Outcomes after Unrelated Umbilical Cord Blood Transplantation for Children with Osteopetrosis

Chiesa, Robert; Ruggeri, Annalisa; Paviglianiti, Annalisa; Zecca, Marco; González‐Vicent, Marta; Bordon, Victoria; Stein, Jerry; Lawson, Sarah; Dupont, Sophie; Lanino, Edoardo; Abecasis, Manuel; Al-Seraihy, Amal; Kenzey, Chantal; Bierings, Marc; Locatelli, Franco; Gluckman, Éliane; Schulz, Ansgar; Gennery, Andrew R.; Page, Kristin; Kurtzberg, Joanne; Rocha, Vanderson

doi:10.1016/j.bbmt.2016.07.015

Cited by 20 publications

(13 citation statements)

References 16 publications

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“…The prognosis is poor with most untreated children not surviving past their first decade. For most genetic forms, allogeneic HSCT from MSD, MUD or MMAD is recommended as an effective therapeutic option for these patients, with resolution of skeletal radiographic features, and with recent improved outcomes using reducedintensity protocols [254][255][256][257].…”

Section: Osteopetrosismentioning

confidence: 99%

Indications for haematopoietic stem cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2019

Duarte

Labopin

Bader

et al. 2019

Bone Marrow Transplant

239

172

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This is the seventh special EBMT report on the indications for haematopoietic stem cell transplantation for haematological diseases, solid tumours and immune disorders. Our aim is to provide general guidance on transplant indications according to prevailing clinical practice in EBMT countries and centres. In order to inform patient decisions, these recommendations must be considered together with the risk of the disease, the risk of the transplant procedure and the results of non-transplant strategies. In over two decades since the first report, the EBMT indications manuscripts have incorporated changes in transplant practice coming from scientific and technical developments in the field. In this same period, the establishment of JACIE accreditation has promoted high quality and led to improved outcomes of patient and donor care and laboratory performance in transplantation and cellular therapy. An updated report with operating definitions, revised indications and an additional set of data with overall survival at 1 year and non-relapse mortality at day 100 after transplant in the commonest standard-of-care indications is presented. Additional efforts are currently underway to enable EBMT member centres to benchmark their risk-adapted outcomes as part of the Registry upgrade Project 2020 against national and/or international outcome data.

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Section: Osteopetrosismentioning

confidence: 99%

Indications for haematopoietic stem cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2019

Duarte

Labopin

Bader

et al. 2019

Bone Marrow Transplant

239

172

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“…In cases where no matched related donor is found, we immediately initiate a search for an unrelated donor in the national and international registries. Umbilical cord blood transplantations for MIOP should be avoided as they are known to have poor outcomes, with high rates of graft failures 36 . However, haploidentical transplantation is a feasible alternative option, 37 especially with new modalities such as alpha‐beta T‐cell depletion and posttransplant cyclophosphamide 38,39 .…”

Section: Donor Selectionmentioning

confidence: 99%

How we approach malignant infantile osteopetrosis

Even‐Or

Stepensky

2020

Pediatric Blood & Cancer

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Malignant infantile osteopetrosis (MIOP) is a rare hereditary disorder characterized by excessive bone overgrowth due to a defect in bone marrow resorption by osteoclasts. In most cases, hematopoietic stem cell transplantation (HSCT) may correct bone metabolism but the rapidly progressing nature of this condition necessitates early diagnosis and prompt treatment to minimize irreversible cranial nerve damage. The management of patients with MIOP presents many unique challenges. In this review, the clinical management of patients with MIOP is discussed, including diagnosis, preparation for HSCT and special transplant considerations, management of unique HSCT complications, and long-term follow-up.

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“…The most recent report of transplants performed by Ulm and Paris Transplant Units highlighted the improved outcomes of HSCT with 93% of survival using T cell replete matched donor and 80% of survival using T cell depleted haploidentical donor (7). Unrelated cord blood is not recommended because its use is associated to high risk of primary engraftment failure (73). Fludarabine-based conditioning, performed better than the conventional cyclophosphamide-based one, in terms of higher engraftment and reduced toxicity with a higher 5-years DFS.…”

Section: Current Therapies and Management Of Osteopetrosismentioning

confidence: 99%

One Disease, Many Genes: Implications for the Treatment of Osteopetroses

et al. 2019

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Osteopetrosis is a condition characterized by increased bone mass due to defects in osteoclast function or formation. In the last decades, the molecular dissection of osteopetrosis has unveiled a plethora of molecular players responsible for different forms of the disease, some of which present also primary neurodegeneration that severely limits the therapy. Hematopoietic stem cell transplantation can cure the majority of them when performed in the first months of life, highlighting the relevance of an early molecular diagnosis. However, clinical management of these patients is constrained by the severity of the disease and lack of a bone marrow niche that may delay immune reconstitution. Based on osteopetrosis genetic heterogeneity and disease severity, personalized therapies are required for patients that are not candidate to bone marrow transplantation. This review briefly describes the genetics of osteopetrosis, its clinical heterogeneity, current therapy and innovative approaches undergoing preclinical evaluation.

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Outcomes after Unrelated Umbilical Cord Blood Transplantation for Children with Osteopetrosis

Cited by 20 publications

References 16 publications

Indications for haematopoietic stem cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2019

Indications for haematopoietic stem cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2019

How we approach malignant infantile osteopetrosis

One Disease, Many Genes: Implications for the Treatment of Osteopetroses

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