B-Cell and T-Cell Combined Disorders Treatment & Management
- Author: Terry W Chin, MD, PhD; Chief Editor: Harumi Jyonouchi, MD more...
As with other immunodeficiencies, aggressive antibiotic administration and supportive care may prolong the patient's survival, though no current therapy cures ataxia-telangiectasia (AT). Careful observation for the early development of AT is indicated because patients with T-cell deficiency have an increased susceptibility to develop malignancies. Likewise, regular determination of serum autoantibody level and constant clinical evaluation for endocrinopathy (eg, hypoparathyroidism, hypoadrenalism, diabetes) is needed in patients with chronic mucocutaneous candidiasis (CMC).
Unlike other combined immunodeficiency syndromes, AT and CMC do not generally warrant gamma-globulin replacement therapy because of the marked variation in humoral immunodeficiency with the concomitant variable susceptibility to infections. On the other hand, an individual patient may benefit from such treatment. If a trial of intravenous immunoglobulin (IVIG) is considered in these patients, the dosage is 400-600 mg/kg every 2-4 weeks for 6 months. A high dosage is indicated in those with bronchiectasis. Monitor the patient's clinical response rather than specific serum immunoglobulin G (IgG) levels.
Bone marrow transplantation is difficult to justify because of potential adverse effects of cellular radiosensitivity in patients with AT. Transplantation is also unlikely to alter the progressive neurologic symptoms of the disease. The present authors know of no report of successful bone marrow transplantation in a patient with CMC.
The Primary Immune Deficiency Treatment Consortium analyzed the results of hematopoietic stem cell transplantation in children with SCID. Researchers collected data from 240 infants who had received transplants at 25 centers from 2000-2009 and concluded that transplants from donors other than matched siblings resulted in high survival rates if the infants were identified before infection developed. Asymptomatic infants responded well to all graft sources.
Use of thymic hormones (eg, thymosin) offers promise, but, to the author's knowledge, no clinical studies have been conducted.
Irradiation of cellular blood products is indicated in patients with AT and CMC to prevent transfusion-associated graft versus host disease.
Treatment of patients with AT who also have malignancies requires extremely careful planning and caution in the use of chemotherapy because of their increased chemosensitivity.
Because patients with AT and Nijmegen breakage syndrome (NBS) have an increased risk of developing malignancy, careful monitoring is required by a hematologist-oncologist. Because patients with CMC may be at risk of developing various endocrinopathies, careful monitoring is required by an endocrinologist. Thymoma may also develop in adulthood. Regular screening by a gastroenterologist has been suggested for patients with CMC with a history of recurrent candidal esophagus or a family history positive for esophageal or oral carcinoma.
Primary care physicians who are less experienced in interpreting results of immune function tests should refer patients to an immunologist.
Refer parents of children with AT and CMC to a genetic counselor because they are at risk for affected additional offspring.
The poor growth in patients with AT and NBS has not been shown to respond to nutritional intervention.
Because of the increased sensitivity to radiation in patients with AT or NBS, advise these patients to avoid excessive sun exposure and to use sunscreens when outdoors.
The typical patient with AT usually requires a wheelchair for mobility by early teenage years.
For patients with CMC, good oral hygiene and aggressive treatment of oral and esophageal candidiasis are needed.
Avoidance of additional risk factors for oral and esophageal cancer such as cigarette smoking and excessive alcohol consumption may also be warranted.
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|Brand(Manufacturer)||Manufacturing Process||pH||Additives (IVIG products containing sucrose are more often associated with renal dysfunction, acute renal failure, and osmotic nephrosis, particularly with preexisting risk factors [eg, history of renal insufficiency, diabetes mellitus, age >65 y, dehydration, sepsis, paraproteinemia, nephrotoxic drugs].)||Parenteral Form and Final Concentrations||IgA Content mcg/mL|
|Kistler-Nitschmann fractionation, pH 4, nanofiltration||6.4-6.8||6% solution: 10% sucrose, < 20 mg NaCl/g protein||Lyophilized powder 3%, 6%, 9%, 12%||Trace|
|Cohn-Oncley fractionation, PEG precipitation, ion-exchange chromatography, pasteurization||5.1-6||Sucrose free, contains 5% D-sorbitol||Liquid 5%||< 50|
|Gammagard Liquid 10%
|Cohn-Oncley cold ethanol fractionation, cation and anion exchange chromatography, solvent detergent treated, nanofiltration, low pH incubation||4.6-5.1||0.25 M glycine||Ready-for-use liquid 10%||37|
|Cohn-Oncley fraction II/III, ultrafiltration, pasteurization||6.4-7.2||5% solution: 5% sucrose, 3% albumin, 0.5% NaCl||Lyophilized powder 5%||< 20|
|Cohn-Oncley fractionation, caprylate-chromatography purification, cloth and depth filtration, low pH incubation||4-4.5||Contains no sugar, contains glycine||Liquid 10%||46|
|Solvent/detergent treatment targeted to enveloped viruses; virus filtration using Pall Ultipor to remove small viruses including nonenveloped viruses; low pH incubation||4.8-5.1||Contains sorbitol (40 mg/mL); do not administer if fructose intolerant||Ready-for-use solution 5%||< 10|
|Cohn-Oncley fraction II/III, ultrafiltration, pasteurization||6.4-7.2||5% solution: 5% glucose, 0.3% NaCl||Lyophilized powder 5%||< 10|
(Baxter Bioscience for the American Red Cross)
|Cohn-Oncley cold ethanol fractionation, followed by ultra centrafiltration and ion exchange chromatography, solvent detergent treated||6.4-7.2||5% solution: 0.3% albumin, 2.25% glycine, 2% glucose||Lyophilized powder 5%, 10%||< 1.6 (5% solution)|
9/24/10: Withdrawn from market because of unexplained reports of thromboembolic events
|Cohn-Oncley fraction II/III, ultrafiltration, low pH incubation, S/D treatment pasteurization||5.1-6||10% maltose||Liquid 5%||200|
(Swiss Red Cross for the American Red Cross)
|Kistler-Nitschmann fractionation, pH 4, trace pepsin, nanofiltration||6.6||Per gram of IgG: 1.67 g sucrose, < 20 mg NaCl||Lyophilized powder 3%, 6%, 9%, 12%||720|