malignant disease was still a possibility, especially in those patients with nodules or plaques as noted on the MT and a recurrence of pleural effusion. One year of clinical follow-up for NSP patients is likely sufficient. These updated results further confirm our previous study’s conclusions.

scholarly journals Hyperglycemia in the Posttransplant Period: NODAT vs Posttransplant Diabetes Mellitus

2018 ◽  
Vol 2 (11) ◽  
pp. 1314-1319 ◽  
Author(s):  
Suruchi Gupta ◽  
Teresa Pollack ◽  
Candice Fulkerson ◽  
Kathleen Schmidt ◽  
Diana Johnson Oakes ◽  
...  
Keyword(s):  
Insulin Resistance ◽  
Liver Transplant ◽  
Controlled Trial ◽  
Organ Transplant ◽  
Long Term Outcomes ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Persistent Hyperglycemia

Abstract Objective To characterize the types of hyperglycemia that occur up to 1 year following liver transplant and to clarify the nomenclature for posttransplant hyperglycemia. Design We analyzed 1-year glycemic follow-up data in 164 patients who underwent liver transplant and who had been enrolled in a randomized controlled trial comparing moderate to intensive insulin therapy to determine if patients had preexisting known diabetes, transient hyperglycemia, persistent hyperglycemia, or new-onset diabetes after transplantation (NODAT). Results Of 119 patients with posttransplant hyperglycemia following hospital discharge, 49 had preexisting diabetes, 5 had insufficient data for analysis, 48 had transient hyperglycemia (16 resolved within 30 days and 32 resolved between 30 days and 1 year), 13 remained persistently hyperglycemic out to 1 year and most likely had preexisting diabetes that had not been diagnosed or insulin resistance/insulinopenia prior to transplant, and 4 had NODAT (i.e., patients with transient hyperglycemia after transplant that resolved but then later truly developed sustained hyperglycemia, meeting criteria for diabetes). Conclusions Distinct categories of patients with hyperglycemia following organ transplant include known preexisting diabetes, persistent hyperglycemia (most likely unknown preexisting diabetes or insulin resistance/insulinopenia), transient hyperglycemia, and NODAT. Those with preexisting diabetes for many years prior to transplant may well have very different long-term outcomes compared with those with true NODAT. Therefore, it would be prudent to classify patients more carefully. Long-term outcome studies are needed to determine if patients with true NODAT have the same poor prognosis as patients with preexisting diabetes (diagnosed and undiagnosed) undergoing transplant.

scholarly journals Long-Term Outcomes After Reversible Cerebral Vasoconstriction Syndrome

Stroke ◽  
2020 ◽  
Vol 51 (2) ◽  
pp. 670-673 ◽  
Author(s):  
Rosalie Boitet ◽  
Solène de Gaalon ◽  
Claire Duflos ◽  
Grégory Marin ◽  
Jérôme Mawet ◽  
...  
Keyword(s):  
Thunderclap Headache ◽  
Reversible Cerebral Vasoconstriction Syndrome ◽  
Long Term Outcomes ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Follow Up Study ◽  
Cerebral Vasoconstriction ◽  
History Of

Background and Purpose— We aimed to further investigate the long-term outcomes after reversible cerebral vasoconstriction syndrome (RCVS). Methods— A longitudinal follow-up study was conducted in 173 RCVS patients. Results— Of the 172 patients who completed a mean follow-up of 9.2±3.3 years, 10 had a recurrent RCVS that was benign in all. Independent predictors of relapse were having a history of migraine and having exercise as a trigger for thunderclap headache during initial RCVS. After new delivery, the rate of postpartum RCVS was 9%. Conclusions— Overall, long-term outcome after RCVS is excellent.

Results of Reinsertion of the Distal Achilles Tendon

2018 ◽  
Vol 157 (03) ◽  
pp. 246-253 ◽  
Author(s):  
Heinz Lohrer
Keyword(s):  
Achilles Tendon ◽  
Long Term Outcomes ◽  
Heel Spur ◽  
Tendon Tear ◽  
Double Row ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Tendon Tears

Abstract Background Reattachment of the distal Achilles tendon to the posterior calcaneus following distal Achilles tendon tears/avulsions or after excision of large posterior heel spurs is a challenging task for the orthopaedic surgeon. Patients/Material and Methods Between 2005 and the end of 2015, 22 patients who underwent repair/reconstruction of a lesion of the distal Achilles tendon were identified from our electronic records. Calcaneal reinsertions were performed following distal Achilles tendon tears/avulsions (n = 15) or following excision of a large symptomatic posterior heel spur (n = 7). The respective outcome was evaluated comparatively using the VISA-A – G (Victorian Institute of Sports Assessment-Achilles tendon, German version) questionnaire (100 = maximum). Data were collected preoperatively (n = 11/22), and prospectively at three, six, and 12 months postoperatively (n = 5 – 11/22). Two further retrospective follow-ups were performed 12 – 114 and 21 – 149 months postoperatively (n = 17/22 and 22/22, respectively). All data were analysed retrospectively. Complications were searched from the electronic files. Results Transosseous sutures and different anchor techniques (Panalok®, Corkscrew®, SpeedBridge®) were generally performed. Preoperatively, the VISA-A – G score was 27.3 ± 13.5 (6 – 45) following distal Achilles tendon reinsertions for avulsions and 45.3 ± 49.0 (0 – 100) when reattachment was performed after resection of a large posterior heel spur (p = 0.831). At the final follow-up, VISA-A – G values were 89.4 ± 13.9 (54 – 100) following distal Achilles tendon tear/avulsion and 82.5 ± 24.5 (51 – 100), when Achilles tendons were reattached after posterior heel spur excisions (p = 0.969). There were no complications in the “heel spur group”. In the Achilles tendon tear/avulsion group, four out of seven patients with Panalok® repairs developed a fistula. One patient suffered a thromboembolism. None of these complications affected the long-term outcome. Conclusions This study demonstrates good long-term outcomes after distal Achilles tendon reinsertion. Knotless double row anchor repair provides a greater area of compression, simplifies and standardises the repair/reconstruction, and provides safety against fistula. These implants are therefore recommended for safe and effective reattachment of the distal Achilles tendon.

Long Term Outcome After Low Dose TBI Based Conditioning Hematopoietic Stem Cell Transplantation (HSCT) From Related and Unrelated Donors for Older Patients with AML

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 2030-2030
Author(s):  
Dietger Niederwieser ◽  
Leo F Verdonck ◽  
Jan J Cornelissen ◽  
Michael Cross ◽  
Rainer Krahl ◽  
...  
Keyword(s):  
De Novo ◽  
Chronic Gvhd ◽  
Disease Free Survival ◽  
Study Groups ◽  
Long Term Results ◽  
Term Outcome ◽  
Hematopoietic Stem ◽  
Long Term Outcome

Abstract Abstract 2030 HSCT after reduced or minimal intensity conditioning is increasingly used to treat AML patients not eligible for conventional HSCT. Short term outcome has been reported frequently and risk factors have been identified; long term results still await in depth evaluation. We report here 5 years follow up results from a prospective phase II study conducted by two AML study groups in Europe. Patients were recruited from AML protocols HOVON/SAKK AML 43 and the OSHO AML 1997 study. The regimen consisted of fludarabine (FLU), 30 mg/m2/d on days −4, −3, and −2, 2 Gy TBI on day 0 (the day of HSCT) with mycophenolate mofetil, [15 mg/kg p.o. b.i.d. from 5 hours after HSCT to day +40], and cyclosporine, [CSP; 6.25 mg/kg p.o. bid from day –3 to day +180] after HSCT. Cyclosporine was adjusted to trough levels and reduced according to a predetermined tapering schedule and donor type. A total of 96 patients were recruited between 5/2002 and 8/2005 in the study. Age was median 62 (range 40 – 74) years, 54 patients were male (56%) and 73 patients (76%) had de novo AML. The remission status on entry was CR1 in 83 (86%) patients and CR2 in 13 (14%). Of the 96 patients, 20% had high risk cytogenetics and SCT was performed a median of 75 days after chemotherapy. There were no statistical differences in the above described characteristics except for more secondary AML (p=0.04) and more CR2 patients (p=0.07) among the 59 unrelated SCT (61%) as compared to the 37 related SCT (39%). Graft rejection at two years was observed in 6% of the patients. Absence of chronic GvHD was diagnosed in 40% and limited chronic GvHD in 29% of the patients, with no difference between related and unrelated SCT. Probability of overall survival (OS) at 6 years with a median follow up of 64 (49–92) months reaches a plateau after 5 years at 0.33±0.05 and was not significantly better in CR1 than in CR2. However, there was a trend towards better OS at 6 years for unrelated 0.41±0.11 as compared to related 0.29±0.07 SCT (p=0.08) in CR1 only. This difference was significant for disease free survival (DFS) (0.48±0.09 unrelated vs 0.27±0.06 related; p=0.04), the major reason being a higher relapse rate in related as compared to unrelated SCT (0.62±0.08 vs 0.40±0.09). The overall non-relapse mortality at 6 years was 0.21±0.05. We conclude that OS and DFS reach a stable plateau from 5 years after SCT to more than 8 years after SCT. In CR1 patients, DFS is superior after unrelated as compared to related SCT. Accordingly, strategies designed to decrease relapse, especially after related SCT, have already been implemented in the ongoing protocols. The preferential use of unrelated rather than related donors may be beneficial and should be considered in future protocols. Disclosures: Off Label Use: Transplantation in elderly patients.

Long Term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Beta Thalassemia Major

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 4606-4606
Author(s):  
Fouzia NA ◽  
Sindhuvi E ◽  
Kavitha ML ◽  
Korula A ◽  
Abraham A ◽  
...  
Keyword(s):  
Chronic Gvhd ◽  
Thalassemia Major ◽  
Beta Thalassemia ◽  
Endocrine Disorders ◽  
Term Outcome ◽  
Term Survival ◽  
Hematopoietic Stem ◽  
Long Term Outcome

Abstract Introduction:Allogeneic hematopoietic stem cell transplantation (HSCT) cures beta thalassemia major (TM). Such individuals, ex-thalassemics, have good long term survival. However, there is limited data on long term outcome (LTO) of this therapy. This is particularly relevant as these patients often have organ dysfunction pre-transplant due to secondary hemosiderosis apart from the impact of post-transplant factors such as chronic GVHD, chimerism status and iron depletion therapy (IDT). In this report, we describe the LTO of patients with TM who underwent HSCT with busulfan (Bu) and cyclophosphamide (Cy) conditioning at our center from 2000 to 2011 and had a minimum of 2 year follow-up. Method: Data was extracted from prospectively maintained standardized case record forms for details of HSCT and long term follow-up with particular reference to GVHD, chimerism (evaluated at day +30, +60, +100 and thereafter as indicated), IDT (initiated at variable periods post-HSCT) and metabolic and endocrine disorders evaluated on physician discretion or as per clinical indications. Results:A total of 190 patients underwent matched related donor HSCT from 2001 to 2011 with Bu/Cy based conditioning. After excluding those who expired or had primary graft failure or did not have at least 2 years of follow-up, 124 patients were available for analysis of LTO. 44 patients (35.5%) class 3, 69 patients (55.6%) class 2 and 11 patients (8.9%) class 1. The median age was 7 years (range: 2-24) with 81 males (65.3%). The median follow-up was 7 years (range: 2 to 14). Chronic GVHD was present in 22 patients (17.7%]. Mixed chimerism (MC) occurred in 40 patients (32%) in the first year after HSCT: level I in 21 (52.5%), level II in 10 (25%), level III in 7 (17.5%), and level unknown in 2(5%). At last follow-up, 20/40 (50%) patients with MC went on to CC, 18 maintained stable MC (level I-5, level II-9 and level III-4) with hemoglobin of 11.35g/dl (range: 9-13.5), while 2 (5%) with level 3 MC remained transfusion dependent. Median serum ferritin (SF) at HSCT was 2367 ng/ml (range: 685-7660). IDT was initiated in 90 (72.6%) patients at a median of 15 months (range: 6-53) post-HSCT - 13 patients (14.4%) were treated with phlebotomy alone, while 39 (43.3%) received chelation and 38 (42.2%) the combination. Reduction in SF/month [absolute quantity (ng/ml/month) and percent] was as follows: 40.5 (range: 11.68 - 125.78); 1.67% (range: 0.5-4.58), 54.9 (range: 9.3- 278.7); 2.1% (range: 0.41- 13.8) and 36.6 (range: 3.51-590.7); 1.3% (range: 0.42-42.99), in the phlebotomy, chelation and combination groups, (p=0.077 & 0.017, respectively). SF level of <300 ng/ml was achieved in 33 patients (31%) at last follow-up. Anthropometry measurements (at last follow up) revealed short stature in 53 patients (42.7%; 38M/15F), underweight in 32 patients (25.8%; 20M/12F) and overweight in 14 (11.3%) patients (11M/3F). A total 48 patients (38.7%) had the following endocrine disorders: hypogonadism in 33 (73.3%), primary hypothyroidism in 9 (18.8%), hypopituitarism in 4 (8.3%), diabetes mellitus in 3 (6.2%), and hypoparathyroidism, dyslipidemia and hypertension in 1 patients each. 40 patients were vitamin D deficient (83.3%). Endocrine complications were more common in female patients (55.8% versus 29.6%; p=0.006). Two patients (1.3%) developed malignancies at 7 and 8 years, post-HSCT. Among different patient, donor and graft characteristics, there were no predictors of MC, nor did the ferritin levels or chelation therapy post-HSCT affect the incidence of endocrine complications in this cohort. Conclusion: Our data shows that even though the long term survival of ex-thalassemics is extremely good, at least 40% of them suffer from several co-morbidities related to iron overload and various metabolic and endocrine disorders which requires a coordinated plan for their management. The aim therefore should be to transplant these patients as early as possible before such complications occur and implement IDT intensively early after HSCT. Disclosures No relevant conflicts of interest to declare.

Radioscapholunate fusion with triquetrum and distal pole of scaphoid excision: long-term follow-up

2017 ◽  
Vol 43 (2) ◽  
pp. 168-173 ◽  
Author(s):  
Ngoc B. Ha ◽  
Joideep Phadnis ◽  
Simon B. M. MacLean ◽  
Gregory I. Bain
Keyword(s):  
Long Term Outcomes ◽  
Level Of Evidence ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Distal Pole ◽  
Long Term Follow Up ◽  
Radioscapholunate Fusion ◽  
The Mean

The purpose of this study was to assess the long-term outcomes of radioscapholunate fusion, with and without distal pole of scaphoid excision and excision of the triquetrum. These compromised three operative groups. Seventeen patients were identified with a minimum of 10 years follow-up, with a mean of 15 years (range 10–19). Fifteen of the 17 patients were satisfied with their outcome. Two were converted to total wrist fusion. The mean outcomes scores were; pain visual analogue scale score 2.1/10, Quick Disabilities of the Arm, Shoulder, and Hand 29 and Modified Mayo Wrist score 60. Patients with excision of the triquetrum had a mean radial–ulnar arc increase of 10° compared with the other two groups, but this was not statistically significant. The mean space for the scaphocapitate joint was 1.7 mm and lunocapitate joint was 1.3 mm at latest follow-up. Close adherence to the indications and surgical technique provided a sustainable good clinical outcome. Patients who obtained a good result at 2 years were likely to achieve a good long-term outcome. Level of evidence: IV

scholarly journals Review and Long-Term Outcomes of Cruciate Ligament Reconstruction versus Conservative Treatment in Siblings with Congenital Anterior Cruciate Ligament Aplasia

2017 ◽  
Vol 2017 ◽  
pp. 1-8 ◽  
Author(s):  
Diego Davanzo ◽  
Paolo Fornaciari ◽  
Geoffroy Barbier ◽  
Mauro Maniglio ◽  
Daniel Petek
Keyword(s):  
Anterior Cruciate Ligament ◽  
Conservative Treatment ◽  
Cruciate Ligament ◽  
Patient Counseling ◽  
Long Term Outcomes ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Anterior Cruciate

There is no consensus on the best treatment for anterior cruciate ligament hypoplasia or aplasia. To our knowledge, no comparative study between operative and conservative treatment of this condition has ever been performed. Conservative treatment is a viable alternative to surgery for ACL aplasia. Two siblings were examined at our outpatient clinic. The male patient underwent bilateral ACL reconstruction, while his sister was treated conservatively. Our results show a worse long-term outcome for the operative patient. At her last follow-up, the female patient treated conservatively showed subjective improvement in stability and gait. A review of the literature shows inconsistent outcomes after reconstruction in contrast to reports with cruciate ligament agenesis that did not undergo reconstruction with acceptable to good outcomes. Cruciate reconstruction should be reserved for cases of impaired articular instability, objectively manifest in the frequency of giving-way episodes. Treatment depends on the patient’s condition and expectations. Surgery should therefore only be suggested after proper patient counseling.

scholarly journals Long-Term Outcome of Reduced Intensity Conditioning Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Mycosis Fungoides and Sézary Syndrome: The Milan Experience

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 4655-4655
Author(s):  
Francesco Onida ◽  
Giorgia Saporiti ◽  
Silvia Alberti Violetti ◽  
Elena Tagliaferri ◽  
Federica Grifoni ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Median Time ◽  
Term Outcome ◽  
Hematopoietic Stem ◽  
Long Term Outcome ◽  
Reduced Intensity

Abstract Introduction: Although a few novel drugs have recently shown promising activity in mycosis fungoides (MF) and Sézary syndrome (SS), prognosis of patients with advanced stages or refractory disease remain poor, with median survival ranging from 1.4 to 3.4 years (Agar NS et al. JCO 2010). In selected patients, allogeneic hematopoietic stem cell transplantation (allo-HSCT) represents a potential curative strategy. By decreasing transplant-related mortality, reduced intensity (RIC) and nonmyeloablative conditioning (NMA) regimens lead to better outcomes in comparison to myeloablative ones. Here we report long-term outcome of our RIC allo-HSCT experimental program, initiated in 2001. Patients & Methods: As of July 2018, in our Center 40 patients underwent RIC allo-HSCT from HLA-identical sibling (n=16), unrelated donors (UD, n=22 - fully-matched in 10, 1 or 2-mismatch in 12) or haploidentical related donors (n=2). Median age was 52 years (range 19-66). All patients (24 M and 16 F) had stage IIB/IV refractory MF (n=27) or SS (n=13). Median number of previous treatment lines was 6 (range 2-12) while median time from diagnosis to transplantation was 46 months (range 11-264). The source of stem cells has been peripheral blood in 35 patients (87.5%), bone marrow in 4 (10%) and cord blood in 1 (2.5%). Conditioning regimens included FC/TBI200, pentostatin+TBI200, and fludarabine/melphalan in transplants from HLA-identical sibling donor or UD, whereas the TT/Flu/CTX/TBI200 regimen was used in the haploidentical setting. GvHD prophylaxis included CsA/MMF in all patients, with the addition of ATG in cases with UD and post-transplant CTX (50 mg/kg giorni +3 e +4) in haploidentical setting. Results: Full donor chimerism was obtained in 32 out of 37 evaluable patients, in a median time of 2 months (range 1-12). Acute GvHD occurred in 18 patients out of the 32 evaluable (56%), being of grade III-IV in 9 (28%). Chronic GvHD was observed in 10 patients (31%), being extensive in 4 (12%). Of note, the latter were all patients transplanted from HLA-identical sibling (i.e. without ATG). Following transplantation, a complete remission (CR) was achieved in 26 out of the 37 evaluable patients (70%), of whom 4 experienced relapse at +2 (2 pts), +25 and +35 months, respectively. At the last follow-up, 19 patients were alive and 17 (89%) maintained CR after a median follow-up of 80 months (range 4-210). Out of the 11 patients who did not achieve CR, 9 died from progressive disease (median follow-up of 12 months, range 3-31), 1 from a secondary malignancy, while 1 is still alive with disease 62 months after transplant. Transplant-related death occurred in 7 patients (17%), of whom 5 were in CR. In the whole population, the 5-year OS was 52% (95% CI 34-70) [Fig.1] and the 5-year DFS was 43% (95% CI 27-62). However, when MF and SS were analysed separately, 5-yrs DFS were 30% (95% CI 12-51) and 72% (95% CI 38-99), respectively (Fig.2). Apart from diagnosis, outcome appeared to be primarily associated with chemosensitivity and status of disease at transplantation. Conclusions: After a median follow-up longer than 6.5 years, we confirm the efficacy of RIC allo-HSCT as a powerful therapeutic strategy in inducing and maintaining remission in selected patients with chemosensitive advanced-stage CTCL, with results particularly encouraging in SS. Disclosures Cortelezzi: roche: Consultancy; abbvie: Consultancy; novartis: Consultancy; janssen: Consultancy.

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