ArticleBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2017
Haploidentical Related Donor Hematopoietic Stem Cell Transplantation for Dedicator-of-Cytokinesis 8 Deficiency Using Post-Transplantation Cyclophosphamide.
Article in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 28 papers.
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Who cites it
28 citing papers in PubMed, 49 citations in OpenAlex.
- Hematopoietic cell transplantation for DOCK8 deficiency: Results from a prospective clinical trial.The Journal of allergy and clinical immunology · 2025Trial
- Prospective Study of a Novel, Radiation-Free, Reduced-Intensity Bone Marrow Transplantation Platform for Primary Immunodeficiency Diseases.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2020Trial
- Pediatric DOCK8 Deficiency Beyond Infections and Atopy: An Immunoactinopathy with Immune Dysregulation and Multisystem Involvement.Children (Basel, Switzerland) · 2026Article
- Haploidentical stem cell transplantation in DOCK8 deficiency: a case report of successful outcomes.Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis · 2025Article
- High malignancy rate in IgE-deficient children.International journal of cancer · 2025Observational
- Secondary bone marrow graft loss after third-party virus-specific T cell infusion: Case report of a rare complication.Nature communications · 2024Article
- Case report: Advanced age at transplantation and pre-emptive treatment with dupilumab in DOCK8 deficiency.Frontiers in immunology · 2024Article
- Atypical Localization of Eczema Discriminates DOCK8 or STAT3 Deficiencies from Atopic Dermatitis.Journal of clinical immunology · 2023Article
- Successful treatment of DOCK8 deficiency by allogeneic hematopoietic cell transplantation from alternative donors.International journal of hematology · 2023Article
- Tandem hematopoietic stem cell transplant considerations in families with multiple siblings affected by DOCK8 deficiency.Bone marrow transplantation · 2022Article
- Contemporary haploidentical stem cell transplant strategies in children with hematological malignancies.Bone marrow transplantation · 2021Review
- Case Report: Fatal Complications of BK Virus-Hemorrhagic Cystitis and Severe Cytokine Release Syndrome Following BK Virus-Specific T-Cells.Frontiers in immunology · 2021Article
- Allogeneic hematopoietic stem cell transplantation in adults with primary immunodeficiency.Hematology. American Society of Hematology. Education Program · 2020Review
- Clinical, immunological features and follow up of 20 patients with dedicator of cytokinesis 8 (DOCK8) deficiency.Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology · 2020Article
- HLA-Haploidentical Hematopoietic Cell Transplantation for Treatment of Nonmalignant Diseases Using Nonmyeloablative Conditioning and Post-Transplant Cyclophosphamide.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2020Article
- T-cell replete haploidentical bone marrow transplantation and post-transplant cyclophosphamide for patients with inborn errors.Haematologica · 2019Article
- Tandem Orthotopic Living Donor Liver Transplantation Followed by Same Donor Haploidentical Hematopoietic Stem Cell Transplantation for DOCK8 Deficiency.Transplantation · 2019Article
- Compound Heterozygous DOCK8 Mutations in a Patient with B Lymphoblastic Leukemia and EBV-Associated Diffuse Large B Cell Lymphoma.Journal of clinical immunology · 2019Article
- Hematopoietic stem cell transplant effectively rescues lymphocyte differentiation and function in DOCK8-deficient patients.JCI insight · 2019Article
- Hematopoietic Stem Cell Transplantation as Treatment for Patients with DOCK8 Deficiency.The journal of allergy and clinical immunology. In practice · 2019Article
Corrections and comments
- Commented on by
- Erratum issued
Authors and funding
12 authors at 7 institutions in 2 countries.
Funding
Abstract
Dedicator-of-cytokinesis 8 (DOCK8) deficiency, a primary immunodeficiency disease, can be reversed by allogeneic hematopoietic stem cell transplantation (HSCT); however, there are few reports describing the use of alternative donor sources for HSCT in DOCK8 deficiency. We describe HSCT for patients with DOCK8 deficiency who lack a matched related or unrelated donor using bone marrow from haploidentical related donors and post-transplantation cyclophosphamide (PT/Cy) for graft-versus-host disease (GVHD) prophylaxis. Seven patients with DOCK8 deficiency (median age, 20 years; range, 7 to 25 years) received a haploidentical related donor HSCT. The conditioning regimen included 2 days of low-dose cyclophosphamide, 5 days of fludarabine, 3 days of busulfan, and 200 cGy total body irradiation. GVHD prophylaxis consisted of PT/Cy 50 mg/kg/day on days +3 and +4 and tacrolimus and mycophenolate mofetil starting at day +5. The median times to neutrophil and platelet engraftment were 15 and 19 days, respectively. All patients attained >90% donor engraftment by day +30. Four subjects developed acute GVHD (1 with maximum grade 3). No patient developed chronic GVHD. With a median follow-up time of 20.6 months (range, 9.5 to 31.7 months), 6 of 7 patients are alive and disease free. Haploidentical related donor HSCT with PT/Cy represents an effective therapeutic approach for patients with DOCK8 deficiency who lack a matched related or unrelated donor.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.