About

Catherine S. Manno is an Emeritus Professor and the Physician-in-Chief at The Children's Hospital of Philadelphia. She is affiliated with the Department of Pediatrics and has a distinguished career in pediatric medicine. Her educational background includes an A.B. from Duke University obtained in 1974 and an M.D. from Hahnemann Medical College in 1978. Dr. Manno's work has focused on pediatric hematology and gene therapy, with significant contributions to the treatment of hemophilia and autoimmune conditions. Her research includes gene transfer techniques, such as AAV mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B, and she has published extensively on gene therapy and autoimmune lymphoproliferative syndromes. Her expertise and research have advanced the understanding and treatment of pediatric hematologic disorders.

Research topics

• Medicine
• Internal medicine
• Immunology
• Surgery
• Intensive care medicine

Selected publications

• The Growth of Regional Children's Campuses and Academic Departments of Pediatrics

  The Journal of Pediatrics · 2024-06-12 · 1 citations

  editorial
  PublisherDOI

• Leadership Development Program Outcomes

  The Journal of Pediatrics · 2022-05-06

  editorialSenior author
  PublisherDOI

• Perioperative Transgender Hormone Management: Avoiding Venous Thromboembolism and Other Complications

  Plastic & Reconstructive Surgery · 2021-03-29 · 24 citations

  review

  SUMMARY: This review discusses the current evidence regarding perioperative hormone therapy for transgender individuals, with an emphasis on strategies to reduce the risk of perioperative venous thromboembolism. Historically, surgeons routinely discontinued estrogen therapy in the perioperative period with the goal of reducing the risk of venous thromboembolism. However, abrupt estrogen cessation may also lead to adverse emotional and physiologic effects, including an exacerbation of one's gender dysphoria. The data on the relationship of feminizing hormones and venous thromboembolism in the perioperative setting are largely based on extrapolation of hormone regimens that are no longer in use and may not accurately reflect the actual risk of venous thromboembolism. Future studies will allow surgeons to engage in evidence-based, patient-centered, informed consent while also minimizing the risk of complications, such as venous thromboembolism.

  PublisherDOI

• Dose of Prophylactic Platelet Transfusions and Prevention of Hemorrhage

  UNC Libraries · 2020-11-10

  articleOpen access

  We conducted a trial of prophylactic platelet transfusions to evaluate the effect of platelet dose on bleeding in patients with hypoproliferative thrombocytopenia.

  PublisherDOI

• Characteristics of Hospitalized Children With SARS-CoV-2 in the New York City Metropolitan Area

  Hospital Pediatrics · 2020-12-23 · 54 citations

  article

  OBJECTIVES: To describe the characteristics of hospitalized children with severe acute respiratory syndrome coronavirus 2 in New York City metropolitan area. PATIENTS AND METHODS: This was a multicenter, retrospective cohort study at 4 hospitals comprising 82 hospitalized children (0–21 years) who tested positive for severe acute respiratory syndrome coronavirus 2 after symptoms and risk screening between March 1 and May 10, 2020. We subdivided patients on the basis of their admission to acute or critical care units and by age groups. Further subanalyses were performed between patients requiring respiratory support or no respiratory support. RESULTS: Twenty-three (28%) patients required critical care. Twenty-nine (35%) patients requiring respiratory support, with 9% needing mechanical ventilation, and 1 required extracorporeal support. All patients survived to discharge. Children with any comorbidity were more likely to require critical care (70% vs 37%, P = .008), with obesity as the most common risk factor for critical care (63% vs 28%, P = .02). Children with asthma were more likely to receive respiratory support (28% vs 8%, P = .02), with no difference in need for critical care (P = .26). Children admitted to critical care had higher rates of renal dysfunction at presentation (43% vs 10%, P = .002). CONCLUSIONS: Children with comorbidities (obesity and asthma in particular) were at increased risk for critical care admission and/or need for respiratory support. Children with renal dysfunction at presentation were more likely to require critical care.

  PublisherDOI

• Transfusion Therapy for Coagulation Factor Deficiencies

  Elsevier eBooks · 2017-09-08 · 2 citations

  book-chapterSenior author
  PublisherDOI

• [Corticosteroid treatment for a first episode of steroid-sensitive nephrotic syndrome (SSNS) in children: guideline from the Italian Society of Nephrology].

  PubMed · 2015-08-20

  article

  BACKGROUND: The current 3rd edition of the Italian Society of Nephrology guidelines has been drawn up to summarize evidence of key intervention issues on the basis of systematic reviews (SR) of randomized trials (RCT) or RCT data only. In the present guideline, evidence of the use of corticosteroid treatment for a first episode of steroid-sensitive nephrotic syndrome (SSNS) in children is presented. METHODS: SR of RCT and RCT on SSNS therapeutic interventions were identified referring to a Cochrane Library and Renal Health Library search (2005 update). Results. One SR including 15 RCT was available on this topic. Methodological quality of available RCT was suboptimal according to current methodological standards. RESULTS: In children with a first episode of SSNS, corticosteroids administered for 3 months or more compared with 2 months' administration are associated with a significant reduction in the risk of relapse at 6, 12 and 24 months, and in frequent relapsing rates, even though complications did not seem significantly increased (psychological, ocular, gastrointestinal disorders, hypertension, growth delay, Cushingoid syndrome, infection and osteoporosis) (evidence from SR). 6-month compared to 3-month treatment regimens are associated with a significant reduction in the risk of relapse at 12-24 months (evidence from SR). Increasing steroids cumulative doses are associated with increasing improvements in the risk of relapse (evidence from RCT). The risk of relapse at 12-24 months correlates inversely with duration of treatment (evidence from SR). CONCLUSION: In SSNS children, current available evidence supports the hypothesis that primary intervention should be a high dose of corticosteroids administered for 3 months or more. Further studies are necessary to test this hypothesis in adult patients.

  Publisher

• [Evidence-based guidelines in nephrology].

  PubMed · 2015-09-17

  article

  INTRODUCTION: The preparation of evidence-based guidelines by the Nephrology Societies is fundamental to improve long-term outcomes of patients with chronic kidney diseases. However, this is a complex process and requires the interaction of clinicians and experts in epidemiology methods, and researchers and research enterprises. METHODS: In this review, we present the potential structure of a body for the coordination and development of evidence-based guidelines in a nephrology society and we address the major problems that can arise in this process describing strategies that could be used to overcome them. RESULTS: The development of evidence-based nephrology guidelines requires a structure; this should consist of a coordinating center and a number of working groups. The working groups is to identify specific research questions and to develop and synthetize the evidence in answer to the questions proposed. This shall be done in collaboration with the coordinating center. Draft guidelines produced by this process should be peer reviewed, disseminated and implemented. CONCLUSIONS: The development of evidence-based nephrology guidelines is a challenge for individual nephrology societies. These guidelines are different from typical research publications in that their success does not lie in the final publication, but in the actual dissemination and implementation, which is in the improvement of patient outcomes and its measurement.

  Publisher

• Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial

  Blood · 2015-10-27 · 201 citations

  articleOpen access

  Patients with autoimmune multilineage cytopenias are often refractory to standard therapies requiring chronic immunosuppression with medications with limited efficacy and high toxicity. We present data on 30 patients treated on a multicenter prospective clinical trial using sirolimus as monotherapy. All children (N = 12) with autoimmune lymphoproliferative syndrome (ALPS) achieved a durable complete response (CR), including rapid improvement in autoimmune disease, lymphadenopathy, and splenomegaly within 1 to 3 months of starting sirolimus. Double-negative T cells were no longer detectable in most, yet other lymphocyte populations were spared, suggesting a targeted effect of sirolimus. We also treated 12 patients with multilineage cytopenias secondary to common variable immunodeficiency (CVID), Evans syndrome (ES), or systemic lupus erythematosus (SLE), and most achieved a CR (N = 8), although the time to CR was often slower than was seen in ALPS. Six children with single-lineage autoimmune cytopenias were treated and only 2 responded. Sirolimus was well tolerated with very few side effects. All of the responding patients have remained on therapy for over 1 year (median, 2 years; range, 1 to 4.5 years). In summary, sirolimus led to CR and durable responses in a majority of children with refractory multilineage autoimmune cytopenias. The responses seen in ALPS patients were profound, suggesting that sirolimus should be considered as a first-line, steroid-sparing treatment of patients needing chronic therapy. The results in other multilineage autoimmune cytopenia cohorts were encouraging, and sirolimus should be considered in children with SLE, ES, and CVID. This trial was registered at www.clinicaltrials.gov as #NCT00392951.

  PublisherOA PDFDOI

• [Treating lupus nephritis: guideline from the Italian Society of Nephrology].

  PubMed · 2015-08-20 · 4 citations

  article

  BACKGROUND: The current 3rd edition of the Italian Society of Nephrology guidelines has been drawn up to summarize evidence of key intervention issues on the basis of systematic reviews (SR) of randomized trials (RCT) or RCT data only. In the present guideline, evidence of lupus nephritis (LN) treatment is presented. METHODS: SR of RCT and RCT on different therapeutic options for LN were identified referring to a Cochrane Library and Renal Health Library search (2005 update). RESULTS: One SR of 25 RCT and 6 further RCT were available to address this issue. Methodological quality of available RCT was suboptimal according to current methodological standards. In LN patients, combining cyclophosphamide (CyA) and steroids as induction therapy results in a reduced risk of serum creatinine doubling compared to steroids alone, although there is no evidence of significant survival advantage and risk of ovarian failure was demonstrated (evidence from SR). The association of azathioprine (Aza) and steroids significantly reduces the risk of all-cause mortality compared to steroids alone (evidence from SR). No significant survival advantages from the association of plasma exchange and CyA or Aza are proven (evidence from SR). No significant differences on renal and survival endpoints are demonstrated with different dosing of CyA (evidence from RCT). CONCLUSION: In LN patients available evidence supports the hypothesis that immunosuppressive agents reduce the risk of all-cause mortality and the risk of progressive renal disease. Further studies are necessary to test new immunosuppressive agents such as mycophenolate mofetil in severe LN patients.

  Publisher

Recent grants

• NIH Grant M01RR000240

  NIH · $6.4M · 2006

• NIH Grant U01HL066948

  NIH · $28.5M · 2007

• NIH Grant R01HL071518

  NIH · $1.3M · 2005

Frequent coauthors

• Katherine A. High

  Rockefeller University

  102 shared

• Margaret V. Ragni

  University of Pittsburgh

  75 shared

• Peter J. Larson

  Jackson Laboratory

  75 shared

• Roland W. Herzog

  Indiana University – Purdue University Indianapolis

  72 shared

• Bertil Glader

  Stanford Medicine

  69 shared

• Margareth C. Ozelo

  Universidade Estadual de Campinas (UNICAMP)

  69 shared

• Mark A. Kay

  Stanford University

  69 shared

• Alexis A. Thompson

  University of Pennsylvania

  68 shared