Successful Bone-Marrow Transplantation for Infantile Malignant Osteopetrosis

Coccia, Peter F.; Krivit, William; Cervenka, J; Clawson, Carlyle; Kersey, John H.; Kim, Taehwan H.; Nesbit, Mark E.; Ramsay, Norma K.C.; Warkentin, Phyllis I.; Teitelbaum, Steven L.; Kahn, Arnold J.; Brown, David M.

doi:10.1056/nejm198003273021301

Cited by 427 publications

(146 citation statements)

References 33 publications

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“…5 Since 1977, allogeneic haematopoietic stem cell transplantation (HSCT) had been successfully applied in single cases and small groups of AR-OP patients. [6][7][8][9] In 1994, our group reported the results in 69 patients, showing a favourable outcome of HSCT with a (genotype) HLA-identical donor. 10 Treatments with approaches other than HSCT such as corticosteroids, high doses of calcitriol (vitamin D 3 ) and interferon-g, have been disappointing.…”

mentioning

confidence: 98%

Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT report

Driessen

Gerritsen

Fischer

et al. 2003

Bone Marrow Transplant

163

130

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Summary:A retrospective analysis was made of 122 children who had received an allogeneic haematopoietic stem cell transplantation (HSCT) for autosomal recessive osteopetrosis between 1980 and 2001. The actuarial probabilities of 5 years disease free survival were 73% for recipients of a genotype HLA-identical HSCT (n ¼ 40), 43% for recipients of a phenotype HLA-identical or one HLAantigen mismatch graft from a related donor (n ¼ 21), 40% for recipients of a graft from a matched unrelated donor (n ¼ 20) and 24% for patients who received a graft from an HLA-haplotype-mismatch related donor (n ¼ 41). In the latter group, a trend towards improvement was achieved at the end of the study period (17% before 1994, 45% after 1994, P ¼ 0.11). Causes of death after HSCT were graft failure and early transplant-related complications. Severe visual impairment was present in 42% of the children before HSCT. Conservation of vision was better in children transplanted before the age of 3 months. Final height was related to height at the time of HSCT and better preserved in children transplanted early. Most children attended regular school or education for the visually handicapped. At present, HSCT is the only curative treatment for autosomal recessive osteopetrosis and should be offered as early as possible.

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confidence: 98%

Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT report

Driessen

Gerritsen

Fischer

et al. 2003

Bone Marrow Transplant

163

130

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“…Early studies had demonstrated that OCPs originate from the haematopoietic stem cells (Sheven et al, 1986;Hagenaars et al, 1989;Hattersley & Chambers, 1989;Kurihara et al, 1989;Matayoshi et al, 1996) and circulate in PB (Walker, 1973(Walker, , 1975Kahn & Simmons, 1975;Coccia et al, 1980;Faust et al, 1999;. However, whether OCPs possess surface characteristics that distinguish them from other PBMCs (in particular monocytes) is not known.…”

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confidence: 99%

Characterization of osteoclast precursors in human blood

Shalhoub¹,

Elliott

Chiu

et al. 2000

Br J Haematol

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Osteoclast precursors (OCPs) circulate in the mononuclear fraction of peripheral blood (PB), but their abundance and surface characteristics are unknown. Previous studies suggest that the receptor activator for NF‐κB (RANK) on cytokine‐treated OCPs in mouse bone marrow interacts with osteoprotegerin ligand (OPGL/TRANCE/RANKL/ODF) to initiate osteoclast differentiation. Hence, we used a fluorescent form of human OPGL (Hu‐OPGL‐F) to identify possible RANK‐expressing OCPs in untreated peripheral blood mononuclear cells (PBMCs) using fluorescence‐activated cell sorting analysis. Monocytes [CD14‐phycoerythrin (PE) antibody (Ab) positive (+) cells, 10–15% of PBMCs] all (98–100%) co‐labelled with Hu‐OPGL‐F (n > 18). T lymphocytes (CD3‐PE Ab+ cells, 66% of PBMCs) did not bind Hu‐OPGL‐F; however, B cells (CD19‐PE Ab+ cells, 9% of PBMCs) were also positive for Hu‐OPGL‐F. All Hu‐OPGL‐F+ monocytes also co‐labelled with CD33, CD61, CD11b, CD38, CD45 and CD54 Abs, but not CD34 or CD56 Abs. Hu‐OPGL‐F binding was dose dependent and competed with excess Hu‐OPGL. When Hu‐OPGL‐F+, CD14‐PE Ab+, CD33‐PE Ab+, Hu‐OPGL‐F+/CD14‐PE Ab+ or Hu‐OPGL‐F+/CD33‐PE Ab+ cells were cultured with OPGL (20 ng/ml) and colony‐stimulating factor (CSF)‐1 (25 ng/ml), OC‐like cells readily developed. Thus, all freshly isolated monocytes demonstrate displaceable Hu‐OPGL‐F binding, suggesting the presence of RANK on OCPs in PB; also, OCPs within a purified PB monocyte population form osteoclast‐like cells in the complete absence of other cell types in OPGL and CSF‐1 containing medium.

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“…On the other hand, many in vivo studies in animals as well as in man have shown that mononuclear osteoclast precursors may be disseminated through the body via the blood vascular system (5)(6)(7)(8)(9). This has led several investigators to study the capacity of blood monocytes or tissue macrophages to resorb bone mineral by co-culturing them with devitalized bone tissue or particles of dried, ground bone (10)(11)(12)(13)(14).…”

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confidence: 99%

Osteoclast formation from mononuclear phagocytes: role of bone-forming cells.

Burgér¹,

Meer²,

Nijweide³

1984

The Journal of Cell Biology

151

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In a previous study, using co-cultures of embryonic bone rudiments stripped of periosteum, and mononuclear phagocytes of various sources, we found that multinucleated mineral-resorbing osteoclasts developed in vitro from radiosensitive mouse bone marrow mononuclear phagocytes (BMMP). (Burger, E. H., J. W. M. van der Meer, J. S. van de Gevel, C. W. Thesingh, and R. van Furth, 1982, J. Exp. Med. 156:1604-1614. In the present study, this co-culture technique was used to analyze the influence of bone-forming cells on osteoclast formation and bone resorption by BMMP or peritoneal exudate cells (PEC). BMMP or PEC were co-cultured with liver or dead bone, i.e., in the presence or absence of liver boneforming cells. Mineral resorption and osteoclast formation were monitored via 45Ca release from prelabeled live or dead bone followed by histology.Osteoclasts developed from precultured BMMP as indicated by [3H]thymidine labeling, but only in live and not in dead bone. They formed readily from BMMP but only erratically, and after a longer culture period, from PEC. Macrophages from BMMP and PEC invaded live and dead bone rudiments but did not resorb the intact mineralized matrix. In contrast, ground bone powder was resorbed avidly by both cell populations, without formation of osteoclasts.We conclude that live bone-forming cells are required for osteoclast formation from progenitors. Live bone is only resorbed by osteoclasts, and not by macrophages. Osteoclast progenitors are abundant in cultures of BMMP but scarce in PEC, which makes a direct descendance of osteoclasts from mature macrophages unlikely.The nature of the mononuclear precursor cell of the osteoclast and the interactions between bone-forming and bone-resorbing cells are the subject of many recent studies (1, 2). In an earlier paper we presented evidence that multinucleated mineral resorbing osteoclasts will develop in vitro from cultures of proliferating bone marrow mononuclear phagocytes (BMMP) ~ (3). This was demonstrated by co-culturing adult mouse BMMP with noninvaded embryonic mouse long bones that had been stripped of adhering periosteum-perichondrium to remove the endogenous pool of osteoclast precursor cells (4). As the bones were dissected and stripped before cartilage invasion and osteoclast formation had occurred, the stripped bone rudiments essentially contained only chondrocytes and

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Successful Bone-Marrow Transplantation for Infantile Malignant Osteopetrosis

Cited by 427 publications

References 33 publications

Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT report

Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT report

Characterization of osteoclast precursors in human blood

Osteoclast formation from mononuclear phagocytes: role of bone-forming cells.

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