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203. Activity of Broad-Spectrum T Cells as Treatment for AdV, EBV, CMV, BKV, and HHV6 Infections after HSCT

Author

Papadopoulou, Anastasia, primary, Gerdemann, Ulrike, additional, Katari, Usha L., additional, Tzannou, Ifigenia, additional, Liu, Hao, additional, Martinez, Caridad, additional, Leung, Kathryn, additional, Carrum, George, additional, Gee, Adrian P., additional, Vera, Juan F., additional, Krance, Robert A., additional, Brenner, Malcolm K., additional, Rooney, Cliona M., additional, Heslop, Helen E., additional, and Leen, Ann M., additional

Published
2014
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204. Long-term outcome after haploidentical stem cell transplant and infusion of T cells expressing the inducible caspase 9 safety transgene

Author

Zhou, Xiaoou, primary, Di Stasi, Antonio, additional, Tey, Siok-Keen, additional, Krance, Robert A., additional, Martinez, Caridad, additional, Leung, Kathryn S., additional, Durett, April G., additional, Wu, Meng-Fen, additional, Liu, Hao, additional, Leen, Ann M., additional, Savoldo, Barbara, additional, Lin, Yu-Feng, additional, Grilley, Bambi J., additional, Gee, Adrian P., additional, Spencer, David M., additional, Rooney, Cliona M., additional, Heslop, Helen E., additional, Brenner, Malcolm K., additional, and Dotti, Gianpietro, additional

Published
2014
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205. Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: The Primary Immune Deficiency Treatment Consortium experience

Author

Shearer, William T., primary, Dunn, Elizabeth, additional, Notarangelo, Luigi D., additional, Dvorak, Christopher C., additional, Puck, Jennifer M., additional, Logan, Brent R., additional, Griffith, Linda M., additional, Kohn, Donald B., additional, O'Reilly, Richard J., additional, Fleisher, Thomas A., additional, Pai, Sung-Yun, additional, Martinez, Caridad A., additional, Buckley, Rebecca H., additional, and Cowan, Morton J., additional

Published
2014
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206. The Hematopoietic Cell Transplant Comorbidity Index predicts survival after allogeneic transplant for nonmalignant diseases

Author

Thakar, Monica S., Broglie, Larisa, Logan, Brent, Artz, Andrew, Bunin, Nancy, Burroughs, Lauri M., Fretham, Caitrin, Jacobsohn, David A., Loren, Alison W., Kurtzberg, Joanne, Martinez, Caridad A., Mineishi, Shin, Nelson, Adam S., Woolfrey, Ann, Pasquini, Marcelo C., and Sorror, Mohamed L.

Abstract

Despite improvements, mortality after allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases remains a significant problem. We evaluated whether pre-HCT conditions defined by the HCT Comorbidity Index (HCT-CI) predict probability of posttransplant survival. Using the Center for International Blood and Marrow Transplant Research database, we identified 4083 patients with nonmalignant diseases transplanted between 2007 and 2014. Primary outcome was overall survival (OS) using the Kaplan-Meier method. Hazard ratios (HRs) were estimated by multivariable Cox regression models. Increasing HCT-CI scores translated to decreased 2-year OS of 82.7%, 80.3%, 74%, and 55.8% for patients with HCT-CI scores of 0, 1 to 2, 3 to 4, and ≥5, respectively, regardless of conditioning intensity. HCT-CI scores of 1 to 2 did not differ relative to scores of 0 (HR, 1.12 [95% CI, 0.93-1.34]), but HCT-CI of 3 to 4 and ≥5 posed significantly greater risks of mortality (HR, 1.33 [95% CI, 1.09-1.63]; and HR, 2.31 [95% CI, 1.79-2.96], respectively). The effect of HCT-CI differed by disease indication. Patients with acquired aplastic anemia, primary immune deficiencies, and congenital bone marrow failure syndromes with scores ≥3 had increased risk of death after HCT. However, higher HCT-CI scores among hemoglobinopathy patients did not increase mortality risk. In conclusion, this is the largest study to date reporting on patients with nonmalignant diseases demonstrating HCT-CI scores ≥3 that had inferior survival after HCT, except for patients with hemoglobinopathies. Our findings suggest that using the HCT-CI score, in addition to disease-specific factors, could be useful when developing treatment plans for nonmalignant diseases.

Published
2019
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207. SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery

Author

Haddad, Elie, Logan, Brent R., Griffith, Linda M., Buckley, Rebecca H., Parrott, Roberta E., Prockop, Susan E., Small, Trudy N., Chaisson, Jessica, Dvorak, Christopher C., Murnane, Megan, Kapoor, Neena, Abdel-Azim, Hisham, Hanson, Imelda C., Martinez, Caridad, Bleesing, Jack J. H., Chandra, Sharat, Smith, Angela R., Cavanaugh, Matthew E., Jyonouchi, Soma, Sullivan, Kathleen E., Burroughs, Lauri, Skoda-Smith, Suzanne, Haight, Ann E., Tumlin, Audrey G., Quigg, Troy C., Taylor, Candace, Dávila Saldaña, Blachy J., Keller, Michael D., Seroogy, Christine M., Desantes, Kenneth B., Petrovic, Aleksandra, Leiding, Jennifer W., Shyr, David C., Decaluwe, Hélène, Teira, Pierre, Gillio, Alfred P., Knutsen, Alan P., Moore, Theodore B., Kletzel, Morris, Craddock, John A., Aquino, Victor, Davis, Jeffrey H., Yu, Lolie C., Cuvelier, Geoffrey D. E., Bednarski, Jeffrey J., Goldman, Frederick D., Kang, Elizabeth M., Shereck, Evan, Porteus, Matthew H., Connelly, James A., Fleisher, Thomas A., Malech, Harry L., Shearer, William T., Szabolcs, Paul, Thakar, Monica S., Vander Lugt, Mark T., Heimall, Jennifer, Yin, Ziyan, Pulsipher, Michael A., Pai, Sung-Yun, Kohn, Donald B., Puck, Jennifer M., Cowan, Morton J., O'Reilly, Richard J., and Notarangelo, Luigi D.

Abstract

The Primary Immune Deficiency Treatment Consortium (PIDTC) performed a retrospective analysis of 662 patients with severe combined immunodeficiency (SCID) who received a hematopoietic cell transplantation (HCT) as first-line treatment between 1982 and 2012 in 33 North American institutions. Overall survival was higher after HCT from matched-sibling donors (MSDs). Among recipients of non-MSD HCT, multivariate analysis showed that the SCID genotype strongly influenced survival and immune reconstitution. Overall survival was similar for patients with RAG, IL2RG, or JAK3 defects and was significantly better compared with patients with ADA or DCLRE1C mutations. Patients with RAG or DCLRE1C mutations had poorer immune reconstitution than other genotypes. Although survival did not correlate with the type of conditioning regimen, recipients of reduced-intensity or myeloablative conditioning had a lower incidence of treatment failure and better T- and B-cell reconstitution, but a higher risk for graft-versus-host disease, compared with those receiving no conditioning or immunosuppression only. Infection-free status and younger age at HCT were associated with improved survival. Typical SCID, leaky SCID, and Omenn syndrome had similar outcomes. Landmark analysis identified CD4+ and CD4+CD45RA+ cell counts at 6 and 12 months post-HCT as biomarkers predictive of overall survival and long-term T-cell reconstitution. Our data emphasize the need for patient-tailored treatment strategies depending upon the underlying SCID genotype. The prognostic significance of CD4+ cell counts as early as 6 months after HCT emphasizes the importance of close follow-up of immune reconstitution to identify patients who may need additional intervention to prevent poor long-term outcome.

Published
2018
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208. Genetic and mechanistic diversity in pediatric hemophagocytic lymphohistiocytosis

Author

Chinn, Ivan K., Eckstein, Olive S., Peckham-Gregory, Erin C., Goldberg, Baruch R., Forbes, Lisa R., Nicholas, Sarah K., Mace, Emily M., Vogel, Tiphanie P., Abhyankar, Harshal A., Diaz, Maria I., Heslop, Helen E., Krance, Robert A., Martinez, Caridad A., Nguyen, Trung C., Bashir, Dalia A., Goldman, Jordana R., Stray-Pedersen, Asbjørg, Pedroza, Luis A., Poli, M. Cecilia, Aldave-Becerra, Juan C., McGhee, Sean A., Al-Herz, Waleed, Chamdin, Aghiad, Coban-Akdemir, Zeynep H., Jhangiani, Shalini N., Muzny, Donna M., Cao, Tram N., Hong, Diana N., Gibbs, Richard A., Lupski, James R., Orange, Jordan S., McClain, Kenneth L., and Allen, Carl E.

Abstract

The HLH-2004 criteria are used to diagnose hemophagocytic lymphohistiocytosis (HLH), yet concern exists for their misapplication, resulting in suboptimal treatment of some patients. We sought to define the genomic spectrum and associated outcomes of a diverse cohort of children who met the HLH-2004 criteria. Genetic testing was performed clinically or through research-based whole-exome sequencing. Clinical metrics were analyzed with respect to genomic results. Of 122 subjects enrolled over the course of 17 years, 101 subjects received genetic testing. Biallelic familial HLH (fHLH) gene defects were identified in only 19 (19%) and correlated with presentation at younger than 1 year of age (P< .0001). Digenic fHLH variants were observed but lacked statistical support for disease association. In 28 (58%) of 48 subjects, research whole-exome sequencing analyses successfully identified likely molecular explanations, including underlying primary immunodeficiency diseases, dysregulated immune activation and proliferation disorders, and potentially novel genetic conditions. Two-thirds of patients identified by the HLH-2004 criteria had underlying etiologies for HLH, including genetic defects, autoimmunity, and malignancy. Overall survival was 45%, and increased mortality correlated with HLH triggered by infection or malignancy (P< .05). Differences in survival did not correlate with genetic profile or extent of therapy. HLH should be conceptualized as a phenotype of critical illness characterized by toxic activation of immune cells from different underlying mechanisms. In most patients with HLH, targeted sequencing of fHLH genes remains insufficient for identifying pathogenic mechanisms. Whole-exome sequencing, however, may identify specific therapeutic opportunities and affect hematopoietic stem cell transplantation options for these patients.

Published
2018
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212. Outcome after Allogeneic Stem Cell Transplant for Patients with Hematological Malignancies and Associated Chromosome 7 Deletions

Author

Naik, Swati, primary, Okur, Fatma, additional, Omer, Bilal, additional, Rao, Pulivarthi, additional, Martinez, Caridad, additional, Leung, Kathryn, additional, Wu, Jesse, additional, Ahmed, Nabil, additional, Allen, Carl, additional, Gottschalk, Stephen, additional, Heslop, Helen E., additional, Brenner, Malcolm K., additional, and Krance, Robert A., additional

Published
2014
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213. Safety and Clinical Efficacy of Rapidly-Generated Virus-Specific T Cells with Activity Against Adv, EBV, CMV, HHV6 and BK Virus Administered after Allogeneic Hematopoietic Stem Cell Transplant

Author

Papadopoulou, Anastasia, primary, Katari, Usha L., additional, Gerdemann, Ulrike, additional, Tzannou, Ifigenia, additional, Martinez, Caridad, additional, Leung, Kathryn, additional, Carrum, George, additional, Gee, Adrian P., additional, Vera, Juan F., additional, Krance, Robert A., additional, Brenner, Malcolm K., additional, Rooney, Cliona M., additional, Heslop, Helen E., additional, and Leen, Ann M., additional

Published
2014
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214. Umbilical Cord Blood Transplantation Can be an Effective Therapy for Patients with Primary Immunodeficiency Diseases Who Lack an HLA-Matched Related Donor

Author

Martinez, Caridad, primary, Shearer, William, additional, Orange, Jordan, additional, Rosenblatt, Howard, additional, Leung, Kathryn, additional, Naik, Swati, additional, Gottschalk, Stephen, additional, Allen, Carl, additional, Ahmed, Nabil, additional, Sasa, Ghadir, additional, Brenner, Malcolm K., additional, Heslop, Helen E., additional, Leen, Ann M., additional, Hanson, Imelda C., additional, and Krance, Robert A., additional

Published
2014
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215. Outcome after Stem Cell Transplant in Patients with Dyskeratosis Congenita

Author

Naik, Swati, primary, Sasa, Ghadir, additional, Bertuch, Alison, additional, Martinez, Caridad, additional, Omer, Bilal, additional, Allen, Carl, additional, Ahmed, Nabil, additional, Gottschalk, Stephen, additional, Heslop, Helen E., additional, Brenner, Malcolm K., additional, Krance, Robert A., additional, and Leung, Kathryn, additional

Published
2014
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217. Safety and Clinical Efficacy Of Rapidly-Generated Virus-Specific T Cells With Activity Against Adv, EBV, CMV, HHV6 and BK Virus Administered After Allogeneic Hematopoietic Stem Cell Transplant

Author

Papadopoulou, Anastasia, primary, Katari, Usha L, additional, Gerdemann, Ulrike, additional, Martinez, Caridad, additional, Leung, Kathryn, additional, Carrum, George, additional, Gee, Adrian P., additional, Vera, Juan F, additional, Krance, Robert A., additional, Brenner, Malcolm K., additional, Rooney, Cliona M, additional, Heslop, Helen E., additional, and Leen, Ann, additional

Published
2013
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218. Adoptive transfer of rapidly-generated multivirus-specific T cells to treat Adv, EBV, CMV, BK and HHV6 infections of HSCT recipients

Author

Papadopoulou, Anastasia, primary, Katari, Usha, additional, Gerdemann, Ulrike, additional, Martinez, Caridad, additional, Leung, Kathy, additional, Carrum, George, additional, Gee, Adrian, additional, Vera, Juan, additional, Krance, Robert, additional, Brenner, Malcolm, additional, Rooney, Cliona, additional, Heslop, Helen, additional, and Leen, Ann, additional

Published
2013
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219. Safety and clinical efficacy of rapidly-generated trivirus-directed T cells as treatment for adenovirus, EBV, and CMV infections after allogeneic hematopoietic stem cell transplant

Author

Gerdemann, Ulrike, primary, Katari, Usha L, additional, Papadopoulou, Anastasia, additional, Keirnan, Jacqueline M, additional, Craddock, John A, additional, Liu, Hao, additional, Martinez, Caridad A, additional, Kennedy-Nasser, Alana, additional, Leung, Kathryn S, additional, Gottschalk, Stephen M, additional, Krance, Robert A, additional, Brenner, Malcolm K, additional, Rooney, Cliona M, additional, Heslop, Helen E, additional, and Leen, Ann M, additional

Published
2013
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220. Delayed Marrow Infusion in Mice Enhances Hematopoietic and Osteopoietic Engraftment by Facilitating Transient Expansion of the Osteoblastic Niche

Author

Marino, Roberta, primary, Otsuru, Satoru, additional, Hofmann, Ted J., additional, Olson, Timothy S., additional, Rasini, Valeria, additional, Veronesi, Elena, additional, Boyd, Kelli, additional, Gaber, Mostafa Waleed, additional, Martinez, Caridad, additional, Paolucci, Paolo, additional, Dominici, Massimo, additional, and Horwitz, Edwin M., additional

Published
2013
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221. Pharmacotherapy for Alcohol Use Disorders: Physicians' Perceptions and Practices.

Author

Martinez, Caridad Ponce, Vakkalanka, Priyanka, Ait-Daoud, Nassima, Janiri, Luigi, and Leeman, Robert F.

Subjects
ALCOHOL-induced disorders, DRUG therapy, THERAPEUTICS
Abstract

Background and objectives: Alcohol use disorders (AUDs) are an important cause of morbidity and mortality. Despite the National Institute on Alcohol Abuse and Alcoholism (NIAAA) recommendations that medications be considered for patients with alcohol dependence, the mainstay of treatment has been counseling. We designed a survey to assess the treatment practices of psychiatrists and family medicine (FM) physicians in an effort to identify barriers to the use of pharmacotherapy and develop strategies to increase physician knowledge and utilization of these medications. Methods: An anonymous online survey was sent to FM physicians and psychiatrists nationwide. The survey collected demographic information and assessed prescription of medications in treating AUDs, including FDA-approved medications and other medications used off-label for this purpose. We also examined factors that would lead to an increase in AUDs pharmacotherapy. Results: A total of 491 surveys were completed, with 475 responses included in the final analyses. 45.5% of participants were psychiatrists vs. 54.5% FM physicians. The 74.7% respondents had used medications to treat AUDs, with psychiatrists more likely to have prescribed acamprosate, naltrexone, and several off-label medications. FM physicians were more likely to report efficacy concerns. A majority of all physicians sampled would increase pharmacotherapy of AUDs with increased training. Discussion: In our sample, most physicians have used medications to treat AUDs. There were concerns about efficacy with all non-FDA-approved medications, but limited treatment success even with FDA-approved medications. Greater education about pharmacotherapy, including predictors for treatment response amongst patients, should help alleviate some of the uncertainties reported with medications' efficacy and lead to a more individualized treatment approach. [ABSTRACT FROM AUTHOR]

Published
2016
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222. Safety and Clinical Efficacy of Rapidly-Generated Trivirus-Directed T Cells After Allogeneic Hematopoietic Stem Cell Transplant

Author

Gerdemann, Ulrike, primary, Katari, Usha, additional, Keirnan, Jacqueline, additional, Papadopoulou, Anastasia, additional, Craddock, John, additional, Liu, Hao, additional, Martinez, Caridad, additional, Kennedy-Nasser, Alana, additional, Leung, Kathryn, additional, Gottschalk, Stephen, additional, Gee, Adrian P., additional, Krance, Robert A., additional, Brenner, Malcolm K., additional, Rooney, Cliona M., additional, Heslop, Helen E., additional, and Leen, Ann M., additional

Published
2013
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224. B Cell Engraftment in SCID Patients After Stem Cell Transplantation

Author

Martinez, Caridad, primary, Shearer, William, additional, Nicholas, Sarah K., additional, Orange, Jordan, additional, Chinen, Javier, additional, Rosenblatt, Howard, additional, Wu, Jesse, additional, Bollard, Catherine M., additional, Leung, Kathryn, additional, Brenner, Malcolm K., additional, Heslop, Helen E., additional, Hanson, Imelda C., additional, and Krance, Robert A., additional

Published
2013
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225. Evaluating Donor Lymphocyte Infusions (DLI) for the Treatment of Falling Chimerism, Minimal Residual Disease and Relapse After Pediatric Hematopoietic Stem Cell Transplantation (HSCT)

Author

El-Bietar, Javier Amin, primary, Martinez, Caridad, additional, Bollard, Catherine M., additional, Naik, Swati, additional, Leung, Kathryn, additional, Gottschalk, Stephen, additional, Ahmed, Nabil, additional, Allen, Carl, additional, Heslop, Helen E., additional, Brenner, Malcolm K., additional, and Krance, Robert A., additional

Published
2013
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228. Stem Cell Transplantation and Long-Term Survival for Primary Immunodeficiencies: Outcomes Among the Donor Sources and Different Diagnostic Groups

Author

Martinez, Caridad, primary, Shearer, William, additional, Nicholas, Sarah K., additional, Orange, Jordan, additional, Chinen, Javier, additional, Rosenblatt, Howard, additional, Bollard, Catherine M., additional, Leung, Kathryn, additional, Brenner, Malcolm K., additional, Heslop, Helen E., additional, Hanson, Imelda C., additional, and Krance, Robert A., additional

Published
2013
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230. Safety and Clinical Efficacy of Rapidly-Generated Trivirus-Directed T Cells After Allogeneic Hematopoietic Stem Cell Transplant

Author

Gerdemann, Ulrike, primary, Katari, Usha L, additional, Keirnan, Jacqueline, additional, Craddock, John A., additional, Salinas, Janet, additional, Liu, Hao, additional, Martinez, Caridad, additional, Kennedy-Nasser, Alana, additional, Leung, Kathryn, additional, Gottschalk, Stephen, additional, Gee, Adrian P., additional, Krance, Robert A., additional, Brenner, Malcolm K., additional, Rooney, Cliona M., additional, Heslop, Helen E., additional, and Leen, Ann M., additional

Published
2012
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231. Improving Immune Reconstitution After Cord Blood Transplantation Using Ex Vivo Expanded Virus-Specific T Cells: A Phase I Clinical Study

Author

Hanley, Patrick J, primary, Martinez, Caridad, additional, Leung, Kathryn, additional, Savoldo, Barbara, additional, Dotti, Gianpietro, additional, Brenner, Malcolm K., additional, Rooney, Cliona M., additional, Heslop, Helen E., additional, Krance, Robert A., additional, Shpall, Elizabeth J, additional, and Bollard, Catherine M., additional

Published
2012
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232. Excellent survival after sibling or unrelated donor stem cell transplantation for chronic granulomatous disease

Author

Martinez, Caridad A., primary, Shah, Sweta, additional, Shearer, William T., additional, Rosenblatt, Howard M., additional, Paul, Mary E., additional, Chinen, Javier, additional, Leung, Kathryn S., additional, Kennedy-Nasser, Alana, additional, Brenner, Malcolm K., additional, Heslop, Helen E., additional, Liu, Hao, additional, Wu, Meng-Fen, additional, Hanson, Imelda C., additional, and Krance, Robert A., additional

Published
2012
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233. Immune reconstitution and survival of 100 SCID patients post–hematopoietic cell transplant: a PIDTC natural history study

Author

Heimall, Jennifer, Logan, Brent R., Cowan, Morton J., Notarangelo, Luigi D., Griffith, Linda M., Puck, Jennifer M., Kohn, Donald B., Pulsipher, Michael A., Parikh, Suhag, Martinez, Caridad, Kapoor, Neena, O'Reilly, Richard, Boyer, Michael, Pai, Sung-Yun, Goldman, Frederick, Burroughs, Lauri, Chandra, Sharat, Kletzel, Morris, Thakar, Monica, Connelly, James, Cuvelier, Geoff, Davila Saldana, Blachy J., Shereck, Evan, Knutsen, Alan, Sullivan, Kathleen E., DeSantes, Kenneth, Gillio, Alfred, Haddad, Elie, Petrovic, Aleksandra, Quigg, Troy, Smith, Angela R., Stenger, Elizabeth, Yin, Ziyan, Shearer, William T., Fleisher, Thomas, Buckley, Rebecca H., and Dvorak, Christopher C.

Abstract

The Primary Immune Deficiency Treatment Consortium (PIDTC) is enrolling children with severe combined immunodeficiency (SCID) to a prospective natural history study. We analyzed patients treated with allogeneic hematopoietic cell transplantation (HCT) from 2010 to 2014, including 68 patients with typical SCID and 32 with leaky SCID, Omenn syndrome, or reticular dysgenesis. Most (59%) patients were diagnosed by newborn screening or family history. The 2-year overall survival was 90%, but was 95% for those who were infection-free at HCT vs 81% for those with active infection (P= .009). Other factors, including the diagnosis of typical vs leaky SCID/Omenn syndrome, diagnosis via family history or newborn screening, use of preparative chemotherapy, or the type of donor used, did not impact survival. Although 1-year post-HCT median CD4 counts and freedom from IV immunoglobulin were improved after the use of preparative chemotherapy, other immunologic reconstitution parameters were not affected, and the potential for late sequelae in extremely young infants requires additional evaluation. After a T-cell–replete graft, landmark analysis at day +100 post-HCT revealed that CD3 < 300 cells/μL, CD8 < 50 cells/μL, CD45RA < 10%, or a restricted Vβ T-cell receptor repertoire (<13 of 24 families) were associated with the need for a second HCT or death. In the modern era, active infection continues to pose the greatest threat to survival for SCID patients. Although newborn screening has been effective in diagnosing SCID patients early in life, there is an urgent need to identify validated approaches through prospective trials to ensure that patients proceed to HCT infection free. The trial was registered at www.clinicaltrials.govas #NCT01186913.

Published
2017
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234. Phase I Study to Improve Virus-Specific Immune Reconstitution After Cord Blood Transplantation Using Cord Blood-Derived Virus-Specific Cytotoxic T Lymphocytes

Author

Hanley, Patrick J, primary, Martinez, Caridad, additional, Leung, Kathryn, additional, Savaldo, Barbara, additional, Dotti, Gianpietro, additional, Gee, Adrian P., additional, Rooney, Cliona M., additional, Heslop, Helen E., additional, Krance, Robert A., additional, Shpall, Elizabeth J, additional, and Bollard, Catherine M., additional

Published
2011
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235. Inducible Apoptosis as a Safety Switch for Adoptive Cell Therapy

Author

Di Stasi, Antonio, primary, Tey, Siok-Keen, additional, Dotti, Gianpietro, additional, Fujita, Yuriko, additional, Kennedy-Nasser, Alana, additional, Martinez, Caridad, additional, Straathof, Karin, additional, Liu, Enli, additional, Durett, April G., additional, Grilley, Bambi, additional, Liu, Hao, additional, Cruz, Conrad R., additional, Savoldo, Barbara, additional, Gee, Adrian P., additional, Schindler, John, additional, Krance, Robert A., additional, Heslop, Helen E., additional, Spencer, David M., additional, Rooney, Cliona M., additional, and Brenner, Malcolm K., additional

Published
2011
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236. The clinical use of cord blood-derived virus-specific cytotoxic T lymphocytes reactive against Cytomegalovirus, Adenovirus, and Epstein-Barr Virus (105.50)

Author

Hanley, Patrick, primary, Martinez, Caridad, additional, Leung, Kathryn, additional, Cruz, Conrad Russell, additional, Savoldo, Barbara, additional, Rodgers, John, additional, Leen, Ann, additional, Dotti, Gianpietro, additional, Gee, Adrian, additional, Rooney, Cliona, additional, Heslop, Helen, additional, Krance, Robert, additional, Shpall, Elizabeth, additional, and Bollard, Catherine, additional

Published
2011
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238. CASPALLO: Phase I Clinical Trial of Allodepleted T Cells Transduced with Inducible Caspase 9 Suicide Gene After Haploidentical Stem Cell Transplantation

Author

Di Stasi, Antonio, primary, Tey, Siok K, additional, Fujita, Yuriko, additional, Cruz, Russell, additional, Kennedy-Nasser, Alana, additional, Martinez, Caridad, additional, Gottschalk, Stephen M, additional, Straathof, Karin, additional, Liu, Enli, additional, Durett, April, additional, Grilley, Bambi J, additional, Liu, Hao, additional, Savoldo, Barbara, additional, Eric, Yvon, additional, Lopez, Teresita, additional, Diouf, Oumar, additional, Gee, Adrian P., additional, Schindler, John, additional, Bollard, Catherine M, additional, Krance, Robert A, additional, Spencer, David M, additional, Rooney, Cliona M, additional, Heslop, Helen E, additional, Dotti, Gianpietro, additional, and Brenner, Malcolm K, additional

Published
2010
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239. The Costs and Cost-Effectiveness of Allogeneic Peripheral Blood Stem Cell Transplantation versus Bone Marrow Transplantation in Pediatric Patients with Acute Leukemia

Author

Lin, Yu-Feng, primary, Lairson, David R., additional, Chan, Wenyaw, additional, Du, Xianglin L., additional, Leung, Kathryn S., additional, Kennedy-Nasser, Alana A., additional, Martinez, Caridad A., additional, Gottschalk, Stephen M., additional, Bollard, Catherine M., additional, Heslop, Helen E., additional, Brenner, Malcolm K., additional, and Krance, Robert A., additional

Published
2010
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242. Une métaphore à double sens; le voyage chez Ronsard et Montaigne

Author

Martinez, Caridad and Université de Barcelone

Abstract

Zadanie pt. „Digitalizacja i udostępnienie w Cyfrowym Repozytorium Uniwersytetu Łódzkiego kolekcji czasopism naukowych wydawanych przez Uniwersytet Łódzki” nr 885/P-DUN/2014 dofinansowane zostało ze środków MNiSW w ramach działalności upowszechniającej naukę

Published
1992

245. Outcomes after Second Hematopoietic Stem Cell Transplantations in Pediatric Patients with Relapsed Hematological Malignancies.

Author

Naik, Swati, Martinez, Caridad, Leung, Kathryn, Sasa, Ghadir, Nguyen, Ngoc-Yen, Wu, Meng-Fen, Gottschalk, Stephen, Brenner, Malcolm, Heslop, Helen, and Krance, Robert

Subjects
*HEMATOPOIETIC stem cell transplantation, *DISEASE relapse, *HEMATOLOGIC malignancies, *JUVENILE diseases, *MEDICAL statistics, *PROGNOSIS, *THERAPEUTICS
Abstract

Relapse of hematological malignancies after hematopoietic stem cell transplantation (HCT) is associated with poor prognosis. A second HCT represents one of the few therapeutic options for these high-risk patients. For children undergoing second HCT, the outcome data are particularly limited. We, therefore, conducted a retrospective single-institution study and report the outcomes and prognostic variables associated with overall survival (OS) and relapse in 43 pediatric patients who underwent a second HCT between 2000 and 2013. Eleven of the 43 patients who underwent transplantation remain alive and disease-free at a median follow-up of 49 months (range, 5 to 127 months). The 5-year probability of OS for the entire cohort was 24%. Patients who had early relapse (<6 months) after first HCT had significantly worse OS than those who relapsed late (>6 months), with 5-year OS at 11% versus 34%, respectively (hazard ratio [HR], 2.24; 95% confidence interval [CI], 1.21 to 4.93; P = .013). Active disease at time of second HCT was also associated with a significantly increased risk of relapse (subdistribution hazard ratio [SHR], 2.36; P = .049) for the entire cohort and relapse was the most frequent cause of death (23 of 32; 72%). On subgroup analysis for the 34 patients with leukemia alone, presence of active disease was associated both with a significant decrease in OS (SHR, 2.28; 95% CI, 1.02 to 5.09; P = .044) and significant increase in the rate of relapse (SHR, 2.46; P = .046). By contrast, underlying disease, donor source, conditioning regimen, or development of GVHD did not modify OS or rate of relapse. Hence, a second HCT appears to be a useful therapeutic option in children with relapsed hematological malignancies that is most likely to benefit those individuals with late onset of relapse and with low disease burden at the time of transplantation. [ABSTRACT FROM AUTHOR]

Published
2015
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246. Ronsard i postęp - świadomość zmienności czy kompleks dinozaura?

Author

Martinez, Caridad and Université de Barcelona

Abstract

W swym artykule Caridad Martinez omawia frapujący ciągle temat rozwoju cywilizacji i jej skutków dla człowieka. Materiał do analizy stanowi wybrana z dzieła Ronsarda pewna grupa poematów, charakteryzująca się jako wyróżnikiem obecnością formuły "pierwszy, który". Na podstawie badanych utworów autorka dochodzi do następujących wniosków. Stosunek Ronsarda do rozwoju cywilizacji nie jest jednoznaczny. "Książę Poetów" z jednej strony wita z radością i entuzjazmem rozwój nauki, literatury, postęp w dziedzinach technicznych, dzięki pracy homo faber, z drugiej jednak boleje nad utratą stanu szczęśliwości, owego "złotego wieku", z którego postęp wyrwał ludzkość. Autorka zauważa w wybranych utworach pewną ewolucję w ocenie postępu cywilizacyjnego. Jej zdaniem można wyróżnić u Ronsarda cztery etapy:1) poeta nie wypowiada się w ogóle (Martinez określa to mianem "stopnia zero"); 2 ) poeta jest świadom postępu i zwłaszcza rozwój literatury i sztuki wita z radością; 3) poeta w postępie w dziedzinie technicznej widzi źródło degeneracji i powód wszelkich nieszczęść; 4) okres powstawania utworów,których sens jest często sprzeczny lub niejednoznaczny. W konkluzji autorka dochodzi do wniosku, że Ronsard świadom jest nieuchronności postępu i rozwoju cywilizacji, zdając sobie równocześnie sprawę z konieczności rozwagi i roztropności. Poeta wygłasza pochwałę pracy odnosząc się zarazem z pogardą do lenistwa i bezczynności, a gorąca wiara w dążenie ku nowemu, zgodna z ludyczną koncepcją jego twórczości, nie opuszczała go do końca życia. Zadanie pt. „Digitalizacja i udostępnienie w Cyfrowym Repozytorium Uniwersytetu Łódzkiego kolekcji czasopism naukowych wydawanych przez Uniwersytet Łódzki” nr 885/P-DUN/2014 dofinansowane zostało ze środków MNiSW w ramach działalności upowszechniającej naukę

Published
1990

248. Blinatumomab Therapy Is Associated with Favorable Outcomes after Allogeneic Hematopoietic Cell Transplantation in Pediatric Patients with B Cell Acute Lymphoblastic Leukemia

Author

Llaurador, Gabriela, Shaver, Kristen, Wu, Mengfen, Wang, Tao, Gillispie, Amanda, Doherty, Erin, Craddock, John, Read, Jay, Yassine, Khaled, Morales, Erin, George, Anil, Steffin, David, Krance, Robert, Martinez, Caridad, Heslop, Helen, and Salem, Baheyeldin

Abstract

•Blinatumomab increased leukemia-free survival in patients with B cell acute lymphoblastic leukemia (B-ALL) after allogeneic hematopoietic cell transplantation (allo-HCT).•Blinatumomab did not increase immune toxicities after allo-HCT.•Blinatumomab is a safe and effective salvage therapy for B-ALL before allo-HCT.

Published
2023
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249. Outcomes after Hematopoietic Cell Transplant and Gene Therapy for Adenosine Deaminase (ADA) Severe Combined Immune Deficiency: A Combined Analysis from the Primary Immune Deficiency Treatment Consortium (PIDTC) 6901 and 6902 Studies

Author

Cuvelier, Geoff D.E., Logan, Brent, Prockop, Susan E., Buckley, Rebecca H., Kuo, Caroline Y., Griffith, Linda M., Liu, Xuerong, Yip, Alison, Hershfield, Michael S., Parrott, Roberta E, Ebens, Christen L., Moore, Theodore B., O’Reilly, Richard J., Pai, Sung-Yun, Kapadia, Malika, Kapoor, Neena, Forbes Satter, Lisa R., Martinez, Caridad, Burroughs, Lauri M., Petrovic, Aleksandra, Thakar, Monica S., Chellapandian, Deepak, Heimall, Jennifer, Shyr, David C., Rayes, Ahmad, Bednarski II, Jeffrey J., Chandra, Sharat, Chandrakasan, Shanmuganathan, Gillio, Alfred P., Madden, Lisa, Quigg, Troy C., Caywood, Emi H., Dávila Saldaña, Blachy J, DeSantes, Kenneth, Eissa, Hesham, Goldman, Frederick, Rozmus, Jacob, Shah, Ami J, Vander Lugt, Mark T., Pulsipher, Mike A., Notarangelo, Luigi D., Cowan, Morton J., Dvorak, Christopher C., Haddad, Elie, Puck, Jennifer, and Kohn, Donald B.

Published
2022
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250. A Primary Immune Deficiency Treatment Consortium (PIDTC) Study of Chronic and Late Onset Medical Complications after Initial Hematopoietic Cell Transplantation (HCT) for Severe Combined Immunodeficiency Disease (SCID)

Author

Eissa, Hesham, Thakar, Monica S., Shah, Ami J, Buckley, Rebecca H., Logan, Brent, Griffith, Linda M., Dong, Huaying, O’Reilly, Richard J., Kapoor, Neena, Satter, Lisa Forbes, Chandra, Sharat, Bleesing, Jack J., Kapadia, Malika, Parrott, Roberta E, Chandrakasan, Shanmuganathan, Bednarski II, Jeffrey J., Jyonouchi, Soma, Madden, Lisa M., Rayes, Ahmad, Ebens, Christen L., Teira, Pierre, Dávila Saldaña, Blachy J, Burroughs, Lauri M., Prockop, Susan E., Williams, Olatundun, Chellapandian, Deepak, Gillio, Alfred P., Goldman, Frederick, Malech, Harry L., DeSantes, Kenneth, Cuvelier, Geoff D.E., Rozmus, Jacob, Quinones, Ralph, Yu, Lolie C., Broglie, Larisa, Aquino, Victor, Shereck, Evan, Moore, Theodore B., Martinez, Caridad, Vander Lugt, Mark T., Leiding, Jennifer W., Torgerson, Troy, Pai, Sung-Yun, Pulsipher, Mike A., Notarangelo, Luigi D., Puck, Jennifer, Dvorak, Christopher C., Haddad, Elie, Cowan, Morton J., and Heimall, Jennifer

Published
2022
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