scholarly journals Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study

Blood ◽  
2015 ◽  
Vol 125 (13) ◽  
pp. 2164-2172 ◽  
Author(s):  
Mieke Aldenhoven ◽  
Robert F. Wynn ◽  
Paul J. Orchard ◽  
Anne O’Meara ◽  
Paul Veys ◽  
...  
Keyword(s):  
Hematopoietic Cell Transplantation ◽  
Residual Disease ◽  
Hurler Syndrome ◽  
Term Outcome ◽  
Early Referral ◽  
Long Term Outcome ◽  
Key Points ◽  
Long Term Prognosis ◽  
International Multicenter Study

Key Points Patients with Hurler syndrome show significant residual disease burden despite HCT. Early referral for HCT, using noncarrier donors and regimens designed to achieve full-donor chimerism, offers the best long-term prognosis.

scholarly journals Diagnosis, Treatment and Long-term Outcome in Fetal Hydrocephalus(Pediatric Neurosurgery and Long-term Prognosis)

2011 ◽  
Vol 20 (5) ◽  
pp. 322-329
Author(s):  
Mami Yamasaki ◽  
Masahiro Nonaka ◽  
Yohei Bamba ◽  
Chika Teramoto ◽  
Chiaki Ban ◽  
...  
Keyword(s):  
Pediatric Neurosurgery ◽  
Special Issue ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Fetal Hydrocephalus ◽  
Long Term Prognosis

The Ethics of Infant Dialysis

1996 ◽  
Vol 16 (1_suppl) ◽  
pp. 505-509 ◽  
Author(s):  
Timothy E. Bunchman
Keyword(s):  
Standard Of Care ◽  
Health Care Team ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Number Of Factors ◽  
Long Term Prognosis ◽  
Infant Dialysis ◽  
Stage Renal Disease ◽  
End Stage

The proper treatment of an infant with end-stage renal disease depends upon a number of factors including parental willingness to take on the task, experience of the health-care team, local and regional resources, and society's willingness to accept this support as a standard of care. Whereas the abilityto keep infants aliveon peritoneal dialysis (PD) is obtainable, it is not without physical, financial, as well as emotional cost. In order for a family to agree to take on such a task, an understanding of the risks and long-term prognosis should be offered. This “informed consent” is difficult to obtain in such a highly charged situation when emotions often dictate choice independently of logic. Long-term outcome of infants on PD has improved over time, yet is still fraught with complications. Options of treatment or nontreatment are explored.

scholarly journals Very long-term follow-up of aplastic anemia treated with immunosuppressive therapy or allogeneic hematopoietic cell transplantation

2020 ◽  
Vol 99 (11) ◽  
pp. 2529-2538
Author(s):  
Beatrice Drexler ◽  
Felicitas Zurbriggen ◽  
Tamara Diesch ◽  
Romaine Viollier ◽  
Joerg P. Halter ◽  
...  
Keyword(s):  
Aplastic Anemia ◽  
Immunosuppressive Therapy ◽  
Hematopoietic Cell Transplantation ◽  
Clonal Evolution ◽  
University Hospital ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Long Term Survivors

Abstract Introduction Since the 1970s outcome of aplastic anemia (AA) patients has improved significantly due to the introduction of immunosuppressive therapy (IST) and allogeneic hematopoietic transplantation (HCT). However, patients may suffer from persistent disease, relapse, clonal evolution, graft-versus-host disease and other late effects. Here, we analyse very long-term outcome of all AA patients at our institution comparing not only survival, but also response status and complications. Methods Patient charts of all 302 AA patients treated between 1973 and 2017 at the University Hospital Basel, Switzerland, were retrospectively analysed. Results First line treatment was IST in 226 (75%) and HCT in 76 (25%) patients. Overall survival at 30 years was similar in patients treated initially by HCT and IST (44% (±14%), and 40% (± 9%) respectively, with better results in more recent years. Partial and no response occurred more frequently after IST, relapse incidence after IST was 24 %, whereas non-engraftment and graft failure was documented in 15 patients (19 %) after HCT. Clonal evolution to myelodysplastic syndrome / acute myeloid leukemia was 16 % at 25 years in IST patients, 1.3 % in HCT patients, iron overload (18 versus 4 %, p = 0.002) and cardiovascular events (11 versus 1 %, p=0.011) occured significantly more often in IST than HCT treated patients. The majority of long-term survivors, 96% of those alive at 25 years, were in complete remission at last follow up, irrespective of the initial treatment modality. Conclusion Very long term survivors after AA are those with stable hematopoietic recovery.

97. Long-term outcome of hematopoietic cell transplantation for Wolman disease

2008 ◽  
Vol 93 (2) ◽  
pp. 39
Author(s):  
Jakub Tolar ◽  
Anna Petryk ◽  
Khalid Khan ◽  
Kendra Bjoraker ◽  
Jose Jessurun ◽  
...  
Keyword(s):  
Cell Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Wolman Disease

Time-Sequenced G-CSF (Lenograstim) for the Treatment of Adult Acute Lymphoblastic Leukemia - Seven Year Follow-Up of the Polish Adult Leukemia Group 4–96 Study.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 2733-2733
Author(s):  
Sebastian Giebel ◽  
Jerzy Holowiecki ◽  
Malgorzata Krawczyk-Kulis ◽  
Slawomira Kyrcz-Krzemien ◽  
Andrzej Hellmann ◽  
...  
Keyword(s):  
Acute Lymphoblastic Leukemia ◽  
Hematopoietic Cell Transplantation ◽  
Lymphoblastic Leukemia ◽  
High Dose ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Chemotherapy Protocol ◽  
Adult Leukemia ◽  
Leukemia Group

Abstract The goal of the study was to evaluate long-term outcome of patients treated within 4–96 trial by the Polish Adult Leukemia Group (PALG). Sixty four patients with newly diagnosed acute lymphoblastic leukemia (ALL) (median age 26 years, range 16–58) were randomly assigned to receive chemotherapy alone (n=31) or chemotherapy and glucosylated G-CSF (lenograstim) (n=33). Both groups were well-balanced in terms of age, initial WBC, immunophenotype and bcr/abl positivity. Induction therapy consisted of epirubicin+vincristin (Epi/Vcr) on days 1, 8, 15, 22, prednisone on days 1–28, L-asparaginase 8 doses starting from day 13; consolidation treatment included twice methotrexate+etoposide (Mtx/Vep), twice high dose cytarabine and cyclophosphamide (HDAraC/Ctx), CNS irradiation and intrathecal Mtx. In T-derived ALL, additional HDAraC/Ctx was administered instead of the first Mtx/HDAraC. During induction patients received G-CSF 150 μg/m2 sc. on days 2–6, 9–13, 16–20, 23-until the neutrophil recovery >1.0x109/L, starting 36 hours after Epi/Vcr, finishing 48 hours before the next dose; in consolidation - following each HDAraC/Ctx course on days 5–16. High risk patients having a donor were performed allogeneic hematopoietic cell transplantation in first complete remission, whereas those without a donor were given autologous transplant. At seven years the overall survival rate equalled 42% for G-CSF group and 24% for the controls (p=0.11). There was also a trend to higher probability of leukemia-free survival in advantage of patients receiving the cytokine (38% vs. 20%, p=0.17). The above differences might have resulted from: 1) Higher CR rate in the G-CSF group compared to the controls (94% vs. 87%), 2) Better adherence to the chemotherapy protocol (faster completion of induction-consolidation programme by 19 days, p=0.005, less dose reductions or delays), which in turn might have influenced the risk of relapse. We conclude that time sequenced G-CSF administration may improve long-term outcome of adult ALL patients although the study including larger population is required to confirm this hypothesis.

Long-Term Outcome of High-Dose Sequential Chemotherapy with Autografting (i-HDS) in Follicular Lymphoma at Diagnosis: An Update of the Prospective Multicenter Consecutive Trial of the "Gruppo Italiano Trapianto Di Midollo Osseo" (Gitmo).

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 903-903
Author(s):  
Marco Ladetto ◽  
Sonia Vallet ◽  
Paolo Corradini ◽  
Fabio Benedetti ◽  
Umberto Vitolo ◽  
...  
Keyword(s):  
High Risk ◽  
Residual Disease ◽  
Elevated Serum ◽  
Stage Iv ◽  
Experimental Treatment ◽  
High Dose ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Treatment Implementation

Abstract Introduction. i-HDS is a promising though experimental treatment for FL patients at diagnosis. We have published that i-HDS is feasible also in the context of a multicenter trial with limited toxicity and promising results (Ladetto et al, Blood, 2002). This analysis is an update at 42 months from the previous closing date. Particular attention has been devoted to late toxicities and outcome according to molecular remission (MR). Patients and methods. 92 untreated patients aged 18–60 years with stage III-IV FL requiring treatment were enrolled by 20 Italian centers between 1997 and 1999 and evaluated on an intention-to-treat basis. Inclusion criteria have been previosly described. Clinical features at diagnosis were: Ann Arbor stage IV: 84%; BM involvement: 80%; extranodal and extra-BM disease: 55%; bulky mass: 51%; elevated serum LDH: 37%; "B" symptoms: 30%; leukemic disease: 12%; aaIPI ≥ 2: 37%. The i-HDS schedule included 2APO, 2DHAP, Etoposide 2g/sqm, Methotrexate 8g/sqm and Cyclophosphamide 7g/sqm with PBPC collection. The myeloablative regimen was Mitoxantrone 60mg/sqm + Melphalan 180mg/sqm followed by 5–8x106 CD34+ cells/kg. Minimal residual disease analysis with the bcl-2/IgH rearrangement was available in 47% of patients. Results: 87% of patients completed the planned treatment. CR rate was 88%. Currently the median follow-up is 62 months. Late toxic effects included five myelodysplastic syndromes and /or secondary leukemias (MDS/2AL) all occurring within two years from the end of treatment. In 4 of 5 cases MDS/2AML occurred in subjects already re-treated for disease relapse. Two fatal solid tumors (one gastric cancer and one lung cancer) were also recorded. Long-term hematopoietic fuction was satisfactory in all patients not developing MDS/2AML. Heart failure occurred in three patients, always manageable with oral medical treatment. The projected overall survival and progression-free survival (PFS) at 7,5 years are 74% and 56%. Notably, following i-HDS, patients with aaIPI≥2 had an outcome comparable to those with aaIPI ≤ 1 (p=NS). Attainement of MR in the first year following i-HDS was highly predictive even for the long term outcome. Among patients achieving MR the relapse rate (RR) was 13% while in those failing to achieve MR was 81% (p<0001). Conclusions. This long-term analysis indicates the following: a) i-HDS allows long-term PFS in 56% of advanced FL patients regardless of the aaIPI. This result compares favorably with those achieved with Rituximab-free non-intensified therapy at least for high-risk patients; b) MR achievement is a powerful indicator for prolonged PFS; c) although severe side effects occurred in this series, relapse remains the major cause of treatment failure. Thus, treatment implementation is required. Rituximab inclusion is probably one of the most feasible approaches, suitable for multicenter trials. The new GITMO trial, comparing CHOP vs i-HDS (both supplemented with Rituximab), currently ongoing for high-risk (aaIPI≥2) FL patients, will help clarifying wheter dose-intensification has still a role in the monoclonal antibody era.

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.

Sign in / Sign up

Export Citation Format

Share Document