Treatment of patients with immunodeficiency: Medication, gene therapy, and transplantation

Segundo, Gesmar Rodrigues Silva; Condino‐Neto, Antônio

doi:10.1016/j.jped.2020.10.005

Cited by 14 publications

(16 citation statements)

References 37 publications

(44 reference statements)

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“…Examples of antibiotic prophylaxis regimens used in patients with immunodeficiency are Pneumocystis jirovecii : sulfamethoxazole-trimethoprim (SXT-TMP), infants >4 weeks of age and children, 5 mg/kg/day divided into two doses 3x/week (based on TMP, maximum 160 mg per day). Adults and adolescents with normal kidney function: based on TMP 80 mg/day or 160 mg daily or 160 mg 3x/week [ 96 ]. Staphylococcus spp, Gram-negative spp: SXT-TMP.…”

Section: Reviewmentioning

confidence: 99%

“…Infants >4 weeks of age and children: 5 mg/kg/day divided into two daily doses (based on TMP, maximum 160 mg per day). Adults and adolescents: based on TMP 160 mg daily [ 96 ].…”

Section: Reviewmentioning

confidence: 99%

“…Because antibody production is lacking or insufficient in around 50% to 75% of IEI patients, immunoglobulin replacement is necessary. Consequently, it is important to keep in mind the rise in secondary immunodeficiency cases that impair antibody production along with improvements in the treatment of lymphomas, leukemias, and other cancers [ 96 ]. Regarding the therapeutic benefit, immunoglobulin replacement therapy indications can be classified as 1) Proven benefit: immune system defects that affect B-cells, hypogammaglobulinemia, and inefficient antibody production; 2) likely benefit: immunoglobulins with apparently normal levels but with a qualitative defect in the specific antibody production; and 3) no benefit/contraindicated: selective IgA deficiency and IgG4 deficiency [ 96 ].…”

Section: Reviewmentioning

confidence: 99%

See 2 more Smart Citations

Guardians of Immunity: Advances in Primary Immunodeficiency Disorders and Management

Peddi,

Vuppalapati,

Sreenivasulu

et al. 2023

Cureus

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Primary immunodeficiency disorders (PIDs) are a heterogeneous group of genetic conditions profoundly impacting immune function. The investigation spans various PID categories, offering insights into their distinct pathogenic mechanisms and clinical manifestations. Within the adaptive immune system, B-cell, T-cell, and combined immunodeficiencies are dissected, emphasizing their critical roles in orchestrating effective immune responses. In the realm of the innate immune system, focus is directed toward phagocytes and complement deficiencies, underscoring the pivotal roles of these components in initial defense against infections. Furthermore, the review delves into disorders of immune dysregulation, encompassing hemophagocytic lymphohistiocytosis (HLH), autoimmune lymphoproliferative syndrome (ALPS), immune dysregulation, polyendocrinopathy, enteropathy, and X-linked(IPEX), and autoimmunity polyendocrinopathy candidiasis-ectodermal dystrophy(APECED), elucidating the intricate interplay between immune tolerance and autoimmunity prevention. Diagnostic strategies for PIDs are explored, highlighting advancements in genetic and molecular techniques that enable precise identification of underlying genetic mutations and alterations in immune function. We have also outlined treatment modalities for PIDs, which often entail a multidisciplinary approach involving immunoglobulin replacement, antimicrobial prophylaxis, and, in select cases, hematopoietic stem cell transplantation. Emerging therapies, including gene therapy, hold promise for targeted interventions. In essence, this review encapsulates the complexity of PIDs, emphasizing the critical importance of early diagnosis and tailored therapeutic interventions. As research advances, a clearer understanding of these disorders emerges, fostering optimism for enhanced patient care and management in the future.

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Section: Reviewmentioning

confidence: 99%

“…Infants >4 weeks of age and children: 5 mg/kg/day divided into two daily doses (based on TMP, maximum 160 mg per day). Adults and adolescents: based on TMP 160 mg daily [ 96 ].…”

Section: Reviewmentioning

confidence: 99%

Section: Reviewmentioning

confidence: 99%

See 1 more Smart Citation

Guardians of Immunity: Advances in Primary Immunodeficiency Disorders and Management

Peddi,

Vuppalapati,

Sreenivasulu

et al. 2023

Cureus

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“…Prophylactic therapy with antibiotics, antifungal, antiviral and antiparasitic drugs are wider implemented. These include a wide range of agents and sometimes the same drug is used not only for prophylaxis but also for treatment of a confirmed or suspected infection ( 18 ).…”

Section: Overcoming Susceptibility To Infection With Precision Medicinementioning

confidence: 99%

Precision medicine: The use of tailored therapy in primary immunodeficiencies

Pinto

Neves

2022

Front. Immunol.

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Primary immunodeficiencies (PID) are rare, complex diseases that can be characterised by a spectrum of phenotypes, from increased susceptibility to infections to autoimmunity, allergy, auto-inflammatory diseases and predisposition to malignancy. With the introduction of genetic testing in these patients and wider use of next-Generation sequencing techniques, a higher number of pathogenic genetic variants and conditions have been identified, allowing the development of new, targeted treatments in PID. The concept of precision medicine, that aims to tailor the medical interventions to each patient, allows to perform more precise diagnosis and more importantly the use of treatments directed to a specific defect, with the objective to cure or achieve long-term remission, minimising the number and type of side effects. This approach takes particular importance in PID, considering the nature of causative defects, disease severity, short- and long-term complications of disease but also of the available treatments, with impact in life-expectancy and quality of life. In this review we revisit how this approach can or is already being implemented in PID and provide a summary of the most relevant treatments applied to specific diseases.

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“…Since then, additional literature has emerged regarding the benefits of genetic testing of patients with a severe CVID phenotype, as it can uncover a more precise diagnosis in which the hypogammaglobulinemia may just be one piece of the clinical picture [ 81 ]. The push towards a more specific molecular diagnosis is due to the advent of targeted therapy that takes a precision medicine approach to the treatment of primary B cell deficiencies [ 82 , 83 ]. For example, instead of using broad immunosuppression such as systemic corticosteroids to manage cytopenias and lymphoproliferation in patients with activated PIK3CD (APDS-1), a targeted approach such as leniolisib (a selective PI3K δ inhibitor) may be beneficial and have decreased toxicity [ 65 ].…”

Section: The Futurementioning

confidence: 99%

Clinical Aspects of B Cell Immunodeficiencies: The Past, the Present and the Future

Ahmed

Lippner

Khanolkar

2022

Cells

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B cells and antibodies are indispensable for host immunity. Our understanding of the mechanistic processes that underpin how B cells operate has left an indelible mark on the field of clinical pathology, and recently has also dramatically reshaped the therapeutic landscape of diseases that were once considered incurable. Evaluating patients with primary immunodeficiency diseases (PID)/inborn errors of immunity (IEI) that primarily affect B cells, offers us an opportunity to further our understanding of how B cells develop, mature, function and, in certain instances, cause further disease. In this review we provide a brief compendium of IEI that principally affect B cells at defined stages of their developmental pathway, and also attempt to offer some educated viewpoints on how the management of these disorders could evolve over the years.

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Treatment of patients with immunodeficiency: Medication, gene therapy, and transplantation

Cited by 14 publications

References 37 publications

Guardians of Immunity: Advances in Primary Immunodeficiency Disorders and Management

Guardians of Immunity: Advances in Primary Immunodeficiency Disorders and Management

Precision medicine: The use of tailored therapy in primary immunodeficiencies

Clinical Aspects of B Cell Immunodeficiencies: The Past, the Present and the Future

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