Activated PI3-kinase δ Syndrome: Long-term Follow-up after Cord Blood Transplantation

Early immune disorders, including preengraftment syndrome (PES)and acute graft-versus-host disease(aGvHD)are problematic in cord blood transplantation (CBT), and optimal prophylaxis has not been established. Here, we prospectively investigated whether intensive GvHD prophylaxis by adding low-dose methotrexate (MTX)at day 3 after CBThas a prognostic impact on CBT. 16 consecutive single-unit CBT recipients treated for high-risk hematologic malignancies (10 cases) and non-malignancies (6 cases) between February 2019 and March 2019. The patients, 6 female and 10 males, had a median age of 9 years (range 4-40) and a median weight of 26 kg (range 14-68). Myeloablative preparative regimen comprised fludarabine, busulfan and cyclophosphamide for malignancies and reduced-intensity-conditioning comprised fludarabine, cyclophosphamide and total-body irradiation (4 Gy) for non-malignancies. Graft-versus-host disease (GvHD) prophylaxis was cyclosporine and mycophenolate mofetil plus MTX 3 mg/m2 on day+3. 14 patients achieved engraftment at a median of day 19 and had more than 95% (complete) donor chimerism on day+14.Two patients without hematopoietic reconstitution were engrafted after a second CBT. PES was characterized by high-grade fever, rash, pulmonary edema, weight gain, liver and renal dysfunction developed on a median of day 8 in 11 of the 16 evaluable patients, including 2 who did not achieve engraftment. The PES patients received intravenous corticosteroid at a median dose of 1 mg/kg/day, and of these, 2 patients were requiredBasiliximab to improve clinical symptoms. Grade I to IV and aGvHD developed in 4 and only 1 patient developed severe aGvHD with Grade Ⅲ. Follow-up so far, one death occurred at day 103 because of pneumonia. 15 patients are alive well at a median follow-up of 135 days (131-163). Adding low-dose MTX at day +3 may be offer one optimal regimen to reduce severe early immune reactions and improve outcomes in CBT. Disclosures No relevant conflicts of interest to declare.

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Effects of Cryopreservation Duration on the Outcome of Single-Unit Cord Blood Transplantation

Cell Transplantation ◽

10.1177/0963689717753187 ◽

2018 ◽

Vol 27 (3) ◽

pp. 515-519 ◽

Cited By ~ 1

Author(s):

Tang-Her Jaing ◽

Shih-Hsiang Chen ◽

Yu-Chuan Wen ◽

Tsung-Yen Chang ◽

Ya-Chun Yang ◽

...

Keyword(s):

Cord Blood ◽

Clinical Outcomes ◽

Progenitor Cell ◽

Cell Function ◽

Cord Blood Transplantation ◽

Blood Transplantation ◽

The Impact ◽

Transplantation Outcomes ◽

Neutrophil Engraftment

Cryopreservation is widely used in umbilical cord blood (UCB) banking, yet its impact on progenitor cell function remains largely unaddressed. It is unknown whether long-term cryopreservation affects UCB transplantation outcomes. Herein, we evaluated the impact of UCB age on clinical outcomes and investigated the effect of cryopreservation duration of UCB on hematopoietic potency in 91 patients receiving single cord blood transplantations. UCB cryopreservation duration was 0.7 to 13.4 y. The most common indication of transplant was thalassemia (48%). There was no significant association between cryopreservation duration and neutrophil engraftment probability ( P = 0.475). Cryopreservation duration did not affect the post-thaw viability and subsequent neutrophil engraftment rate. Therefore, UCB units can undergo cryopreservation for at least 8 y with no impact on clinical outcomes.

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Clinical and neuropathologic findings in two long-term survivors of Krabbe disease with and without umbilical cord blood transplantation

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2018.12.199 ◽

2019 ◽

Vol 126 (2) ◽

pp. S81

Author(s):

Kofler Julia ◽

Maria L. Beltran-Quintero ◽

Michele Poe ◽

Maria L. Escolar

Keyword(s):

Cord Blood ◽

Umbilical Cord ◽

Umbilical Cord Blood ◽

Cord Blood Transplantation ◽

Krabbe Disease ◽

Umbilical Cord Blood Transplantation ◽

Blood Transplantation ◽

Long Term Survivors

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Cord blood transplantation provides better reconstitution of hematopoietic reservoir compared with bone marrow transplantation

Blood ◽

10.1182/blood-2003-03-0720 ◽

2003 ◽

Vol 102 (3) ◽

pp. 1138-1141 ◽

Cited By ~ 53

Author(s):

Francesco Frassoni ◽

Marina Podestà ◽

Rita Maccario ◽

Giovanna Giorgiani ◽

Gabriele Rossi ◽

...

Keyword(s):

Stem Cells ◽

Bone Marrow ◽

Cord Blood ◽

Mononuclear Cells ◽

Cord Blood Transplantation ◽

Hematopoietic Progenitor Cell ◽

Transplant Recipients ◽

Platelet Recovery ◽

Blood Transplantation

Abstract Delayed hematopoietic recovery is the main factor precluding a wider use of cord blood (CB) transplants. We hypothesized that this delayed engraftment might not be related to an insufficient number of stem cells in the graft, but to an intrinsic difficulty of these cells to undergo differentiation. To test our hypothesis, 2 groups of children were compared; 12 received a CB transplant and 12 an adult bone marrow (BM) transplant. We studied neutrophil and platelet recovery and, at a median time of approximately 1 year after transplantation, the frequency of colony-forming cells (CFCs) and long-term culture initiating cells (LTC-ICs) in the BM of the 2 groups. Recipients of BM transplants received 1-log more cells and had significantly faster neutrophil and platelet recovery. Conversely, the frequency of committed and early progenitors was significantly higher in the BM of children given CB cells compared with BM transplant recipients (median count of CFC/2 × 104 BM mononuclear cells, 20 versus 11, P = .007; median count of LTC-IC/106 BM mononuclear cells, 8.2 versus 0.2 P = .001). CB, but not adult BM stem cells, can better restore the host hematopoietic progenitor cell reservoir; the delayed engraftment after CB transplantation may reflect the difficulty of CB progenitors to reprogram themselves toward differentiation.

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