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  • 1
    Online Resource
    Online Resource
    HLA-Matched Related Donor Hematopoietic Cell Transplantation in 43 Patients with Fanconi Anemia Conditioned with 60 mg/kg of Cyclophosphamide
    Elsevier BV ; 2007
    In:  Biology of Blood and Marrow Transplantation Vol. 13, No. 12 ( 2007-12), p. 1455-1460
    Details
    In: Biology of Blood and Marrow Transplantation, Elsevier BV, Vol. 13, No. 12 ( 2007-12), p. 1455-1460
    Type of Medium: Online Resource
    ISSN: 1083-8791
    URL: Article
    DOI: 10.1016/j.bbmt.2007.08.004
    Language: English
    Publisher: Elsevier BV
    Publication Date: 2007
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  • 2
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    Optimizing Results of Unrelated Donor Hematopoietic Stem Cell Transplantion in Developing Countries: Young Age and Early Disease as Positive Factors for Survival.
    American Society of Hematology ; 2006
    In:  Blood Vol. 108, No. 11 ( 2006-11-16), p. 5404-5404
    Details
    In: Blood, American Society of Hematology, Vol. 108, No. 11 ( 2006-11-16), p. 5404-5404
    Abstract: Introduction: Unrelated donor hematopoietic stem cell transplantation (HSCT) has been carried out in Brazil since 1995, with an increasing number of procedures among the transplant centers. Evaluating this experience is important to improve the outcomes of this therapeutic modality. The aim of this study is to assess risk factors for survival in 125 patients who underwent unrelated donor HSCT for malignant diseases. Material and methods: 125 patients with malignant diseases who received unrelated donor HSCT between july/95 and june/2005 in a single institution in Brazil were analyzed. Kaplan-Meier curves were used to estimate overall survival, log-rank test for comparison of continuous variables and Fisher exact test for categorical variables. Cox proportional hazard model was used for analyzing risk factors for survival. Analyzed risk factors: sex, age, acute graft-versus-host-disease (a-GVHD) grades II-IV, extensive chronic (c) GVHD, diagnosis, stage of disease, source of stem cells, number of infused cells/Kg and HLA matching. Median age was 17 years (range 1–55), male (n=78) and female (n=44). Diseases: CML (n=46), AML/MDS (n=40), ALL (n=34), other (n=5). Source of stem cells: bone marrow (n=95), umbilical cord blood (n=30). ATG or ALG containing regimens were used in 25 patients. Advanced disease was detected in 66 patients (53%) and was defined as CML in advanced phases, AML and ALL ≥ second clinical remission (CR2), relapsed or refractory. Results: Acute GVHD II-IV was observed in 51 (41%) patients and extensive c-GVHD in 32 (26%). Main death causes: a-GVHD (9%), c-GVHD (13%), infections (41%) and relapse (27%). Estimated overall survival (OS) in 10 years was 40%, with a median survival of 189 days. Sex, diagnosis, stem cell source, type of conditioning, number of infused cells/Kg and HLA matching did not influenced survival. OS in 10 years was higher in patients with early stage disease compared to advanced diseases (55% × 20%; p=.0005) and in patients under 18 years of age compared to those ≥ 18 years-old (60% × 20%; p=.0012). The presence of extensive c-GVHD had a positive influence in OS (OR 0,1758 – 0,9092). Conclusions: The OS in 10 years was 40% for this group of patients. Patients in early stage disease and under 18 years of age have a higher OS in 10 years (55% and 60% respectively). Extensive c-GVHD had a positive impact in survival, probably due to graft versus leukemia effect.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V108.11.5404.5404
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2006
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  • 3
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    Online Resource
    Long Term Results of Allogeneic Stem Cell Transplant for CML in Pediatric Patients: A Study of 50 Cases Transplanted over 20 Years in a Single Institution.
    American Society of Hematology ; 2006
    In:  Blood Vol. 108, No. 11 ( 2006-11-16), p. 5361-5361
    Details
    In: Blood, American Society of Hematology, Vol. 108, No. 11 ( 2006-11-16), p. 5361-5361
    Abstract: Introduction: Chronic myeloid leukemia (CML) accounts for 2–3% of the leukemias in childhood. The only potential curative treatment is allogeneic hematopoietic stem cell transplantation (HSCT), although promising results achieved with imatinib mesylate in adults substantiate its use as a therapeutic alternative for children. The aim of this study is to analyze the outcomes of HSCT in pediatric patients regarding overall survival (OS) and main causes of death. Materials and methods: Retrospective analysis of children aged 1–17 years, diagnosed with CML who underwent HSCT in a single institution in Brazil between jan/1984 and aug/2005. Survival was estimated by Kaplan-Meier curves. Log Rank test was used for comparison of continuous variables. Results: Fifty patients were assessed, 31 male and 19 female. Median age of 13,5 years (1–17). Forty one patients (82%) were in first chronic phase (CP1) and 9 in advanced phases. The interval between diagnosis and HSCT had a median time of 17,5 months (5–84). The source of stem cells was bone marrow in 44 patients (88%), umbilical cord blood in 5 (10%) and peripheral blood stem cell in 1 (2%). Thirty nine patients (78%) underwent related HSCT and 11 (22%) unrelated donor HSCT. Conditioning regimens: busulfan and cyclophosphamide in 35 patients (70%) and TBI containing regimens in 15 (30%). Complete engraftment occurred in 82% of the transplants. Acute (a) graft-versus-host-disease (GVHD) grades II–IV occurred in 44% of the patients, with 20% grade IV. Extensive chronic (c) GVHD occurred in 15/40 patients (38%). Fifteen patients (32%) relapsed after HSCT. Mortality in the study population was 48% and the main causes of death were: relapse in 6 patients (25%), a-GVHD in 6 (25%) and c-GVHD in 4 (17%). Estimated OS in 20 years was 50%, with a median survival of 1926 days. When analyzed separately, patients in CP1 who received related HSCT and immuneprophilaxis with three drugs (steroids, cyclosporine and methotrexate) had an estimated OS in 20 years of 70%. Conclusions: Long term follow up of these children with CML who underwent allogeneic HSCT demonstrate an OS of 50%, reaching 70% in low risk patients. Main causes of death were relapse, acute and chronic GVHD.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V108.11.5361.5361
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2006
    detail.hit.zdb_id: 1468538-3
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  • 4
    Online Resource
    Online Resource
    Five Years Update Results of Basiliximab (BAMAB) Therapy for Acute Refractory Graft Versus Host Disease (AGVHD).
    American Society of Hematology ; 2004
    In:  Blood Vol. 104, No. 11 ( 2004-11-16), p. 5083-5083
    Details
    In: Blood, American Society of Hematology, Vol. 104, No. 11 ( 2004-11-16), p. 5083-5083
    Abstract: The IL-2 activation of RAS pathway promotes lymphocyte proliferation, differentiation and development of A-GVHD. This mechanism can be inhibited by basiliximab, a chimeric murin-human monoclonal antibody which binds to the alpha chain of interleukin-2 receptors on activated T-cells. Basiliximab is already largely used to prevent acute rejection after renal transplantation, but the experience with this drug on BMT setting is still limited. We treated 34 patients (pts) with severe refractory A-GVHD with Basiliximab from december 1998 to october, 2003. Median age was 13 years (range: 2 to 38). Most of the patiens had received an unrelated BMT (73%). Cell source was bone marrow in 88% and cord blood in 12% of the patients. Diagnosis included acute leukemia (9 pts), MDS (4 pts), CML(9 pts), Fanconi anemia (9 pts), and SAA (3 pts). Because of a half life of about 7 days, BAMAB schedule was 40mg weekly for 2–3 doses (adults) and 20 weekly for 2–3 doses (children). All pts had a-GVHD grade III-IV, manifesting from day 6–248 after transplant (M: 24 d), while on cyclosporin, and refractory to association with steroids (2–5mg/kg/d). Skin was involved in 32, GI in 25 and liver in 23 patients. Median follow-up was 196 (range: 35–1847) days. Overall response rate was 82% (CR: 11 pts, PR: 17 pts). Two pts (8 %) were not evaluated due to early death. Response varied according to the site involved, being complete in 84% of skin, 48% of GI and 26% of liver GVHD. Duration of response varied from 5–1103 d (M: 38 d). There were no infusion-related reactions. Eleven pts are alive (32%), 7 pts with complete response, 3 with partial response and 1 pt without response. Acute GVHD reactivation was seen on 41% of the patients, but half of them could be rescued with other therapies (MMF, PUVA, steroids). Infection was the main cause of death, being responsible for 83% of all deaths (disseminated CMV-4 pts, fungal infection-6 pts and sepsis-8 pts). Other causes of death included bleeding (2pts) and relapse (2 pts). In conclusion, basiliximab was able to induce response on patients with refractory A-GVHD. Prospective studies with this drug are necessary to address the optimal dose, schedule and incidence of infection.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V104.11.5083.5083
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2004
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  • 5
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    Bone marrow transplantation in patients with storage diseases: a developing country experience
    FapUNIFESP (SciELO) ; 2006
    In:  Arquivos de Neuro-Psiquiatria Vol. 64, No. 1 ( 2006-03), p. 1-4
    Details
    In: Arquivos de Neuro-Psiquiatria, FapUNIFESP (SciELO), Vol. 64, No. 1 ( 2006-03), p. 1-4
    Abstract: O transplante de medula óssea é uma opção terapêutica para os pacientes com doenças de acúmulo. Entre 1979 e 2002, oito pacientes, quatro femininos e quatro masculinos (entre um e 13 anos de idade) foram submetidos a este procedimento em nosso centro. Seis pacientes apresentavam mucopolissacaridose (MPS I em 3; MPS III em um e MPS VI em 2), um paciente apresentava adrenoleucodistrofia e um apresentava doença de Gaucher. Cinco pacientes receberam o transplante de doador aparentado e três de doador não aparentado. Três pacientes desenvolveram doença do enxerto versus hospedeiro (dois com MPS I e um com MPS VI) e faleceram entre 37 e 151 dias após o transplante. Cinco pacientes sobreviveram entre 4 e 16 anos após o transplante. Três tiveram melhora clínica (um MPS I, um MPS VI e o paciente com doença de Gaucher), um paciente não apresentou progressão da doença (adrenoleucodistrofia) e um paciente não teve alteração da história natural da doença (MPS III).
    Type of Medium: Online Resource
    ISSN: 0004-282X
    URL: Article
    DOI: 10.1590/S0004-282X2006000100001
    Language: Unknown
    Publisher: FapUNIFESP (SciELO)
    Publication Date: 2006
    detail.hit.zdb_id: 2053072-9
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  • 6
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    Online Resource
    Long-term Survival, Organ Function, and Malignancy after Hematopoietic Stem Cell Transplantation for Fanconi Anemia
    Elsevier BV ; 2016
    In:  Biology of Blood and Marrow Transplantation Vol. 22, No. 7 ( 2016-07), p. 1257-1263
    Details
    In: Biology of Blood and Marrow Transplantation, Elsevier BV, Vol. 22, No. 7 ( 2016-07), p. 1257-1263
    Type of Medium: Online Resource
    ISSN: 1083-8791
    URL: Article
    DOI: 10.1016/j.bbmt.2016.03.007
    Language: English
    Publisher: Elsevier BV
    Publication Date: 2016
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  • 7
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    Gleevec as Therapy for CML Relapsed after Allogeneic Stem Cell Transplantation: High Rate of BCR-ABL PCR Negativity and Donor Graft Reconstitution.
    American Society of Hematology ; 2006
    In:  Blood Vol. 108, No. 11 ( 2006-11-16), p. 3671-3671
    Details
    In: Blood, American Society of Hematology, Vol. 108, No. 11 ( 2006-11-16), p. 3671-3671
    Abstract: INTRODUCTION: CML relapsed after HSCT has been treated with donor lymphocyte infusions (DLI), with responses achieving 60–70%, and even better results in earlier relapses. However, complications as chronic GVHD and myelossupression can be a problem, especially on those who receive larger DLI doses. The present study has the objective of evaluate quality of response and chimerism in patients who received Gleevec as therapy for relapsed CML after HSCT. PATIENTS AND METHODS: We report 32 CML patients who received Gleevec as therapy for relapse after HSCT. Age: 13 – 56 (Median: 38 y); sex M/F: 17/15; Allogeneic HSCT: 31 pts, syngeneic: 1 pt. Time from HSCT to relapse: 0–122 m (M 16m). Relapse was hematologic in 29 and cytogenetic in 3 patients. Phase at hematologic relapse: CP-14 pts, AP-11 pts and BC-4 pts; clonal evolution: 7 patients. Previous VNTR was available in 16 pts: 10–35% donor in 11, 0% donor in 3 and 95% donor in 2 pts. Previous DLI: 14 pts. Dose: 600mg QD in 15 pts, 400 mg QD - 17 pts. RESULTS: Therapy duration: 7–1795 d (M 365 d). 27 (84%) pts reached complete hematologic response (CHR). Time to CHR: 7–60 d (M 28 d). Cytogenetic response was available for 25 pts, being complete in 16 (48%), partial in 2 and absent in 7 pts. Competitive Q-PCR was available in 21 patients, of whom 10 had major molecular responses (3-log reduction at the BCR-ABL/ABL ratio). In 6 (18%) patients, BCR-ABL was negative by nested PCR. Four (13%) pts relapsed after a initial response (1 BC, 3 AP). Nine from 10 pts with sequential VNTR, improved chimerism to more than 95% donor cells after Gleevec therapy. One patient had autologous recovery of Ph negative cells. Hematologic toxicity grade II–IV was observed in 21 pts (63%). Eleven pts developed grade IV neutropenia with a duration of 0–8m (M 2m). Five pts developed non-hematologic toxicity grade III–IV. Dose was held in 4 pts, increased in 6 and reduced in 11 pts. Only two pts developed skin and liver chronic GVHD, and two patients who had c-GVHD before therapy with imatinib did not flare. Estimated 5y OS is 67%. CONCLUSIONS:Gleevec can be used safely as therapy for BMT relapse, with durable cytogenetic and molecular responses in chronic phase;Complete chimerism is often reestablished by Gleevec therapy after allogeneic BMT;Increased incidence of GVHD was not observed in this group of patients.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V108.11.3671.3671
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2006
    detail.hit.zdb_id: 1468538-3
    detail.hit.zdb_id: 80069-7
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  • 8
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    Paroxysmal Nocturnal Hemoglobinuria (PNH): Long Term Follow up of 48 Patients in a Single Institution.
    American Society of Hematology ; 2006
    In:  Blood Vol. 108, No. 11 ( 2006-11-16), p. 3762-3762
    Details
    In: Blood, American Society of Hematology, Vol. 108, No. 11 ( 2006-11-16), p. 3762-3762
    Abstract: Background: PNH is a clonal acquired disease characterized by poor expression of CD55 and CD59, adding higher lytic activity to the complement pathway, ending up in hemolysis, pancytopenia and thrombosis. The clinical variability and the lack of effective therapeutics make the treatment of PNH a real challenge. Material and methods: A retrospective analysis of 48 patients diagnosed with PNH between feb/1997 and march/2006 admitted to our center was performed. Before 1999, diagnosis was made by HAM and SACAROSIS tests. From this point immunophenotyping analysis was used to establish diagnosis. Bone marrow aspirate and biopsy, cytogenetic and biochemistry analysis were performed for exclusion of comorbities. Patients were divided in two groups: 1) de novo PNH associated with marrow failure (MF) and 2) PNH as an evolution of severe aplastic anemia (SAA) or mielodysplastic syndrome (MDS). Response was defined as reduction at hemolytic episodes as well as improvement of CBC and transfusion requirements. Results: Median age was 24 years (range 9–75); male patients constituted 58% of the sample (male/female: 1,4:1). De novo PNH with associated marrow failure occurred in 22 patients (group 1) and evolutive PNH in 15, being 13 from previous SAA and 2 from previous hypoplastic MDS (group 2). Thirty seven patients were followed at our clinic during this time: 25 (67,5%) received immunesupression with cyclosporine and steroids. Overall response (partial or complete) after one year of therapy was 84% (31/37). The remaining 11 patients (32,5%) received steroids alone, androgens or only observation, four of them remained with stable disease. HLA match hematopoietic stem cell transplantation (HSCT) was performed in 18/48 (38%), being 11 from group 1 and 7 from group 2. Main indications were: pancytopenia (77%), thrombosis (17%) and AML (6%). Only nine patients died (19%): five after HSCT and 4 of those followed at our clinic (massive thrombosis and hemolysis). Overall survival after HSCT was 72% with median follow-up of 890 days. Overall survival among outpatient clinic patients was 86% with a median follow-up of 2340 days. Most frequent outpatient events were: hemolysis (48%), pancytopenia (13%), thrombosis (13 %), renal failure (9%) and infection (8%).Fourteen patients (37%) remained without symptoms. Conclusions:Immunosuppressive therapy with cyclosporine and corticosteroids is an excellent strategy in patients with PNH and associated marrow failure, leading to durable clinical responses.The present data supports previous published indications for HSCT: ASS and thrombosis.Overall survival of PNH patients who received HSCT achieved 72%.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    URL: Article
    DOI: 10.1182/blood.V108.11.3762.3762
    RVK:
    XA 33000
    RVK:
    XA 10000
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2006
    detail.hit.zdb_id: 1468538-3
    detail.hit.zdb_id: 80069-7
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