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  1. Article: Successful Pregnancy Outcome in a Hereditary Angioedema Patient With Previous Pregnancy Losses: A Proposed Delivery Plan.

    Satnarine, Travis / Xavier de Almeida, Alana / Gebbia, Jennifer / Kleiner, Gary / Gans, Melissa

    Cureus

    2024  Volume 16, Issue 2, Page(s) e54939

    Abstract: This case report underscores the effective implementation of a delivery plan for a pregnant patient, focusing on a successful case study where a cesarean section, preceded by the pre-treatment of intravenous plasma-derived C1 inhibitor, resulted in the ... ...

    Abstract This case report underscores the effective implementation of a delivery plan for a pregnant patient, focusing on a successful case study where a cesarean section, preceded by the pre-treatment of intravenous plasma-derived C1 inhibitor, resulted in the delivery of a healthy baby. The proposed delivery plan offers a systematic approach to managing hereditary angioedema during pregnancy. It recommends opting for delivery at an academic center equipped with high-risk obstetric care, obstetric anesthesia, and a level 4 Neonatal Intensive Care Unit. The plan also emphasizes the importance of early admission at the onset of labor and delineates specific protocols for both vaginal and cesarean deliveries.
    Language English
    Publishing date 2024-02-26
    Publishing country United States
    Document type Case Reports
    ZDB-ID 2747273-5
    ISSN 2168-8184
    ISSN 2168-8184
    DOI 10.7759/cureus.54939
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  2. Article ; Online: Post-SARS-CoV-2 Atypical Inflammatory Syndrome in a Toddler with X-Linked Inhibitor of Apoptosis Deficiency After Stem Cell Transplant

    Narahari, Prasanth G. / Gebbia, Jennifer / Alperstein, Warren / Kleiner, Gary / Gans, Melissa

    J Clin Immunol. 2022 Nov., v. 42, no. 8, p. 1600-1603

    2022  , Page(s) 1600–1603

    Abstract: A 10-month-old boy was diagnosed with X-linked lymphoproliferative syndrome type 2 due to X-linked inhibitor of apoptosis deficiency after presenting with failure to thrive and refractory inflammatory bowel disease. He underwent a matched unrelated donor ...

    Abstract A 10-month-old boy was diagnosed with X-linked lymphoproliferative syndrome type 2 due to X-linked inhibitor of apoptosis deficiency after presenting with failure to thrive and refractory inflammatory bowel disease. He underwent a matched unrelated donor stem cell transplant with reduced intensity conditioning at 16 months. At 27 months, he presented with an atypical inflammatory syndrome in the setting of recent COVID-19 infection, Epstein-Barr viremia, and low chimerism (7.3%). He recovered after treatment with intravenous immunoglobulin and steroids.
    Keywords COVID-19 infection ; apoptosis ; boys ; chimerism ; failure to thrive ; immunoglobulins ; inflammatory bowel disease ; intravenous injection ; stem cells ; viremia
    Language English
    Dates of publication 2022-11
    Size p. 1600-1603
    Publishing place Springer US
    Document type Article ; Online
    Note Letter
    ZDB-ID 779361-3
    ISSN 1573-2592 ; 0271-9142
    ISSN (online) 1573-2592
    ISSN 0271-9142
    DOI 10.1007/s10875-022-01316-3
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  3. Article ; Online: Post-SARS-CoV-2 Atypical Inflammatory Syndrome in a Toddler with X-Linked Inhibitor of Apoptosis Deficiency After Stem Cell Transplant.

    Narahari, Prasanth G / Gebbia, Jennifer / Alperstein, Warren / Kleiner, Gary / Gans, Melissa

    Journal of clinical immunology

    2022  Volume 42, Issue 8, Page(s) 1600–1603

    Abstract: A 10-month-old boy was diagnosed with X-linked lymphoproliferative syndrome type 2 due to X-linked inhibitor of apoptosis deficiency after presenting with failure to thrive and refractory inflammatory bowel disease. He underwent a matched unrelated donor ...

    Abstract A 10-month-old boy was diagnosed with X-linked lymphoproliferative syndrome type 2 due to X-linked inhibitor of apoptosis deficiency after presenting with failure to thrive and refractory inflammatory bowel disease. He underwent a matched unrelated donor stem cell transplant with reduced intensity conditioning at 16 months. At 27 months, he presented with an atypical inflammatory syndrome in the setting of recent COVID-19 infection, Epstein-Barr viremia, and low chimerism (7.3%). He recovered after treatment with intravenous immunoglobulin and steroids.
    MeSH term(s) Male ; Humans ; Child, Preschool ; Infant ; X-Linked Inhibitor of Apoptosis Protein ; SARS-CoV-2 ; COVID-19/diagnosis ; Lymphoproliferative Disorders/diagnosis ; Hematopoietic Stem Cell Transplantation/adverse effects ; Apoptosis
    Chemical Substances X-Linked Inhibitor of Apoptosis Protein
    Language English
    Publishing date 2022-07-11
    Publishing country Netherlands
    Document type Letter
    ZDB-ID 779361-3
    ISSN 1573-2592 ; 0271-9142
    ISSN (online) 1573-2592
    ISSN 0271-9142
    DOI 10.1007/s10875-022-01316-3
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  4. Article ; Online: Allergies, Allergic Comorbidities and the Home Environment in Pediatric Asthma in Southern Florida.

    Saif, Nadia T / Kleiner, Gary I / Forster, Lourdes Q / Hershorin, Eugene R / Colin, Andrew A / Mirsaeidi, Mehdi / Kumar, Naresh

    International journal of environmental research and public health

    2021  Volume 18, Issue 8

    Abstract: Background: ...

    Abstract Background:
    MeSH term(s) Allergens ; Asthma/epidemiology ; Child ; Child, Preschool ; Cross-Sectional Studies ; Florida/epidemiology ; Humans ; Prevalence ; Rhinitis, Allergic/epidemiology ; Surveys and Questionnaires
    Chemical Substances Allergens
    Language English
    Publishing date 2021-04-14
    Publishing country Switzerland
    Document type Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
    ZDB-ID 2175195-X
    ISSN 1660-4601 ; 1661-7827
    ISSN (online) 1660-4601
    ISSN 1661-7827
    DOI 10.3390/ijerph18084142
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  5. Article: Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation.

    Corti, Manuela / Byrne, Barry J / Gessler, Dominic J / Thompson, Grace / Norman, Samantha / Lammers, Jenna / Coleman, Kirsten E / Liberati, Cristina / Elder, Melissa E / Escolar, Maria L / Tuna, Ibrahim S / Mesaros, Clementina / Kleiner, Gary I / Barbouth, Deborah S / Gray-Edwards, Heather L / Clement, Nathalie / Cleaver, Brian D / Gao, Guangping

    Molecular therapy. Methods & clinical development

    2023  Volume 30, Page(s) 303–314

    Abstract: Gene replacement therapy is a rational therapeutic strategy and clinical intervention for neurodegenerative disorders like Canavan disease, a leukodystrophy caused by biallelic mutations in the aspartoacylase ( ...

    Abstract Gene replacement therapy is a rational therapeutic strategy and clinical intervention for neurodegenerative disorders like Canavan disease, a leukodystrophy caused by biallelic mutations in the aspartoacylase (
    Language English
    Publishing date 2023-06-19
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2872938-9
    ISSN 2329-0501 ; 2329-0501
    ISSN (online) 2329-0501
    ISSN 2329-0501
    DOI 10.1016/j.omtm.2023.06.001
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  6. Article ; Online: Pharmacokinetics, Safety, and Tolerability of Subcutaneous Immune Globulin Injection (Human), 10 % Caprylate/Chromatography Purified (GAMUNEX®-C) in Pediatric Patients with Primary Immunodeficiency Disease.

    Heimall, Jennifer / Chen, Junliang / Church, Joseph A / Griffin, Rhonda / Melamed, Isaac / Kleiner, Gary I

    Journal of clinical immunology

    2016  Volume 36, Issue 6, Page(s) 600–609

    Abstract: Purpose: This phase 4, multicenter, open-labeled, single-sequence, crossover study in pediatric patients (ages 2 to 16) with primary immunodeficiency disease (PID) evaluated the pharmacokinetics, safety, and tolerability for subcutaneously (SC) ... ...

    Abstract Purpose: This phase 4, multicenter, open-labeled, single-sequence, crossover study in pediatric patients (ages 2 to 16) with primary immunodeficiency disease (PID) evaluated the pharmacokinetics, safety, and tolerability for subcutaneously (SC) administered 10 % caprylate/chromatography purified human immune globulin injection (IGIV-C, GAMUNEX®) compared with intravenously (IV) administered IGIV-C.
    Methods: This study included a screening phase, run-in phase (where required), IV treatment phase, SC treatment phase, and end of study/early termination visit. Eligible patients receiving a stable dose of IGIV-C entered into the IV phase to receive two IV infusions of IGIV-C (200-600 mg/kg per infusion) every 3-4 weeks. The weekly SC dose of IGIV-C was calculated using a conversion factor of 1.37 times the prior IV dose.
    Results: Twelve subjects between the ages of 2 and 16 years participated in the clinical study with the median age being 11 years old. The adjusted weekly mean AUC0-τ,IV was 216,873.7 h*mg/dL for the IV phase versus a mean AUC0-τ,SC of 230,830.0 h*mg/dL for the SC phase. The mean (range) C trough was 997.2 (784-1320) mg/dL in the IV phase and 1325.0 (1077-1690) mg/dL in the SC phase. During the SC phase, 100.0 % of the patients (n = 11) experienced treatment-emergent adverse events (TEAEs) that were local infusion reactions and 9 patients (81.8 %) had TEAEs that were non-infusion site reactions. The majority of TEAEs were mild or moderate in severity.
    Conclusion: In pediatric patients with PID, SC-administered IGIV-C provides comparable overall serum exposure to total IgG to that produced by IV-administered IGIV-C. We have concluded that weekly SC administration of 10 % IGIV-C based on a dose conversion factor of 1.37 is safe and well-tolerated in pediatric patients with PID.
    Trial registration: ClinicalTrials.gov identifier: NCT01465958. https://clinicaltrials.gov/ct2/show/NCT01465958?term=NCT01465958.&rank=1.
    MeSH term(s) Adolescent ; Antibodies, Neutralizing/blood ; Area Under Curve ; Bacterial Infections/etiology ; Child ; Child, Preschool ; Drug Monitoring ; Female ; Humans ; Immunoglobulins, Intravenous/pharmacology ; Immunoglobulins, Intravenous/therapeutic use ; Immunologic Deficiency Syndromes/blood ; Immunologic Deficiency Syndromes/diagnosis ; Immunologic Deficiency Syndromes/drug therapy ; Injection Site Reaction/etiology ; Male ; Treatment Outcome
    Chemical Substances Antibodies, Neutralizing ; Immunoglobulins, Intravenous
    Language English
    Publishing date 2016-06-25
    Publishing country Netherlands
    Document type Clinical Trial ; Journal Article ; Multicenter Study ; Research Support, Non-U.S. Gov't
    ZDB-ID 779361-3
    ISSN 1573-2592 ; 0271-9142
    ISSN (online) 1573-2592
    ISSN 0271-9142
    DOI 10.1007/s10875-016-0311-4
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  7. Article ; Online: Relevance of lymphocyte proliferation to PHA in severe combined immunodeficiency (SCID) and T cell lymphopenia.

    Abraham, Roshini S / Basu, Amrita / Heimall, Jennifer R / Dunn, Elizabeth / Yip, Alison / Kapadia, Malika / Kapoor, Neena / Satter, Lisa Forbes / Buckley, Rebecca / O'Reilly, Richard / Cuvelier, Geoffrey D E / Chandra, Sharat / Bednarski, Jeffrey / Chaudhury, Sonali / Moore, Theodore B / Haines, Hilary / Dávila Saldaña, Blachy J / Chellapandian, Deepakbabu / Rayes, Ahmad /
    Chen, Karin / Caywood, Emi / Chandrakasan, Shanmuganathan / Lugt, Mark Thomas Vander / Ebens, Christen / Teira, Pierre / Shereck, Evan / Miller, Holly / Aquino, Victor / Eissa, Hesham / Yu, Lolie C / Gillio, Alfred / Madden, Lisa / Knutsen, Alan / Shah, Ami J / DeSantes, Kenneth / Barnum, Jessie / Broglie, Larisa / Joshi, Avni Y / Kleiner, Gary / Dara, Jasmeen / Prockop, Susan / Martinez, Caridad / Mousallem, Talal / Oved, Joseph / Burroughs, Lauri / Marsh, Rebecca / Torgerson, Troy R / Leiding, Jennifer W / Pai, Sung Yun / Kohn, Donald B / Pulsipher, Michael A / Griffith, Linda M / Notarangelo, Luigi D / Cowan, Morton J / Puck, Jennifer / Dvorak, Christopher C / Haddad, Elie

    Clinical immunology (Orlando, Fla.)

    2024  Volume 261, Page(s) 109942

    Abstract: Severe combined immunodeficiency (SCID) is characterized by a severe deficiency in T cell numbers. We analyzed data collected (n = 307) for PHA-based T cell proliferation from the PIDTC SCID protocol 6901, using either a radioactive or flow cytometry ... ...

    Abstract Severe combined immunodeficiency (SCID) is characterized by a severe deficiency in T cell numbers. We analyzed data collected (n = 307) for PHA-based T cell proliferation from the PIDTC SCID protocol 6901, using either a radioactive or flow cytometry method. In comparing the two groups, a smaller number of the patients tested by flow cytometry had <10% of the lower limit of normal proliferation as compared to the radioactive method (p = 0.02). Further, in patients with CD3+ T cell counts between 51 and 300 cells/μL, there was a higher proliferative response with the PHA flow assay compared to the
    MeSH term(s) Infant, Newborn ; Humans ; Severe Combined Immunodeficiency/diagnosis ; Lymphopenia/diagnosis ; Neonatal Screening/methods ; T-Lymphocytes ; Cell Proliferation
    Language English
    Publishing date 2024-02-15
    Publishing country United States
    Document type Journal Article ; Research Support, N.I.H., Extramural
    ZDB-ID 1459903-x
    ISSN 1521-7035 ; 1521-6616
    ISSN (online) 1521-7035
    ISSN 1521-6616
    DOI 10.1016/j.clim.2024.109942
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  8. Article ; Online: Flebogamma(®) 5 % DIF Intravenous Immunoglobulin for Replacement Therapy in Children with Primary Immunodeficiency Diseases.

    Ballow, Mark / Pinciaro, Paul J / Craig, Timothy / Kleiner, Gary / Moy, James / Ochs, Hans D / Sleasman, John / Smits, William

    Journal of clinical immunology

    2016  Volume 36, Issue 6, Page(s) 583–589

    Abstract: Purpose: The previous studies with Flebogamma(®) 5 % DIF intravenous immunoglobulin (IVIG) contained insufficient numbers of pediatric subjects to fully warrant a pediatric indication by the FDA. The objective of this study was to evaluate the efficacy, ...

    Abstract Purpose: The previous studies with Flebogamma(®) 5 % DIF intravenous immunoglobulin (IVIG) contained insufficient numbers of pediatric subjects to fully warrant a pediatric indication by the FDA. The objective of this study was to evaluate the efficacy, safety, and pharmacokinetics of Flebogamma® 5 % DIF for replacement therapy in children (age 2-16) with primary immunodeficiency diseases (PIDD).
    Methods: IVIG was administered at eight clinical sites to 24 subjects with well-defined PIDD at a dose of 300-800 mg/kg every 21-28 days for 12 months. The pharmacokinetics endpoint in this study was the dose-adjusted increment of the serum IgG trough levels.
    Results: The calculated serious bacterial infection rate was 0.05/subject/year. The incidence of adverse events considered potentially related to IVIG during or within 72 h after completing an infusion was within the FDA guidance threshold of <40 % at each time point. Dose-adjusted incremental IgG levels remained approximately equal to or slightly greater than pre-study IgG levels (between 800 and 1000 mg/dL throughout) when the subjects were treated with IVIG therapy other than Flebogamma(®) DIF 5 % indicating no evidence of a different pharmacokinetic profile in this pediatric population if compared to those profiles in previous Flebogamma studies in predominately adult populations.
    Conclusions: Flebogamma(®) 5 % DIF is efficacious and safe, has adequate pharmacokinetic properties, is well-tolerated, and maintains the profile of Flebogamma(®) 5 % for the treatment of children with primary humoral immunodeficiency diseases.
    MeSH term(s) Adolescent ; Bacterial Infections/etiology ; Child ; Child, Preschool ; Female ; Humans ; Immunoglobulins, Intravenous/administration & dosage ; Immunoglobulins, Intravenous/adverse effects ; Immunoglobulins, Intravenous/pharmacokinetics ; Immunologic Deficiency Syndromes/diagnosis ; Immunologic Deficiency Syndromes/drug therapy ; Male ; Time Factors ; Treatment Outcome
    Chemical Substances Immunoglobulins, Intravenous
    Language English
    Publishing date 2016-06-08
    Publishing country Netherlands
    Document type Clinical Trial, Phase IV ; Journal Article ; Multicenter Study ; Research Support, Non-U.S. Gov't
    ZDB-ID 779361-3
    ISSN 1573-2592 ; 0271-9142
    ISSN (online) 1573-2592
    ISSN 0271-9142
    DOI 10.1007/s10875-016-0303-4
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  9. Article: Neuron-like differentiation of adipose-derived stem cells from infant piglets in vitro.

    Huang, Tingting / He, Dansha / Kleiner, Gary / Kuluz, John

    The journal of spinal cord medicine

    2007  Volume 30 Suppl 1, Page(s) S35–40

    Abstract: Background/objective: Adipose-derived stem cells (ADSCs) are mesenchymal stem cells (MSCs) that can be extracted from adipose tissue and obtained by a less invasive method and in larger quantities compared with bone marrow-derived MSCs. The objective of ...

    Abstract Background/objective: Adipose-derived stem cells (ADSCs) are mesenchymal stem cells (MSCs) that can be extracted from adipose tissue and obtained by a less invasive method and in larger quantities compared with bone marrow-derived MSCs. The objective of this study was to harvest ADSCs from piglets and to explore their neuronal differentiation potential.
    Methods: Adipose tissue from piglet facial or abdominal fat was digested with collagenase type XI, followed by filter and centrifugation; the isolated adipose stromal cells were cultured in dishes. MSC markers were measured by flow cytometry; 2 to 5 passage cells were used for in vitro differentiation. Adipogenic, chondrogenic, osteogenic, and neuronal differentiation was induced by incubation of the ADSCs with different induction media.
    Results: ADSCs were easily expanded to beyond 15 passages, maintaining the undifferentiated state and exhibiting MSC characteristics and markers CD29, CD44, and CD90. ADSCs differentiated into other mesodermal cells including adipocytes, chondrocytes, and osteocytes. These cells were induced to differentiate into neuron-like cells as evidenced by neuronal morphology and the presence of neuronal markers including microtubule-associated protein 2, neuronal nuclear antigen, and beta-tubulin III.
    Conclusions: ADSCs can be readily obtained from a small amount fat tissue and expanded in culture. Adipose tissue may be an alternative source of stem cell therapy for nervous system injury.
    MeSH term(s) Adipose Tissue/cytology ; Animals ; Animals, Newborn ; Antigens, CD/metabolism ; Cell Differentiation/physiology ; Cells, Cultured ; Flow Cytometry ; Nerve Tissue Proteins/metabolism ; Neurons/physiology ; Stem Cells/physiology ; Swine ; Time Factors
    Chemical Substances Antigens, CD ; Nerve Tissue Proteins
    Language English
    Publishing date 2007-08-12
    Publishing country England
    Document type Journal Article ; Research Support, N.I.H., Extramural
    ZDB-ID 1223949-5
    ISSN 2045-7723 ; 1079-0268
    ISSN (online) 2045-7723
    ISSN 1079-0268
    DOI 10.1080/10790268.2007.11753967
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  10. Article ; Online: Efficacy and safety of ex vivo cultured adult human mesenchymal stem cells (Prochymal™) in pediatric patients with severe refractory acute graft-versus-host disease in a compassionate use study.

    Prasad, Vinod K / Lucas, Kenneth G / Kleiner, Gary I / Talano, Julie An M / Jacobsohn, David / Broadwater, Gloria / Monroy, Rod / Kurtzberg, Joanne

    Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation

    2011  Volume 17, Issue 4, Page(s) 534–541

    Abstract: Preliminary studies using directed-donor ex vivo expanded human mesenchymal stem cells (hMSCs) have shown promise in the treatment of acute graft-versus-host disease (aGVHD). However, their production is cumbersome and standardization is difficult. We ... ...

    Abstract Preliminary studies using directed-donor ex vivo expanded human mesenchymal stem cells (hMSCs) have shown promise in the treatment of acute graft-versus-host disease (aGVHD). However, their production is cumbersome and standardization is difficult. We describe the first experience of using a premanufactured, universal donor, formulation of hMSCs (Prochymal) in children (n = 12; 10 boys; 9 Caucasian; age range: 0.4-15 years) with treatment-resistant grade III and IV aGVHD who received therapy on compassionate use basis between July 2005 and June 2007 at 5 transplant centers. All patients had stage III or IV gut (GI) symptoms and half had additional liver and/or skin involvement. Disease was refractory to steroids in all cases and additionally to a median of 3 other immunosuppressive therapies. The hMSCs (8 × 10(6)cells/kg/dose in 2 patients and 2 × 10(6)cells/kg/dose in the rest) were infused intravenously over 1 hour twice a week for 4 weeks. Partial and mixed responders received subsequent weekly therapy for 4 weeks. HLA or other matching was not needed. The hMSCs were started at a median of 98 days (range: 45-237) posttransplant. A total of 124 doses were administered, with a median of 8 doses (range: 2-21) per patient. Overall, 7 (58%) patients had complete response, 2 (17%) partial response, and 3 (25%) mixed response. Complete resolution of GI symptoms occurred in 9 (75%) patients. Two patients relapsed after initial response and showed partial response to retreatment. The cumulative incidence of survival at 100 days from the initiation of Prochymal therapy was 58%. Five of 12 patients (42%) were still alive after a median follow-up of 611 days (range: 427-1111) in surviving patients. No infusional or other identifiable acute toxicity was seen in any patient. Multiple infusions of hMSCs were well tolerated and appeared to be safe in children. Clinical responses, particularly in the GI system, were seen in the majority of children with severe refractory aGVHD. Given the favorable results observed in a patient population with an otherwise grave prognosis, we conclude that hMSCs hold potential for the treatment of aGVHD, and should be further studied in phase III trials in pediatric and adult patients.
    MeSH term(s) Acute Disease ; Adolescent ; Adult ; Adult Stem Cells ; Cell Culture Techniques ; Cells, Cultured ; Child ; Child, Preschool ; Compassionate Use Trials ; Disease-Free Survival ; Female ; Follow-Up Studies ; Graft vs Host Disease/mortality ; Graft vs Host Disease/therapy ; Hematologic Neoplasms/mortality ; Hematologic Neoplasms/therapy ; Humans ; Infant ; Male ; Mesenchymal Stem Cell Transplantation ; Mesenchymal Stromal Cells ; Retrospective Studies ; Survival Rate ; Time Factors ; Transplantation, Homologous
    Language English
    Publishing date 2011-04
    Publishing country United States
    Document type Clinical Trial ; Journal Article ; Multicenter Study
    ZDB-ID 1474865-4
    ISSN 1523-6536 ; 1083-8791
    ISSN (online) 1523-6536
    ISSN 1083-8791
    DOI 10.1016/j.bbmt.2010.04.014
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